Don’t Drown in Medical Debt

October 2, 2019/in Adrenal Cancer, Adrenal Cancer Access & Affordability, Adrenal Cancer Video AA, Appendix Cancer, Appendix Cancer Access & Affordability, Appendix Cancer Videos AA, Bone Cancer, Bone Cancer Access & Affordability, Bone Cancer Video AA, Brain Cancer, Brain Cancer Access & Affordability, Brain Cancer Video AA, Eye Cancer, Eye Cancer Access & Affordability, Eye Cancer Video AA, General Access and Affordability, General Health Info, General Videos AA /by PEN Editorial Staff

This video was originally published by The SamFund on August 21, 2015, here.

If you’re a young adult with a cancer history, you probably know that feeling in your stomach that you get each time a medical bill shows up, or the phone rings with a collections agency on the other end. As most of us already know well, medical debt can have wide-ranging consequences that negatively impact your credit and your future.

While you may be struggling today, there are steps you can take to reduce and hopefully eventually eliminate that debt entirely! In this Webinar, Monica Bryant, Esq., COO at Triage Cancer will share strategies to: avoid medical debt before you find yourself in over your head, reduce medical debt once it starts to add up, and shift and prioritize your regular expenses.

Additionally, you’ll learn more about resources available to help negotiate medical bills, how credit counseling agencies can be helpful, and why self-advocacy is such an important part of recovering from debt.

Paying for Clinical Trials

This video was originally published by Cancer.gov on September 3, 2019, here.

Paying for Clinical Trials

Find a Clinical Trial

Learn about the different types of costs related to taking part in a clinical trial, and who is expected to pay for which costs.

As you think about taking part in a clinical trial, you will face the issue of how to cover the costs of care. There are two types of costs associated with a clinical trial: patient care costs and research costs.

Patient care costs are those costs related to treating your cancer, whether you are in a trial or receiving standard therapy. These costs are often covered by health insurance. They include:

Doctor visits
Hospital stays
Standard cancer treatments
Treatments to reduce or eliminate symptoms of cancer or side effects from treatment
Lab tests
X-rays and other imaging tests

Research costs are those related to taking part in the trial. Often these costs are not covered by health insurance, but they may be covered by the trial’s sponsor. Examples include:

The study drug
Lab tests performed purely for research purposes
Additional x-rays and imaging tests performed solely for the trial

When you take part in a trial, you may have extra doctor visits that you would not have with standard treatment. During these visits your doctor carefully watches for side effects and your safety in the study. These extra visits can add costs for transportation and child care.

Immunotherapy in the Elderly

October 2, 2019/in Adrenal Cancer, Adrenal Cancer Blog TC, Adrenal Cancer Treatment & Clinical Trials, Appendix Cancer, Appendix Cancer Blog TC, Appendix Cancer Treatment & Clinical Trials, Bone Cancer, Bone Cancer Blog TC, Bone Cancer Treatment & Clinical Trials, Brain Cancer, Brain Cancer Blog TC, Brain Cancer Treatment & Clinical Trials, Eye Cancer, Eye Cancer Blog TC, Eye Cancer Treatment & Clinical Trials, General Blog TC, General Health Info, General Treatment and Clinical Trials /by PEN Editorial Staff

This blog was originally published by Cancer Today by Emma Yasinki here.

Immune checkpoint inhibitors can be effective treatments for elderly people with some types of advanced cancer, but more information is needed on their risks and benefits in this group.

by Emma Yasinski

Photo by graffoto8 / iStock / Getty Images Plus

CHECKPOINT INHIBITORS, a type of immunotherapy drug, help spur the immune system to kill cancer cells. These drugs can be effective treatments for some patients who otherwise would have few options.

Beginning in 2011, with the approval by the U.S. Food and Drug Administration of the first checkpoint inhibitor, seven of these immunotherapy drugs have come onto the market for treatment of various cancer types.

Enthusiasm for these drugs is widespread, including among elderly patients with advanced cancer. Now, some frail elderly patients who might previously have opted out of chemotherapy are choosing immunotherapy in hopes of achieving a long-term response.

But data on immunotherapy side effects and outcomes are more limited in elderly people than in younger patients. Some doctors worry that all the excitement surrounding checkpoint inhibitors is preventing older patients from getting palliative and hospice care that could be more likely to improve their lives.

Rawad Elias, an oncologist at Hartford Hospital in Connecticut, studies immunotherapy in older patients and presented on the topic at the American Society of Clinical Oncology Annual Meeting in Chicago in June 2019. Cancer Today spoke with Elias about the benefits and risks of checkpoint inhibitors and how their availability may affect treatment decisions for older patients.

Q: Are there common misconceptions among patients and families about checkpoint inhibitors?
A: We’re very excited about [immunotherapy] because it’s an option now other than chemotherapy, [but] it doesn’t work in all cancers. Even in the cancer[s] that it works for, it doesn’t work in all patients. And most patients, in fact, do not respond to checkpoint inhibitors.

We often see patients who … ask us, “OK. How about immunotherapy?” And we’ll have to explain that, unfortunately, in your type of cancer, it doesn’t even work.

Q: What do we know about the efficacy of checkpoint inhibitors in older patients?
A: Unfortunately, older adults are underrepresented in clinical trials. Older adults constitute about 60% of cancer patients, and [in] the clinical trials of checkpoint inhibitors, they [made up] about 40% [of participants]. Also, patients who are enrolled on clinical trials are usually the … fit people with [few] medical complications. So we don’t really understand the clinical profile of these drugs in the real-world population.

We did some work in the past looking … if the efficacy of the checkpoint inhibitors is similar across age groups. We published that in the Journal for ImmunoTherapy of Cancer based on [an] age cutoff of 65. The efficacy of checkpoint inhibitors was considerable in younger and older adults. What we don’t know about, though, is what’s the impact of frailty on these medications? And does that make patients more prone to toxicity? Does it make the efficacy of the drug less?

Q: What are the special considerations older patients need to take into account when considering checkpoint inhibitor therapy?
A: What we don’t know about … is the impact of low-grade toxicity or any toxicity on older adults. We tend to call things like fatigue or a little bit of nausea “low-grade” toxicity, but we don’t know the impact of this low-grade toxicity on an 80-year-old person who already has trouble getting out of the house.

When it comes to older patients with an advanced cancer, this is a really critical thing to discuss: What’s your quality of life during this period of time, and what matters most to you as a person? The goal is not to go and treat the cancer. The goal is to treat you as a person. And it’s only you as a patient who gets to determine: What does that mean?

For example, [one of my patients], even though therapy could have been an option for her, she’s a frail older adult. We talked about [the fact that] the impact of treating her with immunotherapy would be potentially more fatigue and coming to the doctor’s office [more frequently]—coming in once every two weeks or once every four weeks … getting bloodwork, waiting in the waiting room to see the doctor and then getting the infusion, then going back home, then coming back again. So the question is: Does that make sense to you? My patient … decided that doesn’t make sense to her based on what we think … [immunotherapy] is going to achieve.

Q: Why are some people concerned that the increasing popularity of checkpoint inhibitors could hinder access to palliative and end-of-life care?
A: Unfortunately, when we’re treating cancer patients, we’re treating a very hard disease and even small things get us excited. In the hype or the excitement about checkpoint inhibitors, many may skip that conversation [about risks and alternatives like palliative care] and go straight to, “Let’s start you on checkpoint inhibitors and see what happens.” And what’s happening in most patients is that they do not respond, and we forget about palliative care which we know, for sure, makes people have a better quality of life, keeps them outside the hospital, keeps them at home. This is not to say older adults should not be treated, but to say that there are concerns about these drugs. They do not work for everyone.

Emma Yasinski is a Florida-based freelance science and medical journalist.

Focusing on Proton Therapy

This blog was originally published by Cancer Today by Sue Rochman here.

Proton therapy, an alternative to standard radiation therapy, is safe and effective. But evidence is lacking that it’s always a better option than standard radiation, and some insurers balk at the higher price tag.

by Sue Rochman

Photo by gorodenkoff / iStock / Getty Images Plus

IN AUGUST 2017, Hauli Sioux Warrior Gray noticed a lump in her left breast. Two months later, after having seen three different health care providers, the then 33-year-old mother of two from Yukon, Oklahoma, learned she had stage IIB breast cancer. In November, she started chemotherapy to shrink the 7-centimeter tumor in her left breast and kill the cancer cells that had spread to her lymph nodes. In March 2018, she had a mastectomy. When it was time to start radiation, Gray says, her radiation oncologist at the Integris Cancer Institute in Oklahoma City explained that proton therapy would be a better option than standard radiation therapy because “it would save my heart and lungs.”

Gray’s doctor sent a treatment proposal for proton therapy to her health insurer. The request was denied. “I didn’t know insurance companies did that,” says Gray. Aided by a media consultant brought in by her doctor, Gray used social media and local news outlets to tell her story. Time was ticking—the first of 34 proton therapy radiation treatments that would target her lymph nodes and any breast tissue remaining in her chest wall was scheduled for May 10, just three weeks away. When her insurer wouldn’t budge, the proton therapy center, ProCure, agreed to front the cost. The same day, says Gray, the Indian Health Service, which also provided her with health benefits, called to say they would cover the cost of the treatment. “I was surprised, shocked and happy,” says Gray. “I had been praying and asking God if this is what needed to be done.”

For about a century, radiation therapy has been a mainstay of cancer treatment. Standard radiation systems use photons, or X-rays, to kill cancer cells. Proton therapy uses particles that can be targeted at the tumor more precisely. Studies have shown that proton therapy is safe and effective. Less clear is which patients with which types of cancer should receive it instead of standard radiation. Clinical trials that compare proton and photon therapies are now underway, but enrolling patients hasn’t been easy. And in the years that it takes for the answers to come in, thousands more cancer patients will find themselves in a position similar to Gray’s.

Photons and Protons

Radiation kills a cell by damaging its DNA. The photon beam used in standard radiation therapy travels through normal cells in the body, gets into the cancer cells, and then travels again through normal cells as it comes out the other side of the body. Protons are particles with a different set of physical characteristics. They accelerate and penetrate the skin quickly, explains Steven Lin, a radiation oncologist at the University of Texas MD Anderson Cancer Center in Houston. Then the particles stop at the tumor, where they deposit all their energy at once.

The U.S. Food and Drug Administration (FDA) approved proton radiation as a cancer treatment in 1988. Before the FDA can approve a new cancer drug, clinical trials must show that the treatment is safe and effective for a specific type of cancer. New devices and technologies like proton therapy are held to a different benchmark. They only have to be proved safe and effective overall, not for a specific use. This means “there is no clear indication where proton [therapy] should be the standard treatment,” says Lin. Instead, “every cancer patient who needs radiation is potentially eligible for proton treatment, but not all patients will benefit.”

When there are no specific indications for a treatment’s use, insurance coverage can vary widely. Medicare typically covers the cost of proton therapy, regardless of the type of cancer. But many private insurers do not want to pay for proton therapy when it has not been shown to be more effective than standard radiation therapy and can cost four to 10 times more. A recent study found that two-thirds of patients with private health insurance initially had their requests for proton therapy denied. (On appeal, about 68% of patients initially denied coverage had their treatment approved.)

Determining the BenefitFor children with cancer, proton therapy is now a routine treatment. “For many pediatric patients, proton therapy offers clear benefits,” says Shannon MacDonald, a radiation oncologist at Massachusetts General Hospital in Boston. When treating children, she explains, “you are treating brain tumors and tumors close to areas that are responsible for future growth.” Before proton therapy was available, some of these children would not have been able to have radiation at all. With proton therapy, she says, they can be treated, and the tissue spared from radiation will continue to grow and develop normally. Proton therapy has also made radiation a possibility for some adults with rare or difficult-to-treat cancers, such as tumors in the central nervous system, brain, head and neck, eye, skull and spine.

In other instances, proton therapy has allowed many patients to avoid some or all of the potential side effects associated with standard radiation therapy, which can include skin problems, pain and swelling, and heart and lung problems. That was the case for Arianne Missimer of Coatesville, Pennsylvania, who was diagnosed in 2015 with a stage III liposarcoma—a rare cancer that can start in muscle tissue—in her right thigh. The 34-year-old physical therapist, registered dietitian and athlete needed radiation therapy to treat her cancer and was concerned about her potential risk for pain, swelling, weakness and long-term bone damage. Her radiation oncologist explained the difference between photon and proton therapies and then suggested proton therapy at Penn Medicine’s Roberts Proton Therapy Center in Philadelphia. Her insurer was willing to cover it.

A Growing Business

Proton therapy centers are now located across the U.S.

Waiting for Answers

It’s unclear whether proton therapy improves outcomes and reduces side effects in other cancer types, including breast and prostate cancer. The National Cancer Institute (NCI) and the Patient-Centered Outcomes Research Institute (PCORI) have funded seven phase III randomized trials comparing proton therapy and photon therapy in patients with breast, esophageal, liver, lung and prostate cancer and two types of brain tumors, glioblastoma and low-grade glioma. Some of the trials are comparing overall survival; others are looking at reductions in symptoms and side effects.

New Research Sheds Light on Side Effects

When combined with chemotherapy, proton therapy is associated with fewer severe side effects than standard radiation therapy, according to a study.

The results of these trials have the potential to inform future treatment guidelines, but finding patients for the studies has been laborious. In 2018, almost two years after it opened, the breast cancer trial had enrolled only 317 of 1,716 patients needed; after five years, the prostate cancer trial, which needs 400 patients, had enrolled only 254. Radiation oncologists point to multiple factors contributing to the slow patient accrual. In some cases, says Lin, doctors may believe proton therapy is better, and they don’t want their patients to participate in a clinical trial where there is a chance they won’t receive the newer approach. In other instances, patients don’t want to take the chance they will be assigned to the treatment arm that doesn’t receive proton therapy.

There is also an insurance barrier. In the major proton therapy trials, insurers are asked to pay for patients’ radiation treatment, whether it’s proton or photon therapy. Justin Bekelman, a radiation oncologist at the Penn Medicine Abramson Cancer Center, says it’s all too common for insurers to say they won’t pay for an unproven treatment when a patient is selected for the proton therapy arm. Bekelman was the lead investigator for the breast cancer trial and a co-lead investigator for the prostate cancer trial.

“Naturally, insurance companies are going to question the value,” says Bekelman. “That’s precisely why we need to run these trials. We want to determine if there are benefits and if there are harms to proton therapy, and in which cancer patients which treatment will be most successful for cancer control and reducing side effects.” But researchers can’t do that if insurers won’t cover that care.

In 2012, the University of Texas MD Anderson Cancer Center launched the NCI-funded clinical trial comparing protons and photons in esophageal cancer, which aimed to enroll 180 patients. Enrollment closed this year with 104. (Another 21 patients enrolled but couldn’t be evaluated because their insurer wouldn’t pay for the proton therapy.) Lin, who is overseeing the study, says some patients declined to enroll when they learned their health insurance covered proton therapy. “We explain to [patients] that the proton therapy is experimental, which is why we are trying to do the study,” he says. “But they say they’ve heard good things about it. Others say, ‘I have money and I don’t want standard treatment. I want the best.’”

It’s easy to understand why a patient who has pored over a proton therapy center’s website might feel that way. In a study published online March 15, 2018, in Radiation Oncology, researchers analyzed 46 websites of proton therapy centers—half of which were in the U.S. The analysis found that many centers used language that could lead patients to think that choosing proton therapy would give them a better outcome, says the study’s senior author Alexander Louie, a radiation oncologist and epidemiologist at Sunnybrook Health Sciences Centre in Toronto. “Many of the websites made blanket or generic statements that may not be completely supported by evidence but have some credence potentially or theoretically, blurring the line between evidence and advertising,” he says.

“It’s not as easy as saying if proton therapy is good or bad,” adds radiation oncologist Jeffrey Buchsbaum of the NCI’s Radiation Research Program. “Proton therapy is like a vehicle for getting the patient to a better place. And it has to be used properly.” There are certain situations, he notes, in which patients wouldn’t be alive without proton therapy. “But that doesn’t mean it’s necessary for all cancers.”

Proton Therapy Tips

Follow these suggestions as you consider radiation therapy options.

Moving Forward

The American Society for Radiation Oncology has developed model policies for insurers that delineate where there is sufficient evidence to support coverage of proton therapy. Insurers also use National Comprehensive Cancer Network treatment guidelines to support or deny a patient’s treatment with proton therapy. To move research forward, investigators are trying to work with hospitals to find ways to make insurers more amenable to covering the cost of treating patients in randomized clinical trials comparing photon therapy and proton therapy. In some cases, this may include reducing the cost of proton therapy to make it more comparable to that of standard radiation therapy. “The issues happening here are partially the result of the complexity of the health care delivery system,” says Buchsbaum.

But for patients, treatment choices must be made now. Missimer believes that proton therapy helped treat her cancer without sacrificing her athleticism. She is an active member of Penn Medicine’s proton center alumni group, which provides support to patients who are currently receiving or are considering proton therapy. She also appears in an advertisement for Penn Medicine’s proton therapy center, and an article about her experience is included on the cancer center’s website.

Missimer’s treatment began with chemotherapy, which she admits slowed her down. But during her proton therapy, which started in July 2015, she joined a ninja gym. And as she recovered from the surgery and additional chemotherapy that followed the radiation, she kept going. In May 2016, Missimer competed in the Philadelphia regional American Ninja Warrior competition. “I lost my brother to cancer,” she says. “He had radiation and had significant complications. The only thing I get is a little stiffness. But as long as I keep moving, my leg is good.”

Gray completed her proton beam treatment in June 2018, about a year after she’d first felt the lump in her breast. Skin damage is a common side effect of both types of radiation therapy. Gray says her doctor told her that her skin did well during the proton therapy. “But if that was well,” she says, “I can’t imagine what worse would be like. My chest looked like burnt hot-dog skin. And I still have a dark scar from the burn that might not ever go away.” After being out of work for a full year, Gray returned to her job as an educational specialist for Native American youth in October 2018, and she slowly started back at the gym. She wears a compression sleeve and a glove to manage lymphedema that developed in her arm—caused by either the surgery or radiation—and deals with nerve pain in her arm and chest. None of it has been easy, but, she says, “my faith has gotten me through.”

Sue Rochman is a contributing editor for Cancer Today.

The Right Dose

This blog was originally published by Cancer Today by Kate Yandell here.

Researchers want to find out when cancer patients can benefit from receiving lower doses of drugs or radiation, shortening treatment or skipping certain treatments altogether.

by Kate Yandell

OVER A SPAN OF 15 YEARS, Liza Bernstein was diagnosed with three separate primary, early-stage breast cancers. Even though she was treated by the same oncologist throughout, the treatments she received varied with each diagnosis.

Bernstein, who lives in the Los Angeles area, was first diagnosed with hormone receptor-positive breast cancer in 1994, when she was 29 years old. She recalls that her doctors were pleased to be able to do a lumpectomy, only removing part of the breast, instead of a mastectomy as would once have been standard. However, her surgeon removed about 20 lymph nodes from her armpit, and she received both radiation and chemotherapy.

In the course of receiving her second diagnosis, a hormone receptor-positive cancer in her opposite breast, in 2005, Bernstein underwent a sentinel lymph node biopsy, a less invasive procedure that requires surgeons to remove only a few lymph nodes in areas where the cancer is most likely to have spread.

Bernstein was also able to get testing with a product called Oncotype DX, which measures gene expression in breast tumors and helps estimate the likelihood that chemotherapy will prevent an early-stage, hormone receptor-positive cancer from recurring. The test, released in 2004, helped Bernstein and her oncologist make the difficult decision to skip chemotherapy in 2005, due to little predicted benefit. Bernstein received a lumpectomy, radiation and the hormone therapy tamoxifen. Conversely, when she was diagnosed with another hormone receptor-positive cancer in 2009, genomic tumor testing helped them decide to include chemotherapy, along with a double mastectomy and tamoxifen, in her treatment.

Advances in cancer research can mean making patients’ treatment more onerous and complex. But some of the changes in Bernstein’s breast cancer treatment over the years reflect de-escalation—the process of decreasing the intensity or duration of a treatment, thus reducing side effects and cost, while maintaining the treatment’s effectiveness.

Today, researchers are investigating whether they can identify patients—using genomic tumor testing, imaging of the cancer or other methods—who can receive less intense treatment. Treatment de-escalation aims to spare patients the burden of unnecessary treatments and side effects.

“The key is we want to give people the right treatment that they need without treating them excessively, which just produces too much toxicity,” says Eric Winer, a medical oncologist and chief of the Division of Breast Oncology at the Dana-Farber Cancer Institute in Boston.

Treating the Right Patients

Treatment de-escalation has been successful primarily in cancers where the survival rate is high. “When you have a situation where mortality from a given malignancy is high, then it’s pretty hard to think about backing off [from treatment],” Winer explains.

The effects of treatment can last long after chemotherapy or radiation is completed. For example, 87% of people in the U.S. diagnosed with Hodgkin lymphoma, which until the 1960s was usually fatal, live five years or more. “The issue for this group of people, who are often diagnosed in their 20s and 30s, is that they have a long life ahead of them,” says Peter Johnson, a medical oncologist who specializes in lymphoma at University Hospital Southampton in England. The radiation and chemotherapy typically given for Hodgkin lymphoma can result in serious side effects, including heart disease, second cancers and infertility.

Over time, doctors have adopted techniques for delivering radiotherapy to Hodgkin lymphoma patients that increasingly spare normal tissues from damage, Johnson says. Most recently, researchers have learned that they can perform a form of imaging, called 18F-fluorodeoxyglucose PET, to determine early on whether a patient’s Hodgkin lymphoma is responding to chemotherapy. If the scan indicates a good response, the patient may be able to skip later radiation therapy or receive a less intensive chemotherapy regimen.

“In some ways, it’s a reflection of how successful modern oncology has been that we’re thinking about these things,” Johnson says of the topic of de-escalation.

The rise of genomic testing, among other factors, has contributed to a decline in chemotherapy use for patients with early-stage breast cancer whose disease is driven by hormones. With Oncotype DX and similar tests, patients with hormone receptor-positive, HER2-negative breast cancer can learn how likely they are to benefit from chemotherapy. Their score can help determine whether their drug treatment after surgery should include both chemotherapy and hormone therapy or whether just hormone therapy is enough.

Researchers are investigating de-escalation strategies for patients with early-stage HER2-positive breast cancers as well. These patients are often treated with HER2-targeted therapy and a multidrug chemotherapy regimen. Winer’s research shows that patients with small HER2-positive cancers that have not spread to the lymph nodes can safely use a de-escalated chemotherapy regimen that includes just one drug, paclitaxel, alongside targeted therapy.

Challenges of Stepping Back

Despite some successes in de-escalation, it can be easier to intensify treatment than to take treatment away. This is partly because it is difficult to prove that taking away treatment is not going to harm patients—a different statistical challenge than showing that a therapy is significantly better than standard care.

For example, in 2004, researchers discovered that patients with stage III colon cancer lived longer if oxaliplatin was added to their chemotherapy regimen. The additional chemotherapy drug can lead to peripheral neuropathy, and the effects are cumulative as therapy continues. An international consortium of researchers published a study in the New England Journal of Medicine on March 29, 2018, pooling the results of six randomized clinical trials that included 12,834 participants. The trials investigated the practice of shortening chemotherapy after surgery from six to three months for these patients.

“We thought with such a large number it would be very easy and we’d get a clear answer, [but] we haven’t got as clear an answer as we thought we would,” says Timothy Iveson, a medical oncologist at University Hospital Southampton who co-authored the study.

The study did not meet pre-specified statistical benchmarks to determine that a shorter period  of chemotherapy was not worse than standard chemotherapy for the patients in the trial in general. However, the survival difference between patients using shorter versus longer chemotherapy (six months versus three months) was small, Iveson says, and the decrease in side effects with shorter chemotherapy was large. And for some patients, treatment for three months was sufficient. Cancer treatment guidelines now recommend the shorter chemotherapy regimen as an option for certain patients with low-risk stage III colon cancer.

New information about cancer subtypes can also spur de-escalation. But even when it’s clear that de-escalation is necessary, it can take time to settle on the right strategy, as shown by the experience of researchers trying to back off treatment for head and neck cancer caused by the human papillomavirus (HPV). “There’s been an epidemic of oropharyngeal cancers that are related to HPV,” explains Joshua Bauml, a medical oncologist at the Hospital of the University of Pennsylvania in Philadelphia. “These cancers have a much higher cure rate, and that’s wonderful, but the issue is that our treatment paradigm is still based upon older cancers with a different biology.”

Standard treatment for patients with advanced head and neck cancer—originally developed for patients with smoking- and alcohol-associated cancers—involves some combination of surgery, radiation and chemotherapy. But these treatments can cause troubling side effects, including difficulty swallowing, dry mouth, problems with speech and changes in taste.

One approach for reducing toxicity of chemotherapy for these patients was to replace the chemotherapy drug cisplatin with the targeted therapy Erbitux (cetuximab), in an attempt to spare patients the side effects that cisplatin can cause when combined with radiotherapy. However, recent clinical trial results have shown that patients with HPV-positive oropharyngeal cancer treated with Erbitux have shorter survival than those treated with cisplatin and have similar rates of side effects, indicating that this is not a good de-escalation strategy.

Early trials of approaches to reduce doses of radiation or chemotherapy for patients with HPV-related oropharyngeal cancer have shown promise, Bauml says. However, he urges clinicians to wait for further data before adopting new protocols for HPV-related oropharyngeal cancer. “If a head and neck cancer metastasizes, it is incurable,” he says. “It’s really essential that when we move towards treatment de-escalation, this is done through robust clinical trials.”

Getting Targeted

The term de-escalation is used most often to describe efforts to reduce harms from old modes of therapy, including surgery, radiation and chemotherapy. But researchers are also working to understand the right doses of medication for patients being treated with newer targeted therapies and immunotherapies.

A study in the July 2018 issue of Cancer, for instance, showed that Sprycel (dasatinib), a type of targeted therapy called a tyrosine kinase inhibitor, is effective at a reduced dose in treating chronic myeloid leukemia (CML). The lower dose appears to cause fewer dangerous side effects, such as buildup of fluid near the lungs, and costs around half as much. Other tyrosine kinase inhibitors have also been shown to be effective in treating CML at reduced doses, says study co-author Hagop Kantarjian, an oncologist who specializes in leukemia at the University of Texas MD Anderson Cancer Center in Houston.

Traditional methods of determining doses for cancer drugs aren’t always ideal for dosing targeted therapies, Kantarjian explains. Clinical trials for chemotherapy ramp up doses in people until the highest dose with acceptable side effects is found, a measure known as maximum tolerated dose. Targeted therapies, in contrast, can be effective at doses much lower than the maximum tolerated dose. Researchers are still trying to find the best strategies for determining dosing of targeted therapies.

Researchers are also investigating whether they can reduce the time that patients are on targeted therapies and immunotherapies. For instance, “there are no clear, specific guidelines on exactly how long to treat patients with immune therapy in cancer,” says Michael Postow, a medical oncologist at Memorial Sloan Kettering Cancer Center in New York City who treats patients with melanoma.

Scientifically, it makes sense that patients who respond to immunotherapy drugs might be able to stop taking them at some point, says Janet Dancey, scientific director of the Canadian Cancer Trials Group and a medical oncologist at Queen’s University in Kingston, Ontario.

Most cancer drugs work by directly killing or inhibiting the growth of cancer cells. In contrast, immunotherapies work by stimulating the immune system to attack cancer. It’s possible that once the immune system has been activated, continued administration of the drugs isn’t necessary.

Dancey’s organization is currently enrolling patients for the STOP-GAP study, a randomized trial looking into whether melanoma patients who have responded to a class of immunotherapy drugs called PD-1 inhibitors can stop treatment or whether they would benefit from staying on treatment indefinitely.

There are multiple reasons to stop treatments, says Postow. “People would want to stop mostly to get their lives back to themselves, for flexibility in travel and work. … And I think the idea of being under treatment is still a reminder that there is something wrong with the patient.”

There are also financial implications: Checkpoint inhibitors have generally debuted with list prices of $150,000 per year or more. And treatment comes with other costs like time taken off from work, Postow says.

Currently, Postow works with his patients to make individual decisions on whether to stay on immunotherapy after all evidence of active cancer disappears or after two years of improvement on the treatment. He hopes further research will make choices easier for patients. “As you can imagine, there is a lot of emotional decision-making around this issue, too, which is reasonable in a setting where we don’t have strong science to specifically guide us,” he says.

A Lower Dose of  Financial Toxicity

Researchers are looking into whether some drugs are just as effective when taken at a reduced dose.

A Shared Decision

Whether patients are considering skipping chemotherapy or stopping immunotherapy, having thoughtful discussions about benefits and risks of treatments is key. That includes helping patients understand side effects, says Iveson, who studied shortening chemotherapy for colon cancer patients. For instance, rather than telling patients they might experience peripheral neuropathy, doctors should explain this can mean not being able to button a shirt or feel one’s feet.

“The challenging part is that, for both doctors and patients, there’s a tendency to be risk averse,” Winer notes. People don’t like to feel they are leaving potential benefits of treatment on the table. Doctors sometimes underestimate side effects and overestimate treatment benefits, he says, and “nobody wants to be judged as having done something wrong by backing off if there’s a bad outcome.”

For Bernstein, the lengthy decision-making process that came with skipping chemotherapy after her second cancer diagnosis was difficult because there wasn’t a clear-cut answer of what to do, at least until she got the Oncotype DX test results. But she says she ultimately was glad to have had in-depth discussions with her doctor. Despite progress in treatment de-escalation, Bernstein hopes more can be done both to eliminate unnecessary treatment and to treat cancer more effectively.

“Over time there have been strategies that have come into play and have helped, in a sense, to do less harm, but by no means do they do no harm,” Bernstein says. “I want to make that clear.”

Kate Yandell is the digital editor of Cancer Today.

Immunotherapy

October 1, 2019/in Adrenal Cancer, Adrenal Cancer Treatment & Clinical Trials, Adrenal Cancer Video TC, Appendix Cancer, Appendix Cancer Treatment & Clinical Trials, Appendix Cancer Video TC, Bone Cancer, Bone Cancer Treatment & Clinical Trials, Bone Cancer Video TC, Brain Cancer, Brain Cancer Treatment & Clinical Trials, Brain Cancer Video TC, Eye Cancer, Eye Cancer Video TC, General Health Info, General Treatment and Clinical Trials, General Video TC /by PEN Editorial Staff

This video was originally published by the National Cancer Institute on June 13, 2018, here.

Immunotherapy uses the body’s immune system to fight cancer. This animation explains three types of immunotherapy used to treat cancer: nonspecific immune stimulation, T-cell transfer therapy, and immune checkpoint inhibitors. http://www.cancer.gov/immunotherapy

About Clinical Trials Videos

These videos were originally published by the American Cancer Society here.

Clinical Trials Videos

Knowing all you can about clinical trials can help you feel better when deciding whether or not to take part in one as part of your cancer treatment. Explore these videos to get answers to basic questions and concerns about clinical trials so that you will be better prepared to discuss this option with your doctor and your family. (ACT videos were produced by Genentech in collaboration with the American Cancer Society.)

ACT: Why Should I Consider a Cancer Clinical Trial?

ACT: How Will I Know If a Trial Is Right for Me?

ACT: How Should I Prepare for Discussions With My Doctor?

ACT: What Is Informed Consent?

ACT: What Standards Are in Place to Safeguard Trial Participants?

ACT: Where Can I Turn for Information and Support?

Understanding Diagnostic Technologies and Biomarkers

October 1, 2019/in Adrenal Cancer, Adrenal Cancer Podcast T, Adrenal Cancer Testing, Appendix Cancer, Appendix Cancer Podcasts T, Appendix Cancer Testing, Bone Cancer, Bone Cancer Podcast T, Bone Cancer Testing, Brain Cancer, Brain Cancer Podcast T, Brain Cancer Testing, CancerCare, Eye Cancer, Eye Cancer Podcast T, Eye Cancer Testing, General Health Info, General Podcast T, General Testing, Partner Resources /by PEN Editorial Staff

This podcast was originally published by Cancer Care on May 6, 2015, here.

Topics Covered

Why the Molecular Portrait of Cancer is Important
The Role of the Pathologist
Benefits of Diagnostic Technologies, Biomarkers, Precision Medicine and Predicting Response to Treatment
Clinical Trials: How Research Contributes to Your Treatment Options
Questions to Ask Your Health Care Team
Quality-of-Life Concerns
Questions for Our Panel of Experts

Our Panel of Experts

Richard J. Gralla, MD, FACP

Professor of Medicine, Albert Einstein College of Medicine

Al B. Benson, III, MD, FACP, FASCO

Professor of Medicine, Associate Director for Clinical Investigations, Robert H. Lurie Comprehensive Cancer Center of Northwestern University

Sarah E. Kerr, MD

Senior Associate Consultant, Division of Anatomic Pathology, Department of Laboratory Medicine and Pathology, Assistant Professor, Laboratory Medicine and Pathology, Mayo Clinic

Charles L. Loprinzi, MD

Regis Professor of Breast Cancer Research, Mayo Clinic College of Medicine

Caroline Edlund, MSW

Online Support Group Program Director, CancerCare

Does Pre-Cancer Mean I’m Going to Get Cancer?

October 1, 2019/in Adrenal Cancer, Adrenal Cancer Blog T, Adrenal Cancer Testing, Appendix Cancer, Appendix Cancer Blog T, Appendix Cancer Testing, Bone Cancer, Bone Cancer Blog T, Bone Cancer Testing, Brain Cancer, Brain Cancer Blog T, Brain Cancer Testing, Eye Cancer, Eye Cancer Blog T, Eye Cancer Testing, General Blog T, General Health Info, General Testing /by PEN Editorial Staff

This blog was originally published by Cancer Treatments Centers of America on December 20, 2018, here.

A male and female physician evaluating x-rays for a cancer diagnosis — Every year, thousands of people are diagnosed with pre-cancerous conditions, news that may induce fear and panic in those receiving it. While pre-cancer that goes unchecked may ultimately become cancerous, it’s not a guarantee and, in many cases, not even likely.

Every year, thousands of people are diagnosed with pre-cancerous conditions, news that may induce fear and panic in those receiving it. While pre-cancer that goes unchecked may ultimately become cancerous, it’s not a guarantee and, in many cases, not even likely. “ No one dies of pre-cancer,” says Justin Chura, MD, Chief of Surgery & Director of Gynecologic Oncology and Robotic Surgery at our Philadelphia hospital. “It’s a very treatable condition, if it even needs treatment at all. A pathology report may indicate carcinoma in situ. When patients, and even some clinicians, see the word carcinoma, they get misled into thinking they have cancer. Pre-cancer means there are cells that have grown abnormally, causing their size, shape or appearance to look different than normal cells.”

Whether abnormal cells become cancerous is, in many cases, an uncertainty. Some of the variables are known, others are not. So what exactly does it mean to be told you have a pre-cancerous condition? Does it increase the risk of getting cancer? Are there any prophylactic (preventative) measures that can be taken to reduce the likelihood that a cancer diagnosis is in your future?

According to Elizabeth Min Hui Kim, MD, MPH, FACS, Director of the CTCA Breast Cancer Institute, pre-cancer is becoming an outdated term in breast oncology as well as other specialties, because the condition is more complex than a blanket term can describe. “We’re understanding these precancerous cells have certain genes, and we’re using technology to understand how they differ from each other and have different risks based on their biology,” she explains.

Citing the current body of literature—which includes a 1985 study that re-evaluated 10,366 breast biopsies performed on women at three Nashville hospitals, a 2007 Mayo cohort study, a 2012 study that evaluated breast cancer risk based on atypia type, a 2012 study on the management of high-risk breast lesions and a 2016 study on which Dr. Kim was a lead author—Dr. Kim says doctors can classify the condition into one of three categories: non-proliferative disease, proliferative disease without atypia and proliferative disease with atypia.

How to approach a non-proliferative disease diagnosis depends on the person’s cumulative risk of developing breast cancer, which is a risk assessment based on a variety of factors, including but not limited to family history and breast density as well as personal risk factors that may be modifiable, like body mass index (BMI) and tobacco or estrogen use. If the cumulative risk is determined to be greater than 20 percent, Dr. Kim says enhanced screenings (having both a mammogram and a breast MRI, for example) may be recommended.

Diagnoses in the proliferative disease category indicate that abnormal cells are growing faster than normal cells, but not as fast as cancer cells. “There is abnormal growth or size of the cells, or the cells might be larger than normal,” Dr. Kim explains. An example of a proliferative disease would be an intraductal papilloma of the breast, which is like a polyp. Surgical removal of the area may be advised, depending on the patient.

Proliferative disease with atypia indicates high-risk lesions (abnormal cells) that are growing faster than normal. Depending on the cumulative risk, a form of medical treatment called anti-estrogen therapy or surgery may be recommended.

Modern medicine allows many “pre-cancerous conditions” to be found early. A pap test detects cervical dysplasia (abnormal cells in the cervix), sometimes referred to as pre-cancer. Low-grade dysplasia is typically not treated unless it persists for a couple of years, Dr. Chura says, while high-grade dysplasia would require a biopsy. The biopsy results would dictate the next steps. A colonoscopy detects colon polyps, and skin cancer screenings by a dermatologist are credited with identifying for removal many skin cancers that would have metastasized (spread).

“If you’re diagnosed with some type of dysplasia, whether in the esophagus, colon, cervix, etc., it doesn’t mean you will develop cancer. It means you will need some type of surveillance and treatment plan to manage it,” Dr. Chura says.

The takeaway is that a pre-cancerous condition does not mean you have cancer. It simply means you have an increased risk of cancer, which should serve as a reminder to stay current with medical visits and screening tests and communicate concerns or changes to your doctor.

“These are easily solvable problems and can be addressed with treatments that are a lot less invasive and have a lot more options than if the patient had a malignant disease,” says Dr. Kim. “It’s not cancer until proven otherwise. And if it is, they’ve caught it really, really early.”

Identifying Biomarkers Gives Doctors Known Targets to Treat Many Cancers

October 1, 2019/in Adrenal Cancer, Adrenal Cancer Blog T, Adrenal Cancer Testing, Appendix Cancer, Appendix Cancer Blog T, Appendix Cancer Testing, BC Blog T, BC Testing, Bone Cancer, Bone Cancer Blog T, Bone Cancer Testing, Brain Cancer, Brain Cancer Blog T, Brain Cancer Testing, Breast Cancer, Eye Cancer, Eye Cancer Blog T, Eye Cancer Testing, General Blog T, General Health Info, General Testing, GYNC Blog T, GYNC Testing, Gynecological Cancers, Head & Neck Blog T, Head & Neck Cancer, Head & Neck Cancer Testing /by PEN Editorial Staff

This blog was originally published by Cancer Treatments Centers of America on August 21, 2019, here.

Biomarkers — Doctors are increasingly relying on biomarkers, which help determine a patient’s overall health and/or the presence of disease. Learn what biomarkers are and why they are increasingly important in cancer care.

When faced with opposition, it’s beneficial to learn as much as possible about the opponent. A pitcher reads a scouting report before facing a lineup. An army consults intelligence before engaging the enemy in battle.

The same principles apply to the treatment of some cancers. When treating a tumor, it’s important for a doctor to know as much as possible about that cancer—specifically, what is driving the tumor’s growth.

To get the inside information on a tumor, doctors are increasingly relying on biomarkers, short for biological markers, measurable signs or substances in the body that may indicate a patient’s overall health and/or the presence or progression of disease.

The discovery of biomarkers in cancer drastically changed the course of cancer treatment. For decades, many cancers were treated similarly, with surgery, radiation therapy or chemotherapy. Identifying biomarkers in cancer cells has led to the development of new precision medicine drugs, such as targeted therapy and immunotherapy, designed to target specific features in cancer cells, potentially reducing the damage to healthy cells. “The routine use of a variety of biomarkers has substantially changed the way in which cancer medicine is practiced,” says Maurie Markman, MD, President of Medicine & Science at Cancer Treatment Centers of America^® (CTCA), “from providing more accurate prognostic information to assisting in the prediction of specific therapeutic strategies that are more likely to result in a favorable outcome for an individual patient.”

What are biomarkers?

A biomarker is any measurable indicator of a person’s health. Blood pressure is a biomarker, as are body temperature, blood sugar and cholesterol measurements. In cancer, biomarkers also include proteins, hormones, gene aberrations, such as mutations or rearranged genes, and other molecules found in or on cancer cells. Cancer biomarkers may be found in routine blood, urine or stool tests. Others may require a biopsy and/or advanced genomic testing to uncover. “Genomics has made it so much easier to find gene mutations,” says Arturo Loaiza-Bonilla, MD, MSEd, FACP, Vice Chair for the CTCA^® Department of Medical Oncology. “Now we may be able to target a mutation and potentially get the cancer to stop growing.”

Biomarkers play multiple roles in the treatment of diseases, such as cancer, including:

Diagnostic: Helping confirm the presence of disease, sometimes before symptoms develop

Prognostic: Helping forecast the progression and aggressiveness of the disease and the risk of recurrence

Predictive: Helping doctors identify how patients may respond to certain drugs

Biomarkers may play any or all these roles and more. Some biomarkers may be used to assess a patient’s risk of developing disease, the effectiveness of a treatment or whether a treatment is safe or toxic.

Common cancer biomarkers include:

BRCA1 and BRCA2 genes: Mutations in these genes may increase a woman’s risk of breast and ovarian cancer. In men, it may increase the risk of prostate cancer.
PSA: Prostate specific antigen may indicate prostate cancer. This biomarker may be used not just to diagnose the disease, but to measure its progression and how the treatment is performing.
HER2: Human epidermal growth factor receptor 2 is found in many cancers, especially breast cancer. The targeted therapy drug trastuzumab and other similar monoclonal antibodies may be a treatment option for patients with HER2-positive cancers.
BCR-ABL: This gene, known as the Philadelphia chromosome, is found in patients with chronic myelogenous leukemia. Presence of the gene may indicate the patient may respond well to treatment with a tyrosine kinase inhibitor drug such as imatinib.
PD-L1: Programmed death ligand 1 is the companion receptor to PD-1. It may indicate a cancer’s ability to evade the immune system. Immunotherapy drugs called checkpoint inhibitors may be an option to treat cancers high in PD-L1.
CA-125: High levels of cancer antigen-125 are found in many cancers as well as other diseases. Treatment options for cancers with CA-125 vary depending on where the cancer originated.
MSI-H: Microsatellite instability-high is a mutation in the DNA of cells found in many cancers, especially colorectal cancer. Checkpoint inhibitor drugs have been approved for cancers with MSI-H.

Difficult targets

Biomarkers don’t always tell the full story. Discovery of a biomarker that might indicate an increased cancer risk doesn’t mean a patient will get cancer. Not all cancers have identifiable biomarkers. And identifying a driving biomarker in a cancer does not necessarily lead to a treatment option. Some biomarkers for cancer have no corresponding targeted therapy or immunotherapy drug. For example:

TP53: Tumor protein 53 is a tumor suppressor gene designed to help stop cancer cells from growing. TP53 mutations are the most common found in cancer cells and may be found in most types of cancer.
RAS: About 30 percent of all cancers, including 95 percent of all pancreatic cancers, have known mutations in the RAS family of genes that control cell death and growth.

No targeted therapy drugs have been approved specifically to treat cancers with these mutations. “A number of recognized critical signaling pathways in cancer development, progression and resistance remain very difficult to ‘target’ to influence clinical outcomes,” Dr. Markman says. “The ability to successfully and safely target either or both of these pathways has the potential to be an important advancement in cancer management.”

Many cancers, especially solid tumors, have multiple biomarkers, any one of which may be able to drive a cancer’s growth. Target one biomarker, and another may take over as the driving mutation. And not all the same biomarkers are found in every cancer cell. “As cancer cells grow, they start to develop new abnormalities, mistakes made while the cells are multiplying,” Dr. Bonilla says. These new mutations may make the cancer more resistant to treatment.

Also, doctors need to take steps to prevent the patient from being harmed by the process of targeting a specific biomarker. For instance, patients on a checkpoint inhibitor that targets cancers high in PD-L1 may develop symptoms of autoimmune diseases, such as colitis. “The goal is to find the specific biomarker that every single cell expresses without compromising the normal cells,” Dr. Bonilla says, “because once you tell the immune system to kill a population of cells, it is going to kill all those cells, whether they are good or bad. But if you are able to find the specific biomarker that is the hallmark of this disease and needs to be eliminated, then it’s much easier to find a therapy.”

The discovery of biomarkers has led to game-changing developments in the cancer treatment. Women who learn they have BRCA mutations are now empowered to make potentially life-saving decisions to prevent breast and ovarian cancer. Men with slow-developing prostate cancer can now actively monitor their disease, in part, because their PSA levels can be measured. And research is ongoing to find new biomarkers to help in the treatment of other cancers and diseases, such as diabetes, Parkinson’s disease and heart disease.

“Biomarkers offer an opportunity to apply genomics to population health and see what diseases or conditions people may be predisposed to,” says Pamela Crilley, DO, Chair of the CTCA Department of Medical Oncology. “Am I going to get diabetes? Am I going to get elevated cholesterol? Is there anything I can do about it? Look at hereditary breast and ovarian cancers. The science has led to being able to prevent disease in patients with BRCA1 and BRCA2 mutations. Now we may be able to significantly reduce your risk of disease.”

At Home Genetic Tests Offer Limited Information

This blog was originally published by Caner Today by Sharon Tregaskis here.

At-home genetic tests offer limited information on cancer risk.

KNOWLEDGE IS POWER with regards to lowering cancer risk. But if you’re looking for clues to the hereditary hazards lurking in your genome, testing at home can be risky business.

Dozens of companies sell online genetic tests to consumers interested in exploring their ancestry or health risks. No prescription is needed. The consumer simply needs to send in a saliva sample. The U.S. Food and Drug Administration (FDA) cautions consumers that the results they receive from these direct-to-consumer tests are not intended to be the primary basis for medical decision-making. But consumers may not be aware of how limited the test findings actually are.

Questions to Ask

Find out the following before you purchase an at-home genetic test.

Who will have access to your data?
Learn whether the testing company can sell your data to a third party. Can you opt out of having your data sold?

Will your privacy be protected?
Know the company’s policies. Consider using a pseudonym and a false date of birth to reduce your risk of exposure.

What if there’s bad news?
Identify a genetic counselor you can talk to about cancer risk test results. The National Society of Genetic Counselors can help you find one in your area.

In January 2019, the company 23andMe became the first permitted by the FDA to offer an at-home genetic test to assess colorectal cancer risk. The test looks for two genetic mutations associated with a rare, hereditary colorectal cancer syndrome. It does not screen for Lynch syndrome, the inherited condition responsible for most cases of hereditary colorectal cancer. (Lynch syndrome also increases risk for other types of cancer.)

The FDA previously approved the company’s test for three inherited BRCA mutations associated with an increased risk for breast and ovarian cancer. However, the BRCA genetic variations the test looks for are not the most common. A study presented in April 2019 at the American College of Medical Genetics and Genomics annual meeting found the 23andMe test missed close to 90% of BRCA mutation carriers. The study was conducted by Invitae, a company that sells genetic tests for inherited cancers that can only be ordered by a physician.

“Many of these [direct-to-consumer tests] are in no way equivalent to a medical-grade test,” says genetic counselor Lisa Madlensky, director of the Family Cancer Genetics Program at Moores Cancer Center at UC San Diego Health in California.

Kayla Sheets, a genetic counselor and chair of the Massachusetts Board of Registration of Genetic Counselors, took a direct-to-consumer genetic test so she could experience the process firsthand. She found that the online resources for interpreting her results fell far short of the tailored, comprehensive information genetic counselors like her offer. “There are different levels of DNA testing available,” says Sheets. “Anyone concerned about cancer risk needs something ordered by a genetic counselor who has a really good understanding of what that test can and can’t do for them.”

June 21, 2019

Finding Support in Unlikely Places

This blog was originally published by Cancer Today by Patricia Anne Ward here.

WHEN I WAS DIAGNOSED with stage IC ovarian cancer in February 2018, my life came to a screeching halt. It was the first life-threatening health issue I’d ever faced in my 70 years of life. Despite my fear and anxiety, I knew I didn’t want my cancer or treatment to define me as a person. Honoring that wish turned out to be much easier in theory than in practice, as even a well-intentioned question like “How are you?” served as a reminder of my circumstances.

There were also some insensitive remarks that cut far deeper. One person told me, for example, that God never gives us too much to handle. Another described how cancer was a blessing reserved only for the strong—if this is true, I’d much rather be weak. Someone else suggested my cancer was a result of some transgression to God and that I should make amends.

Despite being surprised by these comments, I understood. People say a lot of things when they are faced with the uncomfortable concept of mortality. And my cancer was a tangible reminder of what we all know and ignore: Each of us has a finite amount of time.

It was shortly after I started losing my hair, a side effect of chemotherapy, that I began noticing others were uncomfortable around me. Some family and friends even avoided making eye contact when talking to me. Others used text messaging to avoid uneasy verbal conversations. With no one in my immediate circle undergoing cancer treatment, I felt alone.

That’s when I started discovering support in the most unlikely places. In the pickle and condiment aisle at the grocery store, a woman turned to look at me, a wide smile across her face. Not too long before our meeting, that woman wore a cap just like mine. She shared that she was two years out of treatment and her scans were still “clean.” We spoke for a bit, tearing up. I held hands with this kind stranger for a few moments. We hugged, wished each other well and went our separate ways.

At a clothing store, another woman stopped me after spotting that cap, which gave my cancer away. Apologizing first for interrupting, she then told a familiar tale of symptoms, diagnosis, surgery, treatment and the worst part: waiting. Still, she was approaching the five-year mark since her diagnosis with no evidence of disease. Speaking from a place of vulnerability but also strength, she described how her cancer had helped her to become more compassionate, a virtue that she would surely lean on, since her husband had been newly diagnosed with cancer. As we said goodbye, I promised her I would stay positive and upbeat—and strangely I wasn’t lying.

All through my treatment, chance encounters with people who knew what I was experiencing came to me just when I needed them. I saw these people as angels, and still do. They came with no judgement or preaching. There was no awkward response or agenda. Our connections were palpable.

There were other signs: I found feathers everywhere, next to my car in a parking lot, the floor in the garage or at the park. One simply blew in on a breeze and landed right at my feet. And each time I made a discovery of these gifts from nature, a sense of warmth and protection would envelope me. I have always believed in angels; I found it encouraging to think that they believed in me too.

For now, my treatments are over. My hair and eyebrows are growing back. My recovery continues. There will be more doctor appointments, more exams, and more wondering about and praying for a future with no more cancer. I’ve changed too. I am smarter, kinder and less quick to judge. When I am out of the house, I scan my surroundings for people like me—signs of treatment, signs of fear—to offer an encouraging word. I don’t know what the future holds, but I can’t help but believe that I am going to be OK.

Patricia Anne Ward of Gaylord, Michigan, is a retired human resources administrator and supervisor who has been married to her husband, Richard, for 30 years. She is the former president of a local animal welfare group, Friends for Life of Otsego County. Her proudest title is survivor.

5 Things Never to Tell Someone Fighting Cancer

This blog was originally published by I Had Cancer on August 6, 2019, here.

Since being diagnosed with non-Hodgkin’s lymphoma in November, I’ve received so much love and support from those around me. Weirdly and surprisingly, I have become closer to some of my friends who were suddenly and unequivocally there for me. I have made new friends from acquaintances who decided it was their job to support me. I have learned to ask for help, which is huge given that I come from the Do Everything Yourself school of thought.

As a prominent member of this school, I also can’t handle unsolicited advice. While most people have been nothing but kind and understanding, some have made it their business to tell me how things “really are”. They think that the fog of cancer is sometimes so all-enveloping that you apparently become confused if not downright mistaken about the why, how and what of your condition. So thank you, casual acquaintances and random strangers in waiting rooms, for enlightening me with your non-specific one-size-fits-all statements. If you’re worried you might be doing this, here are the five hands-down worst things to say to a cancer fighter:

“Sugar feeds cancer”

You will be surprised how, when they find out about you having cancer, some people immediately think “Why”.” And then, almost by magic, their single-track train of thought alights upon “Oh, SUGAR”. The silent killer. The symbol of all that is wrong with society today. Some will whisper, “So, have you changed your diet in any way?” and you know exactly where this is going. Others will just say it outright, like a mantra, “Sugar feeds cancer, y’know.” Initially I was keen to be like, “Yes! Yes! I’m eating so healthily!” Then I thought to myself, I’m the one with cancer here. I’m the one walking around with a 4 inch lump in my chest, sitting in hospital beds while IV fluids pulse their way into me, taking all the pills, feeling all the nausea. I thought, wait a minute. You must be kidding me with your sugar theory.

Because, people, here is the bare-naked truth. It’s a myth. No medical doctor and no reputable cancer institute will tell you that sugar causes cancer, or “feeds” your cancer. One of my oncologists actually had candy on her desk, which frankly might be a little overkill because candy probably isn’t good for anyone. But maybe she was trying to prove a point: You have cancer, have a lollipop! You’ll be fine. FINE. It will not feed it. It will not make it worse, nor will it make it better. Your teeth may rot though.

In the interest of factual accuracy, there has been one recent study using yeast cells (which are similar to cancer cells, but obviously not actual cancer cells) that seemed to suggest that sugar contributed to more growth in these cells. This study has not been replicated so far. And yet, Dr Google will tell you that yes, sugar feeds cancer. The real link here though is that a high sugar diet could make you obese, which is a risk factor for some cancers. THAT’S IT. That is the only proven link at this time.

But let’s assume that it is true, that sugar may actually cause cancer, and then proceed to help it grow. Well, even if this were so, here is my theory: life is short, have it if it makes you happy. Just maybe don’t overdo it, like with everything else. If you ate kale all day it would be just as problematic. But no one is going around saying “You know, kale feeds cancer”. Also, if I want to down packets of sugar from dawn until dusk that is nobody’s business but my own.

“Be positive!”

I don’t know about you, but my first reaction to anyone telling me to “be” anything is, Why the hell should I? You be it! So obviously this one doesn’t go down well with me. And yet, everyone says it.

“You have to stay positive.” There are two things I resent about this statement. Firstly, it implies that by being positive I somehow will have a hand in curing the cancer. Following this thought through, it also implies that if you’re not positive, well, you only have yourself to blame if it all goes hurtling rapidly down the shit-hole. Sound medical thinking there, thanks Denise. Secondly, it implies my outlook on life is unsuitable and that I need to do it this way, the right way. (As a side note, has telling someone to feel a certain thing ever worked? You feel what you feel, no amount of telling is going to change your complex emotions. Just a tip people: Saying it doesn’t make it real. So shut the hell up).

Going back to the outlook on life issue, I feel like I’m generally a cup-half-full girl. I try to believe people are doing their best (except when they tell you sugar feeds cancer), and I strive to find humor and silver linings in the big and little bumps along the way. But I also get down at times. Since being diagnosed, I’ve had moments when I felt like I couldn’t carry on, when I believed I was actually dying, when I had no fight left. When everything seemed bleak, and yes, negative. And even now that I’m cancer-free, I know there is a (small, but real) chance it will come back. That’s not negativity, it’s statistics. Here’s the thing: Be Positive negates the richness and complexity of our human experience, and reduces a heart-breaking, painful, awakening experience to a happy face emoji. I think the reason people say it is that cancer makes them uncomfortable, and they want you to just frozen-smile your way through it so they will feel untarnished. Well, that’s their problem, not mine.

“You will be fine”

Ok. Really?? Will I really be fine?? Because you say so?? A few years ago, I did a doula training course. It was amazing and bonding and revealing and I remember one thing that was emphasized was never to tell a pregnant or birthing woman she will be fine, BECAUSE YOU DON’T KNOW THIS. All you can do is be there to support her, encourage her, and be her advocate. But you can’t tell her how it’s going to pan out. Nobody can, really. So just don’t say this. Just don’t. The other thing is, like Be Positive, it negates the hugeness of the situation. It reduces a life-changing event to “Oh, don’t worry, it will be just fine.” No need to stress. Oh right, thanks for that, I guess I’ll stop crying into my pillow and frantically scribbling my dying wishes, BECAUSE YOU SAID SO.

“Everything happens for a reason”

Oh, and would you care to enlighten me on what this reason you talk of might be? And would it happen to also be the reason why sea turtles swallow plastic bags, mothers and babies die in childbirth, wars happen, and the world is warming up? And if so, pray tell me, should we sit back and watch because we’re all part of a grand plan? If I do nothing, will my cancer just go away if it is meant to? If I die, will that have been part of the plan? If I get better, will there be a reason for that? You weren’t expecting all the questions? I feel like you are getting the point, so I will stop here. And so should you, a couple beats ahead of ever being tempted to say that phrase to anyone going through something as random and pointless as a cancer diagnosis at 36 years of age.

“You should be thankful you have children already”

Not only do you not get to tell me what I should and should not be thankful for, but you don’t get to assume that because I am still alive that from now onwards I should just shut up and be grateful I was spared, and never ever want or desire anything else. I think this statement is perfect in a way because it encapsulates all that cancer steals from you, and all that it brings to life. A cancer diagnosis means that suddenly you go from planning the rest of your life, to trying to figure out if and how you’re going to be alive in a few months’ time. You go from believing you’re only just getting started, to thinking, This is the literal end. So of course when you find that, after all the treatment, you’re still around, you can’t quite believe your luck. You promise you’ll embrace life and all that it has given you with way more gratitude and grace; you say to yourself that your two healthy children, loving husband, friends and family are enough. That the fact you can get out of bed and breathe without coughing, and pick up your child without pain, and walk and swim and (yes!) do cartwheels is the biggest gift and you will forever be thankful. And I am.

But here’s another thing I have learned: feeling desperately sad, or complaining, or having tough days, does not make you ungrateful. More than one thing can co-exist within me. I can be endlessly thankful for my wonderful children, and yet struggle with the fact I will never have another child. We knew this could happen. Infertility, in all shapes and forms, is the worst form of regret – regret over something that you couldn’t possibly have done. There is no redeeming yourself from it. You’re grieving for what never was. So when a doctor asks me if I have children, and I say yes I have two, and I see relief in their eyes and smile, I think, don’t say it, you know nothing about me.

Advocating for Yourself as a Cancer Patient

This blog was originally published by Everyday Health by Cheryl Alkon on August 22, 2019, here.

When Karen Berk was diagnosed with stage 4 ovarian cancer, taking on the role of advocate helped her gain knowledge, emotional centeredness, and a feeling of control.

By Cheryl Alkon

Last Updated: 8/22/2019

The Berk family, from left: Sarah, Emily, Karen, Jeff, and Jason.

Photo Courtesy of Jeff Berk

When Karen Berk first had trouble breathing, her primary care physician sent her to the nearest ER, where doctors extracted almost 2 liters of fluid from her lungs. A few days later, she went to a follow-up appointment. Expecting to hear that she had some kind of infection, she instead learned she had cancer. Later, she and her husband, Jeff, found out it was ovarian cancer, stage 4.

Berk, then 44 and the mother of toddler twins and a first-grader, was shell-shocked.

“I was in denial,” she said. “Cancer? This can’t be. It didn’t make sense. But now I know it doesn’t have to make sense. In the beginning, I blindly listened to my doctors because I didn’t know what else to do.”

Berk died in November 2018 at age 49 after living with cancer for five years — longer than her physicians predicted she would — during which she experienced several cycles of remission and recurrence. More than a year before her death, she spoke in an interview with Everyday Health about how coming to terms with her diagnosis meant becoming an advocate, for both herself and other people with cancer.

At first, this meant learning all she could. “I researched terminal cancer in general, how you can keep going when you know you are going to die sooner than other people,” she said. “I read as much as I could and learned what I found most helpful,” much of which came from a forum on a website called Inspire, where she could read questions and answers from other people with ovarian cancer.

“In the beginning, I read everything that interested me, which was everything,” Berk said. “I would read posts from people with the same diagnosis who had lived for seven years with stage 4, and I could see that I was not the only one in the world with this condition at my age,” which was a good 20 years younger than the typical woman with ovarian cancer.

“It gave me knowledge and power and control, and that made me feel better,” she said.

Getting Emotionally Centered

After receiving a stage 4 cancer diagnosis, patients may feel the need to tamp down their anxiety, anger, and fear and focus their energy on coming up with an action plan. But experts say it’s important for patients to take the time they need to process their feelings and look for emotional support.

“Try not to rush into anything,” says Lidia Schapira, MD, an associate professor of medicine at Stanford University Medical Center in California and the director of Stanford’s cancer survivorship program. “Try to harness compassion toward yourself.”

Coping with a cancer diagnosis means feeling what you need to feel, not just throwing yourself into crisis mode. “It’s a time to let advocacy turn inwards,” Dr. Schapira says.

Finding Your People

For Berk, traditional cancer support groups “weren’t good for me at all,” in part because she was much younger than everyone else. For instance, other participants would share that when they felt sad, they would play with their grandchildren for a mood boost. “My own kids were young, and I knew I’d never [live long enough to meet my] grandkids,” Berk said. “I stopped going to the groups because they made me feel worse.”

Berk found more comfort at a retreat in Stowe, Vermont, called the Stowe Weekend of Hope, an annual, low- or no-cost event for people with cancer and their families. Not only did she meet women she connected with, she learned information that proved invaluable.

It was May 2015, and Berk was dealing with her first recurrence after chemotherapy and radiation had sent her cancer into remission. She had terrible fatigue and was forced to use a walker. None of the doctors who had treated her in Massachusetts, where she lived, felt they could help her. But she’d found an oncologist in California who specialized in helping people with her particular type of recurrence. She just needed to figure out how to pay for the West Coast flight.

When she shared her dilemma with other attendees at the retreat, someone brought up Angel Airlines for Cancer Patients, a charity for people who need financial help to travel for treatment. Berk was able to get a $600 credit to fly to California. She saw the doctor, who recommended a chemotherapy regimen for her to pursue in Boston. Within a few months of starting the chemo, she could walk without a walker again.

Advocating for Others

As Berk’s health improved, she shared her insights on the Inspire forum. For instance, after two recurrences, Berk began taking a new targeted medication called Lynparza (olaparib) in pill form at home. She described how she worked with her insurance provider to avoid having to pay out of pocket for the drug, which can cost up to $10,000 a month. She also posted about her experiences taking the medication and answered questions about side effects.

On the forum she urged others to advocate for themselves: “Be on top of everything — know what your CA125 numbers are [the markers used to determine whether cancer has returned] and what your MRI results are [to determine exactly where in the body cancer has returned]. Push for results when you have an MRI, and call every day until you get results. When you can catch recurrences early, there are more options.”

Berk also encouraged other people on the forum to take advantage of “the stupid little things,” as she called them, that make cancer treatment less onerous. As an example, she described talking to a social worker at her oncologist’s office to work out how to get her parking fees waived: “I’m going there for the rest of my life, at least once a month, and paying $12 to $15 each time.” The social worker was able to get her a pass for free parking. “There are things you can take advantage of if you know who to ask,” Berk said.

There are a lot of ways to become a cancer advocate. “Everyone can do something,” Schapira points out. “I think all of us need to help others through bad times, and however someone can give back, whether it’s starting a new nonprofit that addresses a need, joining a fund-raising walk, or making a small financial donation to a group on the ground doing this kind of work.”

Is It Difficult to Participate in a Clinical Trial?

Clinical Trial Mythbusters

Clinical Trial Mythbusters: Is It Difficult to Participate in a Clinical Trial? from Patient Empowerment Network on Vimeo.

Downloadable Program Guide

Three experts discuss the clinical trial process and the difficulty in participating in a trial. Our expert panel includes:

Ken Getz, MBA – Founder and Board Chair, CISCRP
Andy Lee – Senior Vice President and Head, Global Clinical Trial Operations, Merck
T.J. Sharpe – Melanoma Survivor and Patient Advocate

Transcript:

Andrew Schorr:

And greetings from Southern California. I’m Andrew Schorr from Patient Power. And welcome to this Patient Empowerment Network program, another in our series of Clinical Trials MythBusters. Our goal, of course, is to help you get the treatment for you or a loved one that you need and deserve. I want to thank the financial supporters for this program to the Patient Empowerment Network; AbbVie, Inc., Celgene Corporation. Daiichi Sankyo and Novartis for their support. They have no editorial control and we’re going to have a very freewheeling discussion today. And really what it’s about is how can a clinical trial be made easier for you to participate? Are there barriers? We’ve talked about it in previous programs. But specifically, what are the companies—the pharmaceutical industry mostly, who sponsor trials all around the world, what are they doing to make trial participation easier? For you to know about trials. For the people at your clinic to know about it and what to say and how to administer it. For you to have documents that are understandable for you and your family to know whether you want to participate. To keep you informed. And also related to the requirements of trials. How can they be relaxed a little so that there may be a trial that would benefit you, that you and your doctor agree on, and the requirements of it allow you to be in the trial. Okay, and the logistics of it are not so tough either. All right, I’ve been in two clinical trials, and I believe I’m alive today because of that. So, I’m very grateful. We have some wonderful panelists with us over the next hour. Now as you have questions, send them to questions@patientpower.info. And some of you have. So, you’ll be able to interact with us as we go along. First, I want to go to Ft. Lauderdale, Florida, and T.J Sharpe. And T.J. has been on programs with me over the years. Stage four melanoma patient having been in trials. And T. J., you would agree, you’re alive today because you were in trials, right?

T.J. Sharpe:

Absolutely, Andrew. I think both of us are very fortunate that we found a trial that was the right treatment for us and gave us the ability to combat our disease in areas may not have been available to us if we just waited for standard of care therapies.

Andrew Schorr:

Right. And here you are—we should say that you were diagnosed a number of years ago with melanoma, went through trials. And now you’ve had two years without treatment, right?

T.J. Sharpe:

Yes. It’s been five years of treatment followed by now two good years of a clean bill of health.

Andrew Schorr:

Well, great. And I should mention for our audience, many people are familiar with T.J. T.J. goes around the country, gives speeches. He’s been at many events, consults with industry that are developing trials to try to bring the patient perspective forward. So, T.J., thank you for all you do. We really appreciate it.

T.J. Sharpe:

You’re welcome. It’s my honor to be able to represent all these patients.

Andrew Schorr:
Well, most every family—certainly most have been touched by cancer. But our other guests are not cancer patients but are in national leadership programs. And so, let’s go up to Medford, Massachusetts at Tufts University outside Boston, Ken Getz. Ken, welcome to the program. Ken, ladies and gentlemen, is a true national leader when it comes to clinical trials and really helping us move forward with better processes, better understanding. Ken, tell us a little bit about your organization there, CISCRP. What does that stand for?

Ken Getz:

Thank you. And I have to say your pronunciation was nearly perfect. It’s hard to pronounce it. It’s an acronym and it stands for The Center for Information and Study on Clinical Research Participation. It’s a non-profit organization. It was founded 18 years ago. And it’s really there to help patients and their families navigate the whole clinical research progress which for many is completely unfamiliar terrain until they’ve been diagnosed with an illness or when they have exhausted all other treatment options. So, CISCRP really helps people become more educated and informed so that they can really think of the clinical research process with more confidence. And they can navigate this unknown terrain.

Andrew Schorr:

All right. I’m going to come back to you in a minute because you have such an overview, and you’re also an Associate Professor at Tufts. And so, you study all this, and you’ve written books. But I want to introduce the third guest. And that is a leader from the pharmaceutical industry and one of our most respected and venerable companies in the field, and this is Merck. So, joining us in a senior vice president of clinical operations there around the world. And that’s Andy Lee. Andy, welcome. Thank you so much for being with us.

Andy Lee:

Andrew, thank you. And pleasure to be with some prestigious panelists, both of whom I know. And I’ve met you over the last two weeks. And thank you to T.J. and yourself who have been trial participants and who are representing that part of the organization.

Andrew Schorr:

Okay, and we should mention that both T.J. and Andy are working on a couple of levels. And Ken sounds off on this too. There is a group called TransCelerate where pharmaceutical industry is working together on some of the issues they face in having the proliferation of trials. More trials sites, more accessibility, procedures for that. And then, of course, Andy has helped lead that effort at Merck related to breakthrough therapies that they have been trying to develop there in supporting patients who might be in Merck trials. So, we are going to come back to that. But I want to go to you for a second, Ken. Ken, how low is the participation among adults in clinical trials, at least in the U.S. Now, I’ve heard really low percentages. Where are we now with that?

Ken Getz:

Right, it’s a great myth for us to start with, this notion that only three to five percent of patients—eligible patients, participate in clinical research. That’s actually a statistic that was published by the National Cancer Institute in the early 1990’s. The latest research really shows that it varies widely. For example, when we look at pediatric cancers, the participation rates are extremely high, 80 to 90 percent in same cases—pediatric leukemia. In part because those communities have very engaged healthcare providers, very engaged families that really share their information. It’s just an enabled community where all of the stakeholders support participation. And then there are other areas of course. Some cancers where we do see relatively low participation rates. But I want to point out that low participation is driven by so many factors, Andy, including the strict eligibility criteria. And the demanding protocol designs which are a real burden for some people, and they choose not to participate. As well as low awareness, very low accessibility to trials among minorities and underserved communities. So, there are many factors that contribute to this variation in the participation rates.

Andrew Schorr:

Yeah, you’ve ticked off some now. T.J., in your own experience, one of the breakthrough trials you were in you had to go from Ft. Lauderdale in South Florida and move your whole family to Tampa in central Florida, right. I mean that was a big deal.

T.J. Sharpe:

Absolutely. When you have a young family and a stage four cancer diagnosis, relocating simply across the state during the holidays especially, is no big deal. We were fortunate because we had the means to be able to move there with work situation, with family. But too many people can barely go across the county, much less the state or the country to find a trial that might be the best match for them.

Andrew Schorr:

Andy, so we’ve ticked off some of the obstacles, and Ken touched on some about even the proliferation of trials. Is that a lot of what you do is how can we have trials be more accessible, be more widely distributed to a clinic near you?

Andy Lee:

Yes, let me just explain. When we look at a new cancer therapy, we look at the various cancers that may be affected. And what we do is we go for high probabilities of success. And the challenge is if you bring a new cancer agent. You normally start off in very advanced disease. So, patients would have failed multiple lines of therapy, and often it is a last gasp. And you have to show some sort of clinical efficacy. And then you move sort of backwards in the disease, and you go from sort of third-plus line, second line and first line.

And then you may work downwards into earlier stages of the disease into an adjuvant setting and maybe a neoadjuvant setting. So, as we sit down and design a trial, what we need to look at is what is the population that is most likely to show any benefit at all. And quite often when you are developing a new therapy, it’s difficult to show benefit because many of the patients are very ill. So, what we have to do is optimize the opportunity for success of a compound by going to the right target patients.

And quite often as we have learned a lot more about cancer, this does not mean we test a product broadly in anyone with cancer. We typically try and find a profile of a patient that is likely to respond. And many patients now will realize their predictor biomarkers or prognostic biomarkers. So, for example, with immunotherapies, those that work through the PD1 mechanism would probably want to have a PD1 ligand receptor positive patient who is likely to bind to the drug.

And that gives a higher probability of success. So, it sounds counterintuitive that while we want to develop therapies for all cancer patients, when we start clinical trial development, we have to show efficacy in a population that will benefit. And that’s normally predefined and makes the inclusion criteria fairly strict. As we show efficacy and as we can move into broader populations, it makes it a lot easier for us to design more liberal clinical trials. And then we can actually spread those in the geographic domains.

I could talk more about geographic allocation, but let’s hold that for the time being, and let’s see if there’s time later on.

Ken Getz:

Can I just add to what Andy said because I think it’s really important for your viewers to understand just how active drug development activity is today. We’re looking at over 4,000 pharmaceutical and biotechnology companies, some of them very, very small. But in total, we’re looking at nearly 6,000 drugs that are in active clinical trials. And to Andy’s point, many are really targeting a patient with a very specific genetic profile or a specific biomarker. But it should give anyone who believes that a clinical trial may be an important care option for them, they should recognize that there may be many, many trials out there.

In total we estimate as many as 80,000 clinical trials, nearly 50 just conducted in the U.S. alone—50,000. So, it’s just important that we keep all this activity in perspective.

Andrew Schorr:
Right. So, T.J., that’s why all of us as patients need to ask about them, right? Go to different resources, whether it’s an advocacy group that you ultimately spoke with other patients, and obviously quizzing the doctors we go to. Is there something that may line up with my situation, right T.J.?

T.J. Sharpe:

Absolutely. There is a both top down and bottom up approach here that patients as they become educated—and every patient should be the owner of their healthcare as they become educated. Hopefully they are coming across advocacy organizations, other informed patients, patient support groups—all of which will help inform them different options for disease treatment, including hopefully as Ken mentioned, clinical research as a care option. At the same time, there is certainly very much an opportunity from the top down from the sponsors who develop the trials and from the sites that execute them to educate patients as they come in.

Not just at their own site, but at any site, at any medical facility. That if you have a diagnosis and you are looking into your care options, that you should be asking the question. And we should be giving you more information on the possibility of clinical trials and where you may find clinical trials that are appropriate for you.

Andrew Schorr:

Right, the whole enchilada, if you will, of all your options. Andy, so you mentioned about trial requirements. So, first of all, what efforts either at Merck or are you aware in the industry are being made to really talk to patients early on as you are designing trials? Whether it’s the requirements—how many CT scans you’re going to have. How often you are going to have to go to the main trial site. All the different things that sometimes get in the way.

Andy Lee:

Well, firstly we start with design. And we believe in exquisite trial design, quality by design as well. So, what we want is to run the experiment once and not have a sloppy trial design. We want to make it really robust in terms of scientific integrity and operational execution. So, we have a lot of internal design committees and what we do is we co-op with many groups external to our company. So, we speak to people who run clinical trials at cancer institutes.

We speak to the doctors who manage this. We speak to the trial coordinators. We speak to people involved with the transporting and shipping of medicine how they would do that. And then we of course speak to people in the ecosystem. We quite often speak to investigational review boards before we start trials. We talk to them about our design and what would be best to protect the rights and well-being of patients. And then, of course, the patient-centric approach says that we need patient insights.

And I’ve chosen my words very carefully because the insights are really important. Not all patients—and I’m very respectful that some patients are very intelligent and actually may be involved in this. Some patients can contribute to design, not all can. And so, what we do is we take the insights and we impute those. We often have focus groups. We talk about this disease. We talk about the burden of the disease. And then we talk about how that disease is managed in an ecosystem. And quite often in different countries it’s managed differently.

And so, we have to appreciate the global clinical trials have to navigate a path that may not be a linear path as we’d see it at an exquisite elite cancer center in the United States. It’s community-based, it’s all the rest. So, we take that input, and what we try to do is unburden the trial for the patient. We say, “How can we design a trial that requires the least visits to the clinic—the hospital, the least burden for them. And how can we take some of that burden from the clinic and actually transfer that into an easier environment.

So, document reading and review. Perhaps filling in questionnaires about quality of life. These are things that don’t have to be done in the clinic itself. And then often when we work with clinics, we work with them to help them understand how we as sponsors can make their life easier. And some of those things might be simplifying the informed consent. But I want to stress just one point here is that we can do whatever we like in the design at a company.

One of the things is, the patients are not sponsor patients. Okay, we sponsor clinical trials. The patients are managed by a doctor and a professional. And underneath that principal investigator is a whole oncology team. And it involves radiology. It involves pharmacists. It revolves around a 360 multidisciplinary team. They’re exquisite. They help manage the patient, not the sponsor. We provide the enabling functions for them. And then also that the oversight of the patient’s right, safety and wellbeing is the responsibility of an institutional review board.

And while we may provide templates and simplify templates in text and language, we rely heavily on the institutional review boards to help us with things that may make things easier, such as reimbursement for parking, transport, all of these things. And by and large, the institutional review boards are very supportive of these things. But they are very difficult to quantify in exact terms because of different geographic regions and different norms in different places. So, we rely heavily on exquisitely well-trained 360 team who manages oncology patients with a great PI. They manage patients.

And we work collaboratively with the sites who work with patients on our behalf. So, I just wanted to say the myth is that sponsors interactive with patients. That’s a myth. And the truth is that we engage with clinical sites, and we try and make our design and all the elements—the enabling elements, simpler for the trial sites in order to manage the patients in a simpler way.

Andrew Schorr:

Okay. Thank you for that. So, Ken, I want your comment on that. Because okay, we are downstream patients. We have a doctor, healthcare team. And we know somewhere in the background there’s a sponsor that tried to enable good things to happen to get reliable data and hopefully a cure for us. So, how do we—what’s happening? Are we improving things there in that interaction between clinic and patient?

Ken Getz:

Yes, we absolutely are. And I’ll start by just echoing and acknowledging that Andy has really laid out just an incredible amount of input that goes into the design of a protocol. And that’s really for a really large company. We see many, many examples now of patient advocacy groups or smaller companies turning to a variety of approaches to solicit input from patients and healthcare providers. Some virtual approaches through a social media or digital community. So, there’s lots of ways that feedback is being channeled.

And that’s really important. The flip side, to really answer your question, is that our protocol designs are becoming more and more complex, more and more demanding. A much larger proportion of drugs are now targeting rare diseases that have been stricter inclusion and exclusion criteria. And the designs of the studies—the number of procedures and the number of visits. The number of investigators that are involved, all of that has also continued to grow. And as a result, we do see that our trials are taking longer.

We have yet to see a year when we actually witnessed a reduction in the cycle time to conduct a clinical trial. And we just have to figure out new ways of making the participation process less burdensome and more efficient.

Andrew Schorr:

Oh, my. So, T.J., you had been living with stage four melanoma, a life-threatening condition. We have people even on our team who are living with stage four disease. So, when Ken talks about things slowing, that’s not what we want to hear. We want to hear two things. One is, we can accelerate a development of new medicine. And ideally—because this is an issue certainly in the U.S., but I think worldwide, that by speeding the process, cutting through red tape, improving procedures and us participating, the cost can be less as well.

And when we talk about cancer, the costs are going through the roof as you know for people living with chronic cancer. And you know so well, Andy, people who are on some of the medicines that you’ve come out with at Merck. Where people used to die unfortunately in short order, are living a much longer life thanks to new medicines. We want it to happen faster and be financially achievable. Andy, any comment about the pace of science?

Andy Lee:

Yeah, I would like to make a couple of comments about that. We often hear the sort of story that 80 percent of clinical trials don’t recruit on time, et cetera. We do immense feasibility. Once we have designed a protocol, we send it out to all of the countries that could potentially work with us. We have staff in 47 countries. And they look at two areas of interest. One is the medical durability, is the comparator the one we use in our country. Is the protocol designed the way we practice clinical medicine, not clinical research medicine?

And will that enable us to recruit the patients? That’s the first level. The second level we look at is to ask the question, is this operationally feasible? Can we source the comparator? Do the clinical sites have the equipment? How would we have to ship the biological samples around the world? And based on medical durability and the operational durability, we do a site selection. And we run the indicators through a Monte Carlo simulation. And we simulate this trial. What if we took three countries out? What if we added this more sites? What if we changed this inclusion?

And we come up with a model of what the recruitment would look like. And recruit about 80 percent of our trials according to our model. So, about 80 percent of our trials recruit on our model time. And then if we look at the typical time for drug development, it has been from eight to 10 years for many years in the industry. And when we look at some of the development timelines now—the cycle times. Pembrolizumab (Keytruda), for example, from first study until first approval, was 60 percent reduction in time.

We were looking in the four-year time period. And we are looking at five or six years for many indications. And so, we’ve halved that cycle time for some of the newer oncology products. And there are a number of reasons we’ve done that. One is we have found operational efficiencies. Two is the trial design has enabled us to interim analysis with independent data monitoring committees to assist with that. I’d also like to put in a positive plug for the regulators.

I do believe—and I’ll talk specifically about the FDA, because they are the agency for the United States. They have revolutionized the way they approach the designs and the way they review the data. And they have breakthrough designation status they’ll give to compounds that are really looking like they have strong efficacy. And so, the approval process through the agency has improved remarkably. And they’re open to adaptive designs. And they are open to interim analysis. And they are open to all sorts of things.

So, I really wanted to give credit to our agency who has said, “Where there’s a need for breakthrough medications, we’ll try to find the path.” And so, I do believe there’s a real positive side to this. The challenge is the market is saturated. We have now more than 25 PD1s in development. And to put the 25th one in there, they are so far behind in development. I wonder what that does. It clogs up the system. So, when you look at how can we influence sites, at the top sites we only get one or two patients.

And we compete with 50, 60, 70, 80 other sponsors. And so, it becomes so saturated that, that site has to learn to do systems and process with 70 companies. And what they are doing is almost hedging. They are not focusing on certain things. So, in those cancer centers, they offer treatment for all lines of therapy and all types of cancer, the specialized and nonspecialized. And we are moving out of that sort of geography and moving it community-based oncology practices where it’s less saturated, and we can actually have more traction there and be able to engage more with the clinical trial enterprise for the good of the patients.

Andrew Schorr:

Ken, you write books about all kinds of issues around this. So, if we are getting—particularly in oncology to have trials offered at the community practice where those doctors work night and day—the nurses. They are really stretched. More and more cancers, genomic subtypes, most sophisticated testing. How—what would you say the patient can do. T.J. talked about it a little bit. What would you recommend to patients so that at that community oncology practice the patient and the family can kind of discover what may be available for them as Merck and other companies try to get these trials distributed?

Ken Getz:
Right, well you—talk about the whole enchilada, Andrew. You’re really touching on it. It’s also very exciting times for patients, not just cancer patients, but patients that are dealing with any chronic and severe illness today. And it’s really all about more of a partnership with the clinical care environment and clinical research. And of course, at the heart of it is the patients and their family being as informed as possible, sharing their electronic health and medical information so that they can be connected to trials that might be appropriate for them.

But it’s moving—as Andy said, away from the classic places where trials used to be conducted. And in many cases, they were at these dedicated centers that only conducted clinical trials. It’s a very competitive environment now for patients. So, many sponsor companies like Merck and others are looking at clinical care settings and moving into communities or, in some cases, large health systems where you can have clinical research professionals who will supplement and provide support to the healthcare providers, so they’re not stretched too thin.

But so that they have the clinical research capability onsite at the point of care. For patients it’s a great opportunity because now they have the opportunity to get their own healthcare or treating physician and treating nurse involved in a clinical trial as part of their overall care. And we expect to see more of that over time. We expect to see other virtual trials or opportunities for patients to participate in the comfort of their own home tied in with their clinical care setting.

And all of this is relatively new to the whole world of clinical trials and the investigation of experimental medications.

Andrew Schorr:

You touched on something I just want to follow up on. I’ve heard of this term site-less trials where you said you participate in your home. So, T.J. had to go from Ft. Lauderdale to Tampa. I had to go from Seattle to Houston. There are not—this is a big deal, especially if you have little kids as I did, he has. So—and away from work and whatever your situation is. So, is technology going to come in play so Andy can get the data he needs for the FDA, but that we can have technology help accrue that data in a more efficient way.

Ken Getz:

And I’ll say absolutely. And my colleagues here today I’m sure can comment on this as well. But absolutely. We are seeing wearable technologies and mobile applications that now have the ability measure vital signs and other important baseline information in a validated manner. There are ways that you can access a specific facility for a highly specialized test, specialized imaging for example where the technician can evaluate it remotely. Blood can be drawn at remote locations as well.

So, there are lots of places where we have sort of this more flexible environment that can cater more to the patients and less about a specific physical facility where you have to go to participate in a trial.

Andrew Schorr:
T.J., I want to talk to you about diversity. So, you and I are kind of middle-class white guys. But we want to know how new medicines work for a variety of populations, ethnically, economic groups, et cetera. And Andy needs that data. And he goes to the FDA, and the FDA says, “Well, do you have Hispanic people? Do you have Asian people? Do you have African American people?” or whatever the country is because he works globally. And they say, “We want to understand are there differences?”

How are we doing with that. How can we make a difference there so that we really know what medicines make a difference for broader and also distinct populations?

T.J. Sharpe:
I’m sure Ken can back up some of these things with more hard data than I can. I know that different populations have different levels of trust with the medical system. One thing that you and I both experienced was a lack of options—a lack of good options. And when you get into dire straits, you tend to be a little more trustful of anything that comes along. But we have serious or chronic conditions that have proven treatments that might not be the most effective for certain populations.

And we’re not able to broad the scope to these minority populations or populations that don’t have access to NCI designated cancer centers or top-notch medical facilities. They are not able to get either in a trial that is looking for a drug that would help them or even get access to medicines that have been recently approved simply because their healthcare situation doesn’t allow it. Whether that’s a lack of insurance, a lack of healthcare literacy or simply a mistrust of—there’s a lot of generational mistrust I think in some communities of the clinical trial system.

So, as an advocate, I certainly push caretakers especially—and children caregivers for older populations who are maybe first or second-generation Americans to help facilitate a conversation between the medical professional who’s trusted and a patient that might not be able to get or rely on the information they’re given. Because it really will speak to populations that don’t get the opportunities that you and I have gotten simply because they are either not aware, or there is a barrier there to get to that medical professional.

Ken Getz:

I appreciate, T.J., you mentioned CISCRP. That’s one of the things that we’ve focused on for 18 years is bringing clinical research education into major metropolitan areas around the U.S. and parts of northern and western Europe where we plan for several months, and then we put on what we call an Aware for All events. And we really work very hard to encourage participation by—or from patients based within minority or underserved communities.

And I’m happy to say that we’ve had a lot of success with that. These are really difficult communities to reach through a lot of the traditional approaches. We have to rely on community centers and clergy and other approaches to really help these communities, for a lot of the reasons T.J. mentioned, trust the educational information, and come out to learn more. And I’m happy to say we’re seeing more and more people of diverse backgrounds that are curious and interested in learning more about clinical research, especially knowing that representative populations provide more information that can inform treatment for different types of patient sub-populations.

Andrew Schorr:
I want to go to Andy in a second. Andy, just one second. I wanted to mention and call out—and Andy’s company has been a leader in this. He was talking about PD1 and all of that. But drugs that have been breakthrough in immunotherapy for people like T.J. where—and it’s being explored in broader cancers where otherwise life was going to be short. And how to activate the immune system and really fight the cancer in people living long term. So, the people in those trials—and certainly there were people in the melanoma trials like yourself T.J.

Lung cancer trials and increasingly now others who did get tomorrow’s medicine today. Andy talked about accelerated approval which is great. So, that’s the impetus for the patient and the family. Is there the chance to get tomorrow’s medicine today? Now the obstacles may be distrust. You talked about that, Ken. And also, is maybe accessibility. Is it as a clinic near you? And Andy you talked about pushing that out. And then sometimes it’s related to cost.

Now is there anything that sponsors can do, Andy, related to the costs that people may have in being in certain trials? Where do we stand with that?

Andy Lee:

Yeah, so I’ll just touch on the distribution first and then get into the costs because they are linked. When we prosecute global trials—we’ve had a very U.S.-centric discussion so far. But cancers present differently in different geographic regions of the world. And so, when we want speed out of our trials. You want me to shorten that timeline and get drugs to market quickly. I do it internationally and in some cancers like esophageal cancer or some of the gastrointestinal cancers, Asia has a much higher prevalence of these cancers.

And we do a greater proportion of work there. We always include multi-country studies. And U.S. may have a greater proportion in other areas. So, we balance that out to optimize speed. Of course, with clinical trials the cost structure around the globe is very different. But let’s talk about U.S. We have spoken about a saturated core of clinical trial sites that we all go to. And I speak generally now for all sponsors. And we are all looking to optimize and get great efficiency.

At the same time, we realize we have many underrepresented geographies and ethnic groups—and not just ethnic groups, but under resourced populations. And so, what we’ve been thinking about is how can we support people, and support people at all levels. And so, we start off with thinking about the cost structure, and we obviously pay clinical sites for what they do. But we will support all sorts of things. We’ve been negotiating with Uber and Lyft, so we can build that into automated transport for patients.

Again, the IRB has to approve that. We are looking at ways to augment that they are not out-of-pocket for things. And we’ve been talking a lot with a group called Lazarex Foundation who has really expanded into under resourced communities and found ways to ensure that they have daycare and different access for those patients. We have worked extensively now to look at outreach programs into communities that typically wouldn’t be in trials. We are focusing in two areas right now as we speak.

One is next generation of HIV medicines, and the other one is in prostate cancer. And we’ve got a large program rolling out in prostate cancer. So, what we are doing is going into sites and we have put together training videos and training materials. And we are looking at cultural competency. So, it starts at the site. Are they culturally competent to engage a different community? And we’ve spoken about working with the community churches, community education systems.

And so that starts with cultural competency. I have a woman, Madelyn Goday, who works on this day and night in my organization. And she’s very strong at this. It’s early days, but if we can show that it works in one or two therapeutic areas and cancer types, we’d expand it further and further. But we can’t just have a shotgun approach and just go and do 100 sites and hope it works. Hope isn’t a good strategy. We are working systematically to engage different people. And as appropriate and approved by ethics committees, we will support all of these communities and help build infrastructure and capacity.

Those are important things for us. But as I said, where appropriate and where it’s sustainable. We can’t just throw money at something in the hopes something sticks. We have to have something sustainable and it goes to what Ken says, and that’s education and providing resources and materials. And we’ve used quite a lot of Ken’s materials in multiple clinical trials. Thank you for that, Ken. It’s been really helpful for us.

Andrew Schorr:

Great. I wanted to note for your audience. If you have a question, send it to questions@patientpower.info. We have expert panelists here. And this is really—we are all in this together. I think you hear the dedication from Andy at Merck and T.J. as a patient advocate and Ken as a professor and founder of organizations devoted to this. We want obviously accelerate medicines, but have the accurate data of how it affects different people, who is it right for so that the regulators—and thank you for what you said about the FDA here in the U.S., has the information to make a decision on should this medicine be available for people with that diagnosis.

Okay, so what about staying in the trial. So, T.J., how long—let’s take with the Keytruda trial or one of them. How long were you in to for?

T.J. Sharpe:

Nearly four years. Three-and-a-half years.

Andrew Schorr:

Were there ever times when you said, “I’m done. I want to bail out.” You know.

T.J. Sharpe:

I’ll be very careful how I answer this question for Andy’s sake.

Andy Lee:

It’s okay, T.J., we’re friends.

T.J. Sharpe:

No, probably the biggest crossroads I ever came to was when one of my tumors started growing about a year into it. And we weren’t sure if the medicine stopped working or not. We didn’t know what to do. And as it turned out, it was still working. And I think was just one spot that wasn’t responding. But everything else had responded great. However, at the point, as a patient, you’re thinking about yourself first and your family first and the trial second. It’s easy to stay compliant on a trial when things are going well.

But when you’re ahead of the medicine in some ways, and I think patients with chronic illnesses or in some cases rare diseases, are almost more knowledgeable than some of their doctors or the trial protocols about when they’re stopping. They don’t have the luxury of finishing out a protocol and seeing where their disease journey takes them. And the best example I can give of this is a very passionate advocate by the name of Jack Wheelen who we unfortunately lost a couple of years ago, but whose influence has kind of dominated the patient advocacy world for the last decade or so.

And Jack was able to monitor his health almost better than a doctor. And he knew when his trials weren’t working. When we get to that point in a clinical trial setting where we know the medicine is not being effective or where a patient would be better served to move on to another treatment. That’s when we are going to take the next step in clinical research, because now we’re aligning the trial design and the trial goals with a patient and a patient’s family’s treatment goals. And as those two points merge, that’s where clinical research becomes that much more effective as a care option.

Andrew Schorr:

That was well said. And I think with all those trials, you’re right, the team—that care team, what’s right for you at that time. Obviously to get the data, but also not at all costs. In other words, if the data is showing something is no longer effective for you, is there another treatment or a trial? I’ll just share my story for a second. So, I was in a phase two trial of combination therapies—which are increasingly common certainly in oncology. And after three months—halfway in the trial, my blood was kind of cleaned up.

And I had nausea and some other side effects. And I said to the trial coordinator, “You know, I think I’d like to stop.” And she said, “You know, our belief is that you still have microscopic illness in your bone marrow—in this case with the blood cancer, and the additional three months in this protocol will make a long-term difference for you. That’s what we believe.” They didn’t have the answer, but that’s what they believed. You know what? I stuck it out. She was right. I had 17-year remission.

If I’d stopped after three months, would I have? So, it’s a dialogue with the care team Andy, right? It’s this ongoing discussion not just entering the trial, but remaining in the trial, correct?

Andy Lee:

Yes. Absolutely. And I just wanted to impress a really important thing. People talk about people dropping out of trials. In cancer trials we see extremely low drop out. I mean these are potentially lifesaving medicines for all of the companies. But what we do want to make sure about is that when there is progression of disease, and it’s shown that the drug—whichever it is, the control arm or the active arm or the new agent, where there is progression of disease that they get the best available therapy.

And so that often contaminates trials because we have the crossover effect that now they are getting maybe the experimental agent in the standard of care type of thing. But most important thing for us is to track the survival of the patient, regardless of whether they go on another therapy. And we have put a tremendous amount of effort into looking at the informed consent and making sure we work with IRB to track patients long term survival.

Because as you’ve said, you may have a short-term issue that shows that the drug may not be working short term, but long term it may have prolonged and profound effects. Positive or negative, we don’t know that. And so, what we like to do is get long term survival. And we ask patients to consider when they sign the consent for whatever trial and whichever sponsor is sponsoring this, is to consider that knowing their status throughout their treatment—whether it’s on a sponsor’s drug or another sponsor’s drug or x therapy. It is really important — and I ask people to think about that.

Because that really helps us get as much data out of the individual treatment as possible. And that may prevent nonrequired trials in the future or it may say, “Wow, that really informed.” And we’d like to inform all cancer patients. If data we generate can inform other therapies, we certainly want to do that. We do not want to do wasteful clinical trials. So, tracking patients long term or patients—the message to patients is being cognizant of letting the sponsor—and the sponsor could be an institution. Letting them know your status is really important. All they want to know is are you dead or alive.

Andrew Schorr:

In the end, just one thing is, are we partners. In the end, our viewers here, are we your partner? And can we feel that not just for their doctor but you guys behind the scenes with the labs and everything, that in the end we are partners. And unless we see it that way, we won’t get anywhere.

Andy Lee:

Absolutely. I’m glad you used the term partners. Because when we’ve done a prep for this people have said, “Are they investors in the thing?” So, yes, patients invest their time and everything, but they are partners in research. They are contributing so much. They are contributing—they are going into the absolute unknown. And there is an immense trust level that is there. And we owe that back as research professionals is to treat people with respect, dignity and as partners, to make information available, to publish our data to get it out there as quickly as possible. And to make sure we get that back into the participant’s sort of hands.

Andrew Schorr:

So, Ken, how are we doing on that because you go back over the years and people say, “I don’t want to be in a trial because I’ll be a guinea pig,” and respect was not seen as part of it.

Ken Getz:
Well, that’s also a bit of a myth, right? You had a few that claimed that they felt the process made them feel like a guinea pig. The vast majority of people, over 90% of people who participate in a trial, would do it again. So, once they get past that unfamiliar area where they’ve perhaps only heard a few case examples or a few very vocal people who had bad experiences. Once they’ve done it themselves or they’ve been able to work with a group of advocates that really help them think about this process, and they become more educated, generally they’re very impressed with the level of professionalism, the compassion that exists at all levels.

I work with so many professionals—science professionals and pharmaceutical companies and at the research centers, and they all share that kind of commitment that Andy just mentioned. There’s a real desire to partner with the patient to really inform them. I would say one place where we need to see much, much more however is in the return of clinical trial results in a plain language to people who’ve been in trials. That’s a place where as an enterprise—government, research sponsors as well as industry have not really made this a standard practice at this point. And that’s one thing that we’re really working on actively.

Andrew Schorr:

Right. Great. So, T.J., you and I are investors—and Ken used that term and Andy used it, and I’ve always believed it. We are investors of our tissue, our body, our future to help other people and hopefully help ourselves. And certainly, for profit companies that may greatly benefit if they have a blockbuster therapy. But we need to be kept informed in the long term, right T.J.? We want to know what a difference our participation made.

T.J. Sharpe:

Certainly. And I think to echo what both Andy and Ken said is that patients do become partners. Patients who are involved in clinical research, a significant chunk become altruistically invested. I’ve heard more than once, “Even if this doesn’t help me, I’m glad I participated because it might help somebody else.” I know I’ve felt like that, and I’d venture that you’ve had some of that too, Andrew on your journey. So, it’s only—it’s at the very minimal fair, and it’s certainly very justified to expect as a co-participant in this.

And as kind of a co-creator of science with sites and sponsors that we understand what has come of our sacrifice and our time dedication to helping science out. We shouldn’t have to find it out through press releases from ASCO or hope that we hear about it on the nightly news. We deserve to hear what has happened. Not just because it can affect us as people and as patients, but that we put a lot into this too. And then we did our part to further medical research and we want to be part of the—whatever the end of the trial ends up being. We want to be aware of that. Not just for personal knowledge, but to know that it’s going to help this many other people.

Andrew Schorr:

Right, to be honored. So, Andy, at Merck you’ve established some internet platforms in particular related to keeping people informed, right?

Andy Lee:

Well, we’ve got an internet platform that people can log onto. I’m happy to share that with you; in which they can get access to a list of our trials. So, I didn’t prepare this but especially, but I did make a handmade note. And if anyone wants, it’s a very simple log on. Andrew Schorr:
You’re a great artist.

Andy Lee:

And it’s a simple one. What that will get you access to is two main important things. One is it gives access to information about clinical trials. We have a tab on there that tells everyone about the phases of clinical trials and what to expect in a trial. So, it’s an educational part. Then we have a lot of information about the Keytruda clinical trials were, are running, and they’re called keynote trials. And there you can look at the different indications. And you can look up and it has a telephone number you can call.

Now I must stress is that we run over 1,000 clinical trials in oncology. But many of them are not sponsored by us, they are investigator sponsored trials. So, you can go to clinics, and they run their own clinical trials that are not sponsor-related. And the NCI runs their clinical trials. So, there are a lot of different sources. And many companies will have clinical trials. We also have the website clinicaltrials.gov. I’ve had to use that in the last two days for a colleague.

And you can navigate that and look for different types of trials. And you can look at different products and everything. It’s not perfect. But at least it’s a place to go to. And I don’t want to sound as if I’m one sponsor centric. Many other companies have access to websites, and they really want to try and enhance and direct people to the clinical trials sites at which they are working.

Andrew Schorr:

Right, absolutely. And then you were working at the industry level with a group called TransCelerate, and I know T.J. is involved too, to try and establish common procedures as you establish trial sites, as you have communication, as you have training, right? So, that hopefully all boats will rise, right?

Andy Lee:

That’s correct. TransCelerate is a group that formed about eight or nine years ago. There were 10 initial member companies. I was a founder member of that. And we got together to say, “We have to improve operational efficiency.” So, we do not collaborate on molecular structures and those types—that’s competitive. We collaborate on what we call precompetitive, procompetitive aspects which says, “If we all work together to improve something, we’ll all get the benefit of this.” And we share it publicly.

There’s a website, you can look at it. But we’ve looked at standardizing protocols. We have a common protocol template. We’ve adopted that at our companies, so have other sponsors. The protocol can be developed in a standardized way. We’ve looked at standardizing ways where we can improve monitoring. We’re looking now at ways that we can work with investigative sites through i-platforms, shared investigative platforms. So, a clinical trial site has to provide the information for us as a sponsor and use the exact same standardized questionnaire and information for any other sponsor through a standard portal.

So, we are trying to reduce the burden on clinical trial sites. And we’ve plugged away for many years, and we are seeing greater traction there. We are seeing more efficiency, more standardization. We are seeing greater quality, less rework. And so, while it’s hard to quantify this, what we believe is that the sites are freed up of some of the more burdensome things, and they can direct their attention towards patients, patient safety, and access to clinical trials. So, the work may not be directly related to access for a cancer patient into a cancer trial, but there’s a lot of tangential spin-off of making a site more efficient so they can put their resources and energy in the right place.

Andrew Schorr:

Well, thank you for that effort and your leadership. So, Ken, you’ve been around this a long time. And you’ve deal with all the companies and the government and the various agencies. And as you know, in some quarters there’s a distrust for pharma. We mentioned cancer that you get the price tag of a drug, and it’s very expensive. And some people are struggling to pay for it. And there’s just frustration about it. And often in the news media they are the bad guys who are called out for unethical procedure or something that went awry.

So, how are we doing there in overcoming that because we talk to Andy, he seems very ethical, dedicated guy representing a company that’s been around I think well over 100 years. So, how are we doing to move clinical trials on in this area when people aren’t sure what to make of pharma.

Ken Getz:

Yeah. It’s a huge issue, Andrew. And I think part of the challenge is that all it takes is one questionable behavior, and it makes it difficult for the reputation of the entire industry. Right now, we are dealing with major pharma companies that are actually being fined for having contributed—a judgement, having contributed to the opioid crisis. And when you start looking at some companies aggressive marketing tactics, right? It really sort of sheds a darker light on a lot of the great work that companies do.

What we look at, at the Tufts Center for the Study of Drug Development at the School of Medicine. We look at the overall output, the level of innovation that’s coming from the industry today. And we look at the number of complaints that have been filed with the FDA and other regulatory agencies around the world. And what we see is tremendous growth in the innovation and the quality of the innovation—drugs like Keytruda and other cancer immunotherapies. What an exciting area.

We see that the vast majority of companies really support and live by highly ethical, highly professional, highly compassionate approaches because they all know that it takes just one questionable issue that can really tarnish the reputation of every company operating in the industry. So—again, Andy also mentioned just how regulated we are as an industry, the fact that we have ethical review committees and data safety monitoring boards and so many other external agencies that help to oversee the work that’s done here.

So, I would say for patients who are thinking about clinical trials, it’s good to know the history. It’s good to know what you need to do to protect yourself. But the vast majority find that the people they deal with are ethical, they are professional, they are compassionate. And, as I mentioned, over 90% of people who get involved in trials say that they would do it again.

Andrew Schorr:

Thank you. That was a wonderful response. Andy, you mentioned earlier about starting research with the sickest people basically, where there are no options. But one of the questions that came in is, “Are trials only for the sickest people or are there of all those trials you talked about opportunities for people who maybe are newly diagnosed or could be their fairly initial therapy?

Andy Lee:

Yeah, great question. And thank you to the person who asked that. And the answer is that we start in people—because we don’t know if our experimental agent will work. And everyone assumes that new medicines are all going to succeed. And we work in research and researcher because of that many things fail very early on. They fail in phase one before anyone hears of it. It’s normally a code number at that point. And we may just not make the drug soluble enough, or it may not be distributed enough.

So, we may have a thing that works in a test tube or a petri dish. But to get that into humans and make sure that it’s safe at the dosage we use often fails. We just don’t progress far enough. So, what we want to make sure of is that firstly the drugs are safe. And there’s a trade-off between safety and efficacy. We’re constantly trading off. And so, what we do is we look at that and say when someone has no option and we want to get an option going, that’s where we start.

We’ve actually moved down the disease scale, and we’ve come into adjuvant treatment or secondary prevention. And we’ve gone into newer adjuvant is when you have a small tumor is we pre-treat to manage that tumor before surgery is done. And post-surgery we hope that there’s limited treatment or no treatment. And we actually have removed the cancer, and there would be no evidence of disease. But. of course, using the word cured is something we try not to do, because we prefer to use no evidence of disease.

But absolutely. And the next strategy is prevention of cancer. Our company does a lot of vaccinations in women’s health. We have a product that protects against human papilloma virus which is a precursor for cervical cancer. So, people who are vaccinated with this particular product—and I’m deliberately not using brand names for obvious reasons. But when you vaccinate for HPV, you essentially are preventing the likelihood of a cervical cancer. And there are now prospects in many disease areas where either vaccination or early treatment gives you a tremendous positive prognosis of not getting the disease later on in life.

The answer to your question is yes, we are absolutely looking at ways to prevent getting to a very advanced stage which is very costly to manage and very emotional and stressful and difficult.

Andrew Schorr:

I want to thank you. I just want to get a final comment on what you would say to patients or family member. And I want to start with you, Ken. What do you want patients right now to know so that—what tips would you give them so that they’d consider being part of clinical research or stay in clinical research and the benefit it could be for them.

Ken Getz:

I will say really two things. The first is there’s just a tremendous amount of information out there, and we recommend education before participation. So, do your homework and engage family and friends and people you meet and trust to help you make the decision. And the second point comes off of that. And that is this is not a decision you make alone. Really bring in your treating physician, your nurse. Bring in your support network. And chances are you will learn a lot, and you might even find a trial that is right for you.

Andrew Schorr:

Right. And Andy, what about you? A final point—what would you say to a friend or family member or colleague related to considering trials today.

Andy Lee:

We get this question every single day. And we get it from patients in need. And my answer is we are all patients. We are all going to face this as professionals in our job or professionals outside. And so, I say community of practice. And disease hits all of levels of society in all education professions, et cetera. And so, my thing is to encourage people to do what Ken has said. Work as a team. Get multiple inputs.

And I am sponsor agnostic. Get the best therapy that is available. And that may be the best care option—as I said, the ecosystem in which you get the care is really important as well as the medicines that you get. So, have the discussion. Trust the medical professionals, they are very skilled out there. They are extremely well educated. And I just urge people, “Don’t think on two clicks on Google you are going to solve what your treatment option is.” Really discuss it with people because not all the options are public, and there is not enough information available about how to manage the whole disease through the entire enterprise. Trust the professionals.

Andrew Schorr:

Well said. And T.J., you and I are alive today because of trials. What do you want—what’s the thing you want to leave our viewers with?

T.J. Sharpe:

That they don’t have to be involved in clinical research. I think that’s an important distinction to make. And it’s going to pull together what Andy and Ken said that clinical research should not be considered a hail mary or last gasp option. If you are a patient—and we are all going to be patients as Andy mentioned. You want the best care for you. You want to be able to weigh all of your options. And if you are not considering clinical research, if you don’t know about it or aren’t able to get the information you need about it, then you are not going to be able to make the best healthcare decision long term for your health.

So, take that information that you can get. Find the trusted sources. Be able to reach out to advocates or colleagues or someone that you know that would have the disease or can connect you with good information. And be your own advocate—a little cliché, but really own that healthcare information. And once you are able to collect all of the different treatment options, then you consult with your professional medical team as to what the plan forward—the best plan forward for your individual situation would be.

Andrew Schorr:

Right. T.J., my friend, thank you. It’s a delight to see you again. Andy, with Merck, thank you so much for being with us and bringing your years of expertise. And, Ken, being at an independent non-profit center and also at Tufts University there, thank you for all the work you do. I want to thank the Patient Empowerment Network for pulling this all together. And the sponsors who supported us in this effort, AbbVie Inc., Celgene Corporation. Daiichi Sankyo and Novartis.

All these companies and I’m sure many more, working so that research can move forward. We can be true partners in it. And hopefully get tomorrow’s medicine today to make a difference for the community and live a long life, and hopefully a cure, right? I’m Andrew Schorr in California. Remember, knowledge can be the best medicine of all.

Please remember the opinions expressed on Patient Empowerment Network (PEN) are not necessarily the views of our sponsors, contributors, partners or PEN. Our discussions are not a substitute for seeking medical advice or care from your own doctor. That’s how you’ll get care that’s most appropriate for you.

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