2022 ASH Meeting | Multiple Myeloma Takeaways

This is my 17th year attending ASH (American Society of Hematology), where typically over 30,000 attendees from all over the world (hematologists/oncologists, lab researchers, oncology nurses, scientists and 300 pharma companies) attend. This year ASH was set up as a hybrid meeting where some attended in person and many, including myself, virtually. I’m grateful to the IMF (www.myeloma.org) and their sponsoring pharma donors Takeda, Amgen, and Karyopharm for registering me for ASH so that I could learn and subsequently share my patient perspective with you.

My Takeaways

This year’s ASH continued to expand our knowledge on immunotherapies…more CAR-T’s and bispecific antibodies (“T-cell directing therapies”)…as well as more targets besides BCMA…and most importantly, side effects such as cytopenia (lower blood counts), cytokine release syndrome (CRS), neurotoxicity, and infections.  At present, approved treatments in the area include CAR-T’s Abeca and Carvyti as well as the bispecific Tecvayli (Teclistamab), but these are currently only available for patients relapsed-refractory patients with >=4 lines of previous therapy.  The good news is that all of these CAR-Ts and bispecifics are in clinical trials for patients with fewer prior treatments, even newly diagnosed patients in some cases!

Another area that needs better treatment options are Multiple Myeloma (MM) patients considered High Risk (HR) or ultra-high risk (>1 HR factor), as well as High Risk Smoldering Myeloma (HR SMM). Whereas some current studies show that media Overall Survival for MM is 10 years, HR patients are typically half that.  And for HR SMM patients who have a good chance to progress to full blown MM within 2 years, is it possible that treatment at this pre-MM stage could delay progression or actually cure a patient from getting MM.

We know that if we achieve a Complete Response via blood tests which show no sign of an M-spike, that unfortunately the myeloma will still likely return, indicating that we still have myeloma but these tests are not sensitive enough to see it. Tests with more sensitivity are referred to as MRD (Minimal/Measurable Residual Disease) tests (Next Generation Sequencing and Next Generation Flow) from bone marrow biopsies and Mass Spectrometry tested via a patient’s blood. They are good prognosticators but typically not used to help guide treatment (for example, when to stop maintenance). If we knew when to stop treatment or change treatment, patients would more likely do better.

This leads to the discussion that we have many treatments available these days but what’s the best treatment for a patient being newly diagnosed, transplant-eligible or not, maintenance (for how long), treatment at first relapse, subsequent relapses? Many of the study results from ASH try to answer these questions via clinical trial results (but that’s still not a personalized treatment so it’s always important to ask your doctor questions and be part of that shared decision making).

Finally, the important topic of Diversity, Equity and Inclusion (DEI) was discussed more at this ASH than ever before and got its own Spotlight Education session. We need better representation of underserved populations in clinical trials. For example, 20% of MM patients are Black and yet they represent <5% of patients in MM trials. If we don’t improve upon this, trial results may lack internal validity resulting in poor external validity for the populations they are meant to serve.

For more patient information about ASH, there are many excellent webinars coming up from your favorite myeloma advocacy organization. And another great source are blogs written by patients (including myself) which you’ll find on the IMF website (https://ash2022blogs.myeloma.org/).

In summary, this year’s ASH continued to amaze me with so many studies in Myeloma, focusing on all stages from Smoldering Myeloma to MM Induction through Relapse. Clearly immunotherapy treatments, CAR-T’s and Bi-specific T-cell engagers were predominant among the oral presentations I attended, providing longer-term data on these new treatments. And importantly, other targets besides BCMA are being investigated.

For someone diagnosed with stage III MM 28 years ago with only 2 treatment options available (MP or VAD-SCT) and given 2-3 years expected survival, I’ve seen incredible progress since 2003 when Velcade was first approved followed by 14 more approvals and many combination therapies. While there continues to be unanswered questions, we now have many more effective treatments for MM, providing patients with better opportunities to manage their disease. Newly diagnosed MM patients can justifiably be more optimistic about their new diagnosis than at any other time in history. ASH2022 highlighted the tremendous advances we have made in treating this cancer for both the newly diagnosed and relapsed patient.

Myeloma Patient Profile: Sharing My Cancer Journey with My Daughter

Part 1

Myeloma Patient Profile: Sharing My Cancer Journey with My Daughter Part I from Patient Empowerment Network on Vimeo.

In this part one of three, Lori Sackett shares the journey of her multiple myeloma. She explains some of the symptoms she was facing before diagnosis to having to advocate to receive next-generation sequencing testing.

Part 2

Myeloma Patient Profile: Sharing My Cancer Journey with My Daughter Part II from Patient Empowerment Network on Vimeo.

 In this segment of Lori’s story, Lori and her daughter discuss the importance of seeing a myeloma specialist, having a good support network, and the role her daughter played in Lori’s care.

Part 3

Myeloma Patient Profile: Sharing My Cancer Journey with My Daughter Part III from Patient Empowerment Network on Vimeo.

Lori and her daughter share their biggest takeaways and pieces of advice for other newly diagnosed myeloma patients and their care parters/advocates.

Myeloma patient, Lori’s advice:

  1. Insist on seeing a myeloma specialist
  2. Take care of yourself physically and emotionally
  3. Look for people/support and allow them to help you
  4. Live for now

Myeloma care partner and advocate, Carleigh’s advice:

  1. During every appointment have at least one note taker
  2. Ask for a hard copy or print out of everything
  3. Create a way to stay organized
  4. Keep a list of questions
  5. Have a mindset of persistence and perseverance, and to maintain hope

Myeloma Patient Profile: Jeff Boero

When Jeff Boero shares his multiple myeloma patient journey, it’s clear that self-education has been a vital part of his experience. He was first diagnosed through his primary care physician who referred him to a general oncology group in the San Francisco area. They confirmed it was multiple myeloma. It soon became clear to Jeff and his wife that he perhaps needed a second opinion, and he was connected with the University of California San Francisco (UCSF) to their multiple myeloma specialist. 

The second opinion changed the approach to Jeff’s care rather dramatically. He was quickly scheduled for a stem cell transplant and subsequent maintenance after that. As Jeff recalls, “Through UCSF, I became eligible for a CAR T-cell immunotherapy trial in 2017. That was very successful and kept me disease-free and medication-free for about 2-1/2 years. And then I relapsed and went on another maintenance program. I became eligible for another clinical trial for a bi-specific T-cell engager (BiTE) that I’m on now and am having good results.”

Jeff was almost in complete denial about his diagnosis for the first 6 months. The diagnosis threw him into a world of terminology and treatment that was completely foreign to him. That sense of his diagnosis feeling foreign also started to lead into a certain level of depression — just not knowing what it is, how is it going to be treated, what it meant to his long-term survival. Jeff remembers, “So, with the encouragement of my wife as caregiver, I became more educated as I engaged in various conversations with specialists and participated in some of the PEN webinars. It  became clearer to me about what some of the options are and what they can be. Being engaged with UCSF really opened up the treatment options. With me becoming more educated and able to speak the language of myeloma, I was starting to understand the diagnosis as it was presented by UCSF. And it led to a much richer engagement in conversation with the oncologist and with the nurse practitioners.” 

As a cancer patient, Jeff views self-education as the key to empowering patients toward better care. It was through self-education that he learned about other options. Before becoming more educated, Jeff was mostly just listening and trying to absorb as much as he could and seemed to remember mostly bad news. According to Jeff, “There’s so much good news around myeloma treatment and available therapies. It was through self-educating and those conversations that my outlook brightened too.”

By patients educating themselves, they can start to ask questions about the clinical trial like: “What is it, and why is it going to show better results than my maintenance therapy?” And in conversation, patients can start to better understand the purpose of the clinical trial. “I think it’s important for patients to understand what they’re trying to accomplish through the clinical trial that wasn’t through their maintenance therapy. What is it about this trial that’s different that we haven’t addressed previously?” But patients can’t ask those questions unless they have at least a basic understanding of their cancer and how the various therapies approach the cancer cell. “But if you listen to webinars and things like that, you’re better able to have those conversations. As a matter of education as these opportunities arise, you’re able to have a much richer conversation with your oncologist and your care team about the benefits that could potentially be derived from the clinical trial.” 

Clinical trials have benefitted Jeff, and he recommends seeking an opinion that is dedicated to research of your specific cancer. Learning institutions have more access to emerging research and treatments that likely won’t be FDA-approved until 2 or 3 years later. “So if you as a patient can be at the forefront of some of these trials, that can be tremendous. I’m on therapies now that didn’t even exist when I was diagnosed. Research is moving quickly.”

Jeff senses some hesitancy among patients about clinical trials. “There’s this misconception that if you join a clinical trial, one group is getting the real stuff, and one group is getting the placebo. And the trials that I’ve been in, everybody gets the real thing, and everybody’s progress is tracked on their response to the real thing.” He knows trials can seem intimidating. Jeff went through his initial clinical trial, because he was almost out of options for conventional maintenance therapy. His cancer burden continued to increase, and he’d been through a number of different treatments. “The CAR T-cell program came up and seemed to be a perfect fit for me. So I did the clinical trial partially out of necessity, but I also had extreme confidence in my oncologist that he was promoting something that he thought would be most beneficial for me. I think it’s a matter of putting trust in your oncologist. Maybe I’ve been lucky, but I’ve had good results and good response to both clinical trials.” He also feels that the sponsoring institution will give an honest appraisal of where the program stands and what the progress and success has been up to that point. 

Reflecting on the value of Patient Empowerment Network (PEN) and other resources, Jeff says, “I’ve gotten so much out of the PEN webinars that are provided and some other organizations. I’m a slow learner in this area but am absorbing as much as I can. I need to hear the same thing a few times before I start to absorb it and fully understand it. So I rewatch the PEN webinars, and it works for me.” He also suggests learning as much as one can but was advised early on to stay away from Google. “There’s so much out-of-date information. Whereas websites like Patient Empowerment Network’s and others have updated information that’s far more relevant. And I also find the navigation on the PEN website very easy to use.”

After meeting patients who don’t have the same level of health insurance benefits, Jeff feels a sense of gratitude. “I had tremendous support from my employer who in essence said take the time you need to get yourself well again. So I have a lot of gratitude for that support, my wife as caregiver, family, social support, my faith community, and for my proximity to UCSF that makes treatment very practical and very possible.” It’s opened his eyes in that regard. There are so many benefits that he has that others don’t have. “I’ve joined various support groups initially to gain support. Now things have come full circle, and I find that I’m at the other end of the conversation to give people comfort in what they could possibly be doing to improve their situation.”


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Is It Too Late for a Myeloma Second Opinion?

Sujata Dutta: Sharing the Journey

Check out Part I of Sujata’s story: Normalizing the Word Cancer


 

Sujata Dutta, Part 2 Sharing the Journey from Patient Empowerment Network on Vimeo.

Empowered multiple myeloma patient, Sujata Dutta, shares an overview of her treatment from a stem-cell transplant to a clinical trial, and how she chooses to see the positives in her journey.


Transcript:

So once I was diagnosed with multiple myeloma and I was actually informed about the standard of care. So standard of care with multiple myeloma today is typically a couple of cycles of chemo. So I had about five or six cycles of chemo to bring the M-spike to as low as you can, and then that’s followed with like a stem-cell transplant (an SCT) or bone marrow transplant – both are the same. In my case, it was an autologous stem-cell transplant which meant that I use my own stem cells which were extracted and stored and then given back to me.

 So then post-transplant, if the counts look good then you go into a maintenance routine. So I didn’t have succession of chemo before the stem-cell transplant. I had my stem-cell transplant at Mayo in Rochester, Minnesota and unfortunately, in my case, we did not achieve the results that we were expecting so my disease actually did actually not come down as much as we would have hoped. 

So, I had to go back on a chemo routine and I’m on that one right now. However, I actually am part of a clinical trial. I signed up to be part of a clinical trial that’s looking for newer ways of treatment which are shortening the time of treatment and also with the goal of improving the standard of you know care or like better lifestyle for the patients and like obviously longer life.

So, I’m part of a clinical trial that’s combining Revlimid and Daratumumab, which is like usually you would have an 8-hour hospital visit for the chemo, but in this I am just getting a subcutaneous injection in my belly. It’s a 5-minute injection so that’s not pleasant, but 8 hours compared to 5 minutes, it’s great.

So yes, I am back on chemo just so that we can bring the disease under control. But typically with standard of care with multiple myeloma is like couple cycles of chemo followed by a transplant. If you are eligible for one, and if you are ready for one, and then followed by maintenance. So that’s typically what happens with multiple myeloma.

But there are loads of other treatments that are coming up and researches that are happening, clinical trials that are happening, I would highly encourage it if you come across a clinical trial that interests you, speak to your doctors and see what they say. And if you’re eligible, it would be a great thing to do. I personally wanted to get involved in some kind of volunteering activity. I know that folks before me have done so much and I’m benefiting from that, I wanted to give back as well so I actually signed up for the trial. But other than that, that’s pretty much what the standard of care is today for multiple myeloma or what I know of.

I think one of the biggest takeaways from my cancer journey, I would say is learning to be appreciative of what I have. Learning the value of what I have, not that I did not know that, but I think this life changing kind of event that has happened has taught me even more of the value. For myself, what’s my worth? What’s the worth of somebody else in my life? What’s the worth of things around me in my life? And it has, so my journey has actually helped me understand these things and be appreciative of what I have. 

My husband he’s been my primary caregiver throughout this journey and we have actually like been on the journey together, so it has been an amazing journey I would say. 

We have discovered like a new relationship between us, like going for chemo, going to Mayo for 6 weeks, and we stay together and you know how much I appreciate what he has had to go through because of me. Like looking at me not being able to walk or not even being able to talk or even drink water because of the amounts of … that I had and supporting me through all of that. I really appreciated it. I appreciated my boys, like I have a 7th and a 6th grader, and for them to understand what I was going through and for them to be able to accept in the form that I was, has been great.

I have friends, I have family who have supported me throughout this so I really appreciate them being with me, being around me, supporting me, rooting for me, praying. There’s one thing that I tell everybody like you know there have been so many people known and unknown that have like you know helped me or prayed for me or rooted for me that I have no choice but to get better.

So you know I really appreciate what I have and I think I also appreciate the value of what I have, and like not think about what I don’t have. I am a believer that divine intervention happens, you don’t know why but everything has a reason and I think whatever happens, happens for the best. For even cancer, I think happens for the best.

For me to understand like what all I had and like how grateful I was for everything that I had. For me to go back to a hobby that I had almost forgotten. I paint, I used to paint and I’d almost given up on that through my journey. I was like I need to go back and do something else and I went back to painting. So like so many good things have come out of this, so you know I’m really grateful for whatever has happened and I’m quite positive for the future so I am looking forward to what’s in store for the future and I’m going to be positive keeping my fingers crossed. That’s my story for you.

A New Phase: Bruce Jackson

Bruce Jackson is a multiple myeloma patient who recently found Patient Empowerment Network (PEN) as a resource for his cancer journey. This is the first of two-part series in which he shares his story from diagnosis to living his life with cancer.


“You can do nothing, or you can do something…maybe it is simply advocating for yourself or advocating on behalf of someone else.”

I guess I haven’t thought of my cancer experience as a story, and yet, that is exactly what it is: a story about a new phase in my life. I have multiple myeloma. More specifically, it is a t(4-14) translocation wherein the 4th and 14th chromosome pairs, instead of minding their own respective business, decided to share their genetic information, and that sharing process is at the basis of the disease. I don’t know if researchers yet know the cause of these translocations; some say that they result from a virus, but I know very little more than that. My 4-14 translocation is deemed a moderately aggressive cancer, but there are other much more aggressive translocations which are functionally a one-year death sentence.

I was diagnosed in May 2009. I was 53 at the time and am now 64. In my case, I was seeing my primary care physician (PCP) every six months for treatment of high cholesterol. She was treating me with a statin drug, and she insisted on doing blood work every six months. The blood work revealed an elevated total protein level, and my PCP suspected cancer, so she sent me to an oncologist who confirmed the diagnosis of smoldering myeloma.

I think there are a couple of points to be made here. One, because of the blood panels every six months, my cancer was caught early. Two, while a smoldering myeloma diagnosis may seem relatively benign, it is not. The question is, when does it morph into something else, into what does it morph, and what do you do in the meantime?

For me, this meant tracking the disease through occasional (every six months) to more frequent (every three months) blood tests to track my M protein value, which is a pretty highly correlated indicator of what is happening in the bone marrow. On a lesser frequency, I would have a bone marrow biopsy, just to see whether what was happening in my blood stream still continued to correlate with what was happening in my bone marrow. When my M protein value was around 0.8, I started to see an oncologist regarding what was initially diagnosed as monoclonal gammopathy of otherwise unspecified origin (MGUS). Then in October 2014, my oncologist was citing M protein values of 3.6, but with no other symptomatic phenomena to address, except that an MRI had shown some very small unidentifiable spots on a few of my ribs and on my sternum. The MRI report suggested that I have a re-do in six months, and that is what happened, except I was now in the hands of a myeloma specialist, and she suggested that we re-test using a CT Scan. The scan revealed growth in the spots, enough so that we were now using the term “lesions”, which was the tipping point to starting treatment.

I started my treatment program as a part of a Dana Farber Cancer Institute study, which required a prescribed regimen of Velkade (a subcutaneous injection), coupled with Revlimid (Thalidomide derivative and sister drug to Pomalyst), and Dexamethasone (a common oral steroid, which generates a synergistic effect that aids in combatting the cancer). In my first cycle, the treatment knocked my M protein value down to less than 1.0. However, in the second round, the treatment induced some unplanned side effects, all at the same time. I experienced blood clots in my lower legs, an obstruction in my digestive tract, pulmonary emboli in my lungs, a half-collapsed lung, a respiratory infection, and a massive headache. This earned me a 10-day stint in the hospital, a paranoid reaction to one of the drugs that I was given, and removal from the Dana Farber study.

Unfortunately, the respiratory infection would not go away, and only six weeks later, it was determined that I needed to have a procedure done, wherein the surgeon puts three holes through my rib cage and inside my pleural cavity with the goal of removing scar tissue from the surface of my right lung so that the medication could reach and eliminate the infection. The procedure earned me 12 more days in the hospital.

The good news is I made it through both events, and I am here to share about it!

It was determined that the Dana Farber dosage was too much for my system, so the solution was to cut the dosage back to about two thirds, and then administer more rounds. My rounds of chemo ultimately led to a stem cell transplant in September 2015. The stem cell transplant was a 21-day hospital stint (which is a typical duration), but as can happen, things didn’t automatically jump-start as expected. After my transplant, everything was jump-starting except my platelets. Fortunately, it seems there is always an alternate plan of attack, and the hematologists were able to prescribe a three-day dose of medication that on day three bumped my platelet count from two to four, and I was on my way. Plan B worked, and I’m glad we did not have to go to Plan C, because I don’t know if there was a Plan C. There were other hiccups along the way. I started having blood clots in my lower legs again, and developed pre-ventricular contractions (PVCs), which feel like a skipped beat, but are actually extra beats, and amount to an arrhythmia of the heart.

After my stem cell transplant, I was given a prognosis of four to eight years, and I was only in partial remission. Once sufficiently recuperated, I had to take Velkade as chemo maintenance. However, because of the subsequent neuropathy, and associated deep venous thrombosis (DVT) in my lower legs, the decision after about two years was to switch to Revlimid. However, the truth of the matter is, your M protein does not stop increasing with the chemo maintenance. It simply increases at a slower rate, and if the drug stops working, problems arise. In my case, the Revlimid worked for another two years, but then things started to happen in 2020.

When the medication stops working, the problems that arise are one of two things: either the rate at which the M protein increases starts to accelerate, or your immune system loses the ability to adequately recover during the seven-day rest period. Your neutrophil (white blood cells) count drops due to the chemo, but if the counts do not climb back up, that means you have to take more days to recover, lower the chemo dosage, or get a booster shot to bump your neutrophils. Any of these options would, of course, allow the cancer to progress at a faster rate. In my case, the neutrophils were dropping and my M protein was climbing, which in essence means the chemo drug was no longer effectively slowing the progression of the disease. It was time to switch to another treatment.

I was given the option to investigate my choices, but because of the myriad options available, that turned into a whole bunch of, “I don’t know”. I finally settled on Daratumumab, Pomalyst and Dexamethasone, with Dara being subcutaneously injected (like Velkade was). Pomalyst is an oral Thalidomide-based sister drug of Revlimid, and Dex is well, Dex. Given that I am only just starting a third post-transplant treatment, I think I am doing well, especially if you consider that I am mid-way through my 12th year post-diagnosis and I am more than five years post-transplant that had an original prognosis of four to eight years.

When you consider where I have been, five years is good so far. I have not had any bones break, my cancer was caught early thanks to a competent PCP, I have only a moderately aggressive translocation, which is much better than more highly aggressive versions, which could have buried me in short order. But what bothers me most, regardless of all the other things that have happened during this experience, is the uncertainty of it all. I feel like I am always waiting for the other shoe to drop.

Learn the rest of Bruce’s story in part two of the two-part series in which he shares his story from diagnosis to living his life with cancer.


Read more patient stories here.

The Warrior in Me Saved My Life

After experiencing increasing fatigue over the course of several years, I started to miss gatherings with friends and family and got to the point of taking one day off a month from work to sleep all day. This was unlike me as I was always very involved with professional and volunteer activities and had a very full schedule including parenting my young son with my spouse. After a lingering cold evolved into bronchitis, I began to explore what was wrong with the assistance of my primary care provider (PCP). I had mild persistent anemia, but nothing to warrant the degree of extreme fatigue that I was experiencing. She (my PCP) was very tolerant of my various Google-induced ideas, graciously accepting some to follow up with tests and others to set aside. 

After eleven months, a test showed that I had elevated M-proteins and my PCP sent me to a hematologist/oncologist who after greeting me reviewed several years’ worth of labs and then turned to tell me to come back in six months. She did not examine me. She did not ask me about my symptoms. She prepared to usher exit the room. I felt that I could not leave her office without her understanding how significantly the fatigue was impacting my daily life. This is when the inner warrior in me said NO! I did not move from my chair. I told her, “Nope. Now is the time that I need to tell you about my symptoms.” (Now this was somewhat uncomfortable for me because I have been well-trained to be polite and professional with doctors, but I had had enough. My New York elbows were coming out!) 

I read from a list that I had prepared detailing what I had been able to do prior to feeling unwell and what I could do now. As I went down the list for several minutes, she looked at her watch in a disgruntled manner, finally asking me “What do you want?” I told her that I wanted to feel well. I did not feel well and believed that something was wrong. I wanted her to do more tests. She agreed and also sent me out to schedule an appointment in six months. One week later at 8 AM as I was on my way out to work, SHE called me to tell me that she had scheduled a bone marrow biopsy (BMB) for the next day. I cleared my calendar. The BMB results confirmed that I had stage 2 Myeloma with more than 80 percent involvement in my bone marrow. My husband and I learned of this on the day before Thanksgiving. We were both in shock. We had so much to learn and at that point had no idea how much this diagnosis was going to change our lives. 

After a quick success of additional tests scheduled STAT, I started chemotherapy within two weeks. Getting a diagnosis took A LOT of persistence and determination when specialists minimized what I knew about my body — that something significant was wrong. And it was. Today is five years to the day of that diagnosis and I still wonder whether I would be diagnosed today if I had not INSISTED upon further testing. To her credit, the oncologist/hematologist did eventually acknowledge that I was right to press her to do more tests and that it was through my self-advocacy that I achieved a diagnosis.

What I would hope that others would take away from this story is how essential it is to be aware of your own body and to keep advocating (again and again) for yourself with doctors even when your symptoms are minimized. I was trained to advocate for others as a social worker, but it took intentional work to give myself permission to say no to doctors at first politely and then later not so politely to demand additional testing until an outcome was achieved that explained my health issues. Be persistent. You know more about your symptoms than anyone else. Do not stop until you find out what is going on with your body.

Checking the Pulse on Multiple Myeloma Health Disparities

Even before the coronavirus pandemic arrived, health and patient support organizations made resolute efforts to examine and address health inequities for multiple myeloma patients in Black, Indigenous, and People of Color (BIPOC) communities. Diverse Health Hub and the Patient Empowerment Network partnered to help improve health outcomes for underserved myeloma patients through the Diverse Partners in Your Myeloma Care program. With a tumultuous year filled with the killing of George Floyd, social unrest, and coronavirus health disparities for BIPOC groups, these issues prompted us to focus on where things stand with multiple myeloma health disparities. We’ll take a look at what we know, what we’ve learned, and what help and resources are needed to continue advancing care for BIPOC myeloma patients.

Disparity Facts About BIPOC Myeloma Patients

  • Both Black Americans and Latina and Latino Americans show a myeloma precursor called MGUS, or monoclonal gammopathy of undetermined significance, more frequently than others—.88 percent in Black Americans, .44 percent in Latina and Latino Americans, and .22 percent in white Americans.

  • Although multiple myeloma is diagnosed at a younger age in both Black Americans and Latina and Latino Americans, both groups are less likely to receive a transplant and start treatment later than patients of other races.

  • Black Americans are actually known to have less aggressive myeloma, which should show better health outcomes—yet that is not the case.

multiple myeloma diagnosis.png

Learnings About BIPOC Myeloma Patients

Black and other BIPOC patients often have mistrust of doctors and researchers due to past experiments like the Tuskegee Study and Henrietta Lacks – whose now infamous immortal HeLa cells were taken without her consent. “If I were to walk into any community, African American community, or underserved community, that is one of the first things. They’re going to be mistrustful of me. And it’s a very difficult barrier to overcome. And that also leads over into African Americans contributing, being donors, African Americans participating in trials. It all feeds over into everything that’s done in the African American community or underserved community in regards to healthcare,” says patient navigator Diahanna Vallentine.

Barriers to care must be overcome according to Dr. Sikander Ailawadhi from the Mayo Clinic, “Myeloma patients who are African-American and Hispanic typically get to the right treatment much later. In a lot of cases they may not get to the right treatment at all. We also know that the burden of cost of care is much higher for minority patients.”

Improvements are happening in care as explained by Dr. Ajay Nooka from Emory University School of Medicine, “What’s really interesting in this meeting is that there has been a lot of large database integrations, including one database called the National Cancer Database (NCBD) where people have looked at 20-year history of how these treatments have panned out. Which of the minority populations or which subset of patients gained the most benefit over the last 20 years? And we see minorities have gotten a lot of improvement and a lot of access to care over the last 20 years, but that’s not the end of the story, we have to catch up a lot more.”

The Path to Health Equity

Although the additional focus on health inequities has started to improve access to care, there is still a critical need to raise awareness about the treatment gaps for myeloma patients in BIPOC populations. How can myeloma patients get the best care no matter where they live when factors like age, geography, socioeconomic status, ethnicity, gender, and insurance type heavily influence the path to better health outcomes?

Some valuable steps that patients, community leaders, and healthcare providers can take to improve care include:

  • Support organizations providing educational materials to patients that are target specific BIPOC groups

  • Patients and advocates making the BIPOC voice heard by asking for funds from community and political leaders to improve care

  • Healthcare providers developing relationships and partnerships with political leaders and support organizations to continue building momentum in improving patient care

  • Patients taking advantage of social workers and patient navigators at their clinics and support organizations

  • Patients, advocates, and healthcare providers working to increase clinical trial participation

  • Healthcare providers integrating cultural competency as a universal approach in the healthcare model

Resources like myeloma patient resource guides, informational graphics, and the Myeloma Coach section on the Myeloma Crowd website provide valuable information for patients. And though trust of clinical trials by BIPOC populations remains an issue, there are initiatives like Diversity in Clinical Trials Benefits Everyone. BIPOC patients can take action working together with medical researchers to increase clinical trial participation to improve and refine myeloma treatment developments for specific patient populations. If you want to explore options in your treatment, seek out resources that embrace diversity in clinical trials. The “All of Us” program is a public health initiative designed to remove the barriers that prevent inclusive access.

Participating in clinical trials not only will improve myeloma treatments down the line but also provides a minimum of standard of care treatment at no cost to the patient. It’s a win-win for both the patient who participates in the study and  also helps the progression of treatment for BIPOC patients diagnosed with myeloma in the future. Though progress has been made, patients, advocates, community leaders, and healthcare providers must take action to continue an upward movement to achieve equitable care that BIPOC myeloma patients deserve. Take advantage of the resources below and continue to visit our Multiple Myeloma Hub as we publish more on health equity developments for multiple myeloma.

Resources to Learn About Improving Myeloma Health Disparities

Disparities Around Health Technology Access for Subset of Myeloma Patients

Good News for Myeloma Treatment Today – Still Addressing Race-Associated Risks

2020 Shaping Up to Be a Big Year for Multiple Myeloma Treatment

How Can a Myeloma Patient Advocate/Financial Advisor Help

Is It Possible to Achieve Health Equity in Multiple Myeloma?

Are Myeloma Clinical Trials More Critical for African Americans?

A Multiple Myeloma Advocate’s Uphill Battle to Care

What Do Disparities in Multiple Myeloma Look Like?

How a Second Opinion Saved a Myeloma Patient’s Life 

Myths vs. Facts: Myeloma Health Disparities Care Infographic

How Can I Get the Best Multiple Myeloma Care No Matter Where I Live? Resource Guide

Diversity in Clinical Trials Benefits Everyone

Sources

How Can a Myeloma Patient Advocate/Financial Advisor Help? Patient Empowerment Network website. https://powerfulpatients.org/2020/08/17/how-can-a-myeloma-patient-advocate-financial-advisor-help/ Accessed October 19, 2020.

Confused About Immunotherapy and Its Side Effects? You Aren’t Alone

“You don’t look like you have cancer.”

More than one patient undergoing immunotherapy to treat cancer has reported hearing statements like that. Immunotherapy is one of the recent advances in cancer treatment that belie the stereotypes about the effects of cancer treatment. 

The side effects of immunotherapy are different from those associated with chemotherapy and radiation. However, that does not mean immunotherapy does not have side effects. Patients and care partners need to be aware of these potential side effects and to be vigilant in addressing them with their oncologists because they can signal more serious complications if left untreated.

What is Immunotherapy?

Despite the increase of immunotherapy treatment options in recent years and considerable media attention paid to advancements in this field, there remains confusion about immunotherapy and its side effects. Many cancer patients are unaware of whether immunotherapy treatments are available for their specific diagnosis. Others don’t know that genetic profiling of their tumors is usually required to determine if immunotherapy is an option and not all treatment centers routinely conduct genetic profiles of tumors. A  survey by The Cancer Support Community found that the majority of patients who received immunotherapy knew little to nothing about it prior to treatment and were unfamiliar with what to expect.

Immunotherapy works by manipulating the patient’s immune system to attack cancer cells. It is perceived as gentler and more natural than chemotherapy and radiation, without the same destructive effect on the body’s healthy tissues.  This, combined with a lack of prior understanding of immunotherapy, can lead patients and care partners ill-prepared for possible side effects.

Furthermore, immunotherapy is a category of therapies, not a single type of treatment. There are a variety of immunotherapy drugs, most of which are administered via infusion.  Side effects will vary by drug, the cancer and its location, treatment dose, and the patient’s overall health.

The following are the most common types of immunotherapy.

  • Checkpoint inhibitors use drugs to block proteins in the patient’s immune system that would otherwise restrain the immune system, often referred to as taking the “brakes” off the immune system.
  • CAR-T therapy modifies the patient’s T-cells in a lab to enhance their ability to bind to cancer cells and attack and kill them.
  • Oncolytic virus therapy uses genetically modified viruses to kill cancer cells.
  • Another therapy uses cytokines (small proteins that carry messages between cells) to stimulate the immune cells to attack cancer.

Immunotherapy can be part of combination therapy. It might be combined with chemotherapy. It might be used to shrink a tumor that is then surgically removed.  Or multiple immunotherapy drugs might be used simultaneously.

What Are The Side Effects?

With immunotherapies, side effects typically occur when the immune system gets too revved up from the treatment. The most common side effects for immunotherapy treatments are fatigue, headache, and fever with flu-like symptoms. Some people also experience general inflammation often in the form of a rash. Many melanoma patients report blotchy skin discoloration, called vitiligo, during treatment. These milder side effects can usually be managed with over-the-counter remedies and adjustments to daily activities.

For checkpoint inhibitors, the fastest growing segment of immunotherapy treatments, mild side effects occur in 30% – 50% of patients. Serious side effects typically occur in less than 5% of patients. (See “Understanding Immunotherapy Side Effects” from the National Comprehensive Cancer Network and the American Society of Clinical Oncology.)

Less common side effects are blisters, joint pain, thyroid inflammation, and colitis (inflamed colon resulting in diarrhea with cramping). Some patients who receive CAR T-cell therapy develop a condition known as cytokine release syndrome, which causes fever, elevated heart rate, low blood pressure, and rash. 

In rare cases, immunotherapy has resulted in lung inflammation, hepatitis, inflammation of the pituitary, and detrimental effects on the nervous and endocrine systems. In most cases, the conditions clear up when treatment ends.  However, there have been outcomes in which immunotherapy caused diabetes or tuberculosis.

“Overall there are fewer side effects [with immunotherapy],” explained Dr. Justin Gainor, a lung and esophageal cancer specialist at Mass General during an Immunotherapy Patient Summit hosted by the Cancer Research Institute. “But the immune system can affect anything from the top of the head down to the toes. Any organ has the potential to be affected.”

As the application of immunotherapy has expanded, so has our understanding of the potential side effects. Like most medical treatments, how one person responds to immunotherapy can be different from another even when the cancer diagnosis and drug therapy are the same.

The essential thing patients and care partners need to know about side effects is they should always be reported to their oncologist or nurse oncologist.

Why Patients Should Talk to Their Provider About Immunotherapy Side Effects

Because immunotherapy has created newer therapy options, there isn’t the volume of experiences as with older treatments. The infinite number of variables that patients provide once a treatment moves beyond clinical trials and into the general patient population generate more diverse outcomes.  And, as most therapies are less than 10 years old, there hasn’t been an opportunity to study the long-term effect of these therapies. This is why oncologists advise patients and their caregivers to be extra vigilant in noting any changes experienced during and after treatment.

Many side effects are easy to treat but medical providers want patients to be forthcoming in discussing any and all side effects. This is in part to improve understanding of side effects, but also because a mild cough or a case of diarrhea might be harbingers of a more systemic issue that will grow worse if left untreated.

Patients should not be hesitant to discuss side effects because they fear they will be taken off immunotherapy.  Sometimes a pause in treatment might be necessary, but the earlier the oncologist is made aware of a side effect, the less likely that will be necessary.

In addition, patients undergoing immunotherapy should always take the name(s) of their immunotherapy drugs and the name of their oncologist when seeing medical professionals outside of their cancer treatment team. This is especially important when visiting the ER.  Because immunotherapy drugs are newer and highly targeted to certain cancers, many medical professionals remain unfamiliar with drug interactions and treating related side effects.

Immunotherapy On The Rise

Immunotherapy treatments have resulted in reports of remission in cases that would’ve been deemed hopeless just five or 10 years ago.  The Federal Drug Administration (FDA) has approved various immunotherapy treatments for melanoma, lung cancer, head and neck cancer, bladder cancer, cervical cancer, liver cancer, stomach cancer, lymphoma, breast cancer, and most recently bladder cancer.  (Here is a list of  immunotherapies by cancer type from the Cancer Research Institute.)

“It’s revolutionized how we treat our patients,” says Dr. Gainor of Mass General about immunotherapy’s impact on lung and esophageal cancer.

Advances in immunotherapy research and trials continue to generate optimism and excitement. A clinical study in Houston is looking at using immunotherapy to prevent a recurrence. Researchers in Britain recently announced a discovery that might lead to advances in immunotherapy treatments to a much broader array of cancers.

While there is excitement around the field of immunotherapy and it has resulted in unprecedented success in treating some previously hard-to-treat cancers, it remains an option for a minority of cancer diagnoses.  It works best on solid tumors with more mutations, often referred to as having a high-mutational load or microsatellite instability (MSI) high. And it is not universally successful for every patient.

With hundreds of clinical trials involving immunotherapy alone or in combination with other therapies, it is certain more treatment options are on the horizon. As more therapies are developed and more patients with a greater variety of conditions undergo immunotherapy, we will also increase our understanding of potential side effects.

Side effects should not dissuade patients and care partners from considering immunotherapy if it is available or from advocating for genetic tests to deteimine if it is an option. Many patients undergoing immunotherapy have previously undergone chemotherapy and report that the side effects are fewer and milder by comparison.  The important thing is that patients and their partners know what to expect and communicate with their treatment team.

If the next 10 years in immunotherapy research and development are anything link eth elast 10, we can expect more exciting advancements in the battle against cancer. For more perspective on what’s ahead for immunotherapy see the Cancer Research Institute’s article: Cancer Immunotherapy in 2020 and Beyond.

Understanding Clinical Trials: A Jargon Buster Guide

When it comes to cancer treatment you or a loved one may be considering participating in a clinical trial as a treatment option.  Clinical trials are designed to evaluate the safety and effectiveness of a treatment. They may involve researchers administering drugs, taking blood or tissue samples, or checking the progress of patients as they take a treatment according to a study’s protocol.

Learning about clinical trials can be a steep learning curve – not least because the process comes with a lot of new terms, acronyms and jargon.  To help you, I’ve put together this list of the most common terms you will find when you are researching clinical trial information. This is not an exhaustive list but it is a helpful starting point. At the end of this article you will see links to find more information.

Adverse Effects (AE)

Also called Adverse Events, or Adverse Drug Reaction, AEs are any harmful event experienced by a person while they are having a drug or any other treatment or intervention. In clinical trials, researchers must always report adverse events, regardless of whether or not the event is suspected to be related to or caused by the drug, treatment or intervention.

Arm

Subsection of people within a study who have a particular intervention.

Bias

Bias is an error that distorts the objectivity of a study. It can arise if a researcher doesn’t adhere to rigorous standards in designing the study, selecting the subjects, administering the treatments, analysing the data, or reporting and interpreting the study results. It can also result from circumstances beyond a researcher’s control, as when there is an uneven distribution of some characteristic between groups as a result of randomization.

Blinding

Blinding is a method of controlling for bias in a study by ensuring that those involved are unable to tell if they are in an intervention or control group so they cannot influence the results. In a single-blind study, patients do not know whether they are receiving the active drug or a placebo. In a double-blind study, neither the patients nor the persons administering the treatments know which patients are receiving the active drug. In a triple-blind study, the patients, clinicians/researchers and the persons evaluating the results do not know which treatment patients had. Whenever blinding is used, there will always be a method in which the treatment can be unblinded in the event that information is required for safety.

Comparator

When a treatment for a specific medical condition already exists, it would be unethical to do a randomized controlled trial that would require some participants to be given an ineffective substitute. In this case, new treatments are tested against the best existing treatment, (i.e. a comparator). The comparator can also be no intervention (for example, best supportive care).

Completed

A trial is considered completed when trial participants are no longer being examined or treated (i.e. no longer in follow-up); the database has been ‘locked’ and records have been archived.

Control

A group of people in a study who do not have the intervention or test being studied. Instead, they may have the standard intervention (sometimes called ‘usual care’) or a dummy intervention (placebo). The results for the control group are compared with those for a group having the intervention being tested. The aim is to check for any differences. The people in the control group should be as similar as possible to those in the intervention group, to make it as easy as possible to detect any effects due to the intervention.

Efficacy

How beneficial a treatment is under ideal conditions (for example, in a laboratory), compared with doing nothing or opting for another type of care. A drug passes efficacy trials if it is effective at the dose tested and against the illness for which it is prescribed.

Eligibility Criteria/ Inclusion and Exclusion Criteria

Eligibility criteria ensures patients enrolling in a clinical trial share similar characteristics (e.g. gender, age, medications, disease type and status) so that the results of the study are more likely due to the treatment received rather than other factors.

Follow-up

Observation over a period of time of participants enrolled in a trial to observe changes in health status.

Informed Consent

A process (by means of a written informed consent form) by which a participant voluntarily agrees to take part in a trial, having been informed of the possible benefits, risks and side effects associated with participating in the study.

Intervention

The treatment (e.g., a drug, surgical procedure, or diagnostic test) being researched. The intervention group consists of the study participants that have been randomly assigned to receive the treatment.

Investigator

A person responsible for the conduct of the clinical trial at a trial site. If a trial is conducted by a team of individuals at a trial site, the investigator is the responsible leader of the team and may be called the principal investigator (PI).

Multicentre Trial

A clinical trial conducted according to a single protocol but at more than one site, and therefore, carried out by more than one investigator.

Number needed to treat (NNT)

The average number of patients who need to receive the treatment or other intervention for one of them to get the positive outcome in the time specified.

Outcome Measures

The impact that a test, treatment, or other intervention has on a person, group or population.

Phase I, II, III and IV Studies

Once the safety of a new drug has been demonstrated in tests on animals, it goes through a multi-phase testing process to determine its safety and efficacy in treating human patients. If a drug shows success in one phase, the evaluation moves to the next phase

  • Phase 1 tests a drug on a very small number of healthy volunteers to establish overall safety, identify side effects, and determine the dose levels that are safe and tolerable for humans.
  • Phase II trials test a drug on a small number of people who have the condition the drug is designed to treat. These trials are done to establish what dose range is most effective, and to observe any safety concerns that might arise.
  • Phase III trials test a drug on a large number of people who have the condition the drug is designed to treat. Successful completion of Phase III is the point where the drug is considered ready to be marketed.
  • Phase IV trials can investigate uses of the drug for other conditions, on a broader patient base or for longer term use.

Placebo

A fake (or dummy) treatment given to patients in the control group of a clinical trial.  Placebos are indistinguishable from the actual treatment and used so that the subjects in the control group are unable to tell who is receiving the active drug or treatment. Using placebos prevents bias in judging the effects of the medical intervention being tested.

Population

A group of people with a common link, such as the same medical condition or living in the same area or sharing the same characteristics. The population for a clinical trial is all the people the test or treatment is designed to help.

Protocol

A plan or set of steps that defines how something will be done. Before carrying out a research study, for example, the research protocol sets out what question is to be answered and how information will be collected and analysed.

Randomized Controlled Trial (RCT)

A study in which a number of similar people are randomly assigned to 2 (or more) groups to test a specific drug, treatment or other intervention. One group has the intervention being tested; the other (the comparison or control group) has an alternative intervention, a placebo, or no intervention at all. Participants are assigned to different groups without taking any similarities or differences between them into account. For example, it could involve using a computer-generated random sequence. RCTs are considered the most unbiased way of assessing the outcome of an intervention because each individual has the same chance of having the intervention.

Reliability

The ability to get the same or similar result each time a study is repeated with a different population or group.

Sample

People in a study recruited from part of the study’s target population. If they are recruited in an unbiased way, the results from the sample can be generalised to the target population as a whole.

Subjects

In clinical trials, the people selected to take part are called subjects. The term applies to both those participants receiving the treatment being investigated and to those receiving a placebo or alternate treatment.

Trial Site

The location where trial-related activities are conducted.


References

The Canadian Institutes of Health Research (CIHR)

TROG Cancer Research

ICH.org

NICE

Further Resources

American Society of Clinical Oncology’s Cancer.Net trials site

National Cancer Institute (NCI) Clinical Trials lists open and closed cancer clinical trials sponsored or supported by NCI. 

ClinicalTrials.gov database of privately and publicly funded clinical studies

CenterWatch Clinical Trials Listing

Medication Maintenance Tips for Caregivers

Managing medications can be difficult to do, especially if you’re a senior caregiver. Helping someone else remember to take medications on time and work to find the right balance for them can seem like a daunting task. Thankfully, we’ve got a list of tips and tricks to help make things flow more smoothly.

Make Sure Providers Are Aware Of Vitamins And Supplements

Medical providers should be aware of any vitamins and supplements a person is taking. Regardless of how natural they are, they can interfere with medications and other treatments. For example, someone on blood thinners should not be taking a supplement with vitamin K. Most blood thinners work by inhibiting the production of this vitamin in the body. Taking a vitamin K supplement can negate the work of blood thinners.

Instructions

Make sure to go over medication instructions with the senior you’re caring for. If they are able to, they should know the names of each medication along with dosages and what times to take them. It doesn’t hurt to type up instructions about medications so that all information is in one place and easy to access. Consider adding in what side effects they should seek help for. That can serve as a list for caregivers and seniors to check on in case of adverse events.

Alarms

Set alarms to remind seniors to take their medications. There are many options to choose from. Smartphones allow you to set up reminders with different sounds each time which can help people differentiate between medication doses and other alerts. Electronic personal assistants like Alexa or Google Home can easily be used for reminders as well. If the senior you’re caring for struggles with newer technology, consider a few alarm clocks around the home.

Keep A List

Keeping a list of medications can help seniors and caregivers alike remember what medications are due at what time. Lists that have both a visual of what the medications look like and allow people to check off a medication dose can be useful tools. If you’re going with this kind of list, make sure that you have multiple copies. Placing one next to a pill organizer and another on the fridge can help remind people to take medication before they’ve even missed a dose.

Smartphone apps can also be helpful in tracking this information.

Follow Up

It’s important not to just set alarms or reminders, but check in to ensure that someone has taken their medication. It can be easy to turn off an alarm and still forget to take medication as scheduled. Following up with the senior in your life can remind them that they didn’t take their most recent dose.

Store Medications Properly

Most medications do best when stored between 68 and 77 degrees Fahrenheit. Additionally, many of them need to avoid humidity, direct sunlight and more. Medications should not be stored in vehicles, on windowsills or other sunny and warm spots or even in the bathroom. Consider storing them in a cool, dry space in the kitchen or living space.

When medications aren’t stored properly, it can affect their potency and make them potentially dangerous. If you’re concerned that your senior’s medications have been affected, here’s what you need to watch out for:

  • Odd smells
  • Discolored pills, tablets and injections
  • Cracked or crumbled pills
  • Pills and tablets that are stuck together
  • Creams and ointments that show separation
  • Cloudy injections

If you see these signs, contact your senior’s pharmacist as soon as possible.

Sort Medications Into Pill Organizers

Set aside time each week to go through the medication your senior takes and place them into pill organizers. These can make it easier to remember to take medications as prescribed or even transport them while traveling. Some organizers can remind people to take their medications and even alert others that a dose has been missed.

Make Sure All Caregivers Know About Medications

A sure way to have seniors miss their medication doses is to have senior caregivers who aren’t on the same page. Without everyone being in the know, it becomes increasingly difficult to set reminders and follow up with seniors about medication doses.

Plan Ahead For Refill Needs

Refills may come up on days where a senior is alone. When that’s the case, they may forget or be unable to pick up their refilled medications. Refills may even be due when someone is planning to be out of town. Make sure to plan ahead adequately for refills and work with a person’s pharmacist.

Consider Compounding Medications If Needed

Compounding is a process where medication is tailored to a person’s specific needs. This can help remove any dyes a patient is allergic to or turn a pill into liquid for those who struggle with swallowing pills.

Get Tips from A Medical Provider

When methods to help your senior aren’t working as well as you had hoped, take some time to check in with their medical providers. Nurses have amassed a wealth of information on improving their patients’ quality of life. They are likely to have some ideas on how to make managing medications more effective.

Always Communicate With Family Members

Whatever steps you take to maintain a senior’s medication schedule, make sure that you’re communicating any difficulties with the senior’s loved ones. Family should also always be aware of any medication changes. When so many seniors rely on a variety of paid and family caregivers, it’s incredibly important for everyone to be in the loop on the storage, administration and organization of all medications, vitamins and supplements.

Reinventing the Clinical Trial: Start at Ground Level

If each of us humans is a snowflake, unique in our genomic makeup, where’s my snowflake medicine? I asked that question from the platform at the ePharma Summit in New York in 2013, and have yet to get an answer. The challenge for the bioscience industry is, I believe, the classic randomized clinical trial. That design goes through four phases:

  • Phase 1: a small group of people are given the drug under study evaluate its safety, determine a safe dosage range, and identify side effects
  • Phase 2: a larger group is given the drug to evaluate its efficacy and safety in a larger population
  • Phase 3: large groups – plural – of people are given the drug to confirm its effectiveness, monitor side effects, compare it to other commonly-used treatments, and collect information that will allow the drug /treatment to be used safely
  • Phase 4: the drug is marketed while study continues to assess long-term effects and efficacy

Of course, before they even get to Phase 1, there have to be both the idea for the new treatment, and animal studies to determine what the substance or compound under study might do to a mouse or a monkey.

Science isn’t easy. The phrase “trial and error” came out of science labs, with many trials running up against the error wall by Phase 2. Since bioscience companies can sink about $1 billion-with-a-B into getting just one drug to market, it seems that the traditional clinical trial has turned into a pathway to NOT making scientific discoveries that can benefit humankind.

Then there’s the whole “who’s in charge here?” question. Clinical trials are now a global effort, with US and European pharma companies testing new treatments in Latin America, Russia, and China to gain traction in those emerging markets while simultaneously developing me-too drugs for their domestic markets. So, who’s in charge, the US Food and Drug Administration (FDA)? The European Medicines Agency (EMEA)? A player to be named later? The answer to the question seems to be “all of the above,” which adds to the complexity of the clinical trial process.

As digital technology has made data easier to collect and share, it would seem that clinical trials would be a great place to start intersecting with the quantified-self movement. The shift to electronic health records, the widening adoption of all sorts of health tracking devices, and the rise of (relatively) cheap genomic sequencing should signal an ability to identify conditions, and populations, eager to participate in clinical investigations. But so far, it hasn’t.

What might challenge that stasis? In November 2013, three major pharma companies – Novartis, Pfizer, and Eli Lilly – announced via the White House’s website that they had joined together in a clinical open innovation effort. That page on the White House’s site is gone now – changes in Presidential administrations will do that – but here’s a direct quote from that announcement:

“In order to connect patients and researchers, Novartis, Pfizer and Eli Lilly and Company, are partnering in the U.S. to provide a new platform to improve access to information about clinical trials. The platform will enhance clinicaltrials.gov and will provide more detailed and patient-friendly information about the trials, including a machine readable ‘target health profile’ to improve the ability of healthcare software to match individual health profiles to applicable clinical trials. As part of the project, patients can search for trials using their own Blue Button data.”

Five years later, and we’re still stuck on the slow train when it comes to really reinventing the clinical trial.

I’m one of a growing group of people who think that the entire life-sciences process chain needs to be re-tooled for the 21st century. In my view, the best place to start that re-tool is at ground level, with the patients and clinicians who deal with challenging medical conditions daily. If a doctor has a number of patients who might benefit from some clinical study, why isn’t there an easy way to find a researcher looking into that condition? If a patient has an idea for a clinical investigation into his or her illness or condition, why can’t they find a researcher who’s interested in the same condition to team up and start a science project?

I can only hope that the regulatory agencies involved in life science oversight (hello, FDA!) can move beyond the aftermath of Thalidomide – for which epic disaster we’re still paying a price when it comes to the timeline for drug approval in the US – and toward a process of “all deliberate speed” that doesn’t forsake speed for deliberation. Both are necessary, neither should be more heavily weighted than the other.

We all can, and should, take part in scientific exploration into human life, and human health. Got an idea for a clinical trial? Share that idea in the patient communities you hang out in, and ask your tribe to help you bring that trial to life. To quote Arthur Ashe, “Start where you are. Use what you have. Do what you can.”

We’ve got to start somewhere, right?

Talking To Your Family About Clinical Trial Decisions

Hearing your name and the word “cancer” in the same sentence is a world-shaking moment. After getting a cancer diagnosis, telling your family about it is another big step, one that can be fraught with as much emotion as hearing that diagnosis yourself.

Once the emotional dust has settled, talking with your family about treatment options, including clinical trials, can raise the emotional temperature again. If your family is like mine, everyone has an opinion, and is more than ready to share it. Even in families where everyone is calm about big issues like this – I question that those families exist, but I’ve heard they might – talking about clinical trials as a treatment option means being ready to field questions, and guide the conversation.

The American Cancer Society has a great set of resources for people who are assessing whether clinical trials are a good option for their treatment. I’ll use some of those as a framework for a discussion guide you can use to walk your family through your decision to explore clinical trials for your cancer:

  • Why do I want to participate in a clinical trial?
    • Your reasons can be anything from “I want to try cutting edge treatments” to “my cancer is advanced stage, and I want to throw everything but the kitchen sink at it.” The key here is to have an answer ready to this question when you discuss treatment options with your family.What are the risks?
  • What are the risks?
    • Here’s another question you’ll want to gather answers for, for yourself, before opening a conversation with your family about enrolling in a trial. Your oncology team can help you put together a risk profile for trials, and further help you target the right trials via molecular profiling of your cancer.
  • Will my insurance cover the trial?
    • Federal law requires that most insurers cover routine costs of cancer trials. However, like so much about US health insurance, the answer can still be “it depends.” There’s a great tip-sheet on the National Cancer Institute’s site that addresses this topic. You, and your family, and your oncology team, will be working together to make sure your costs are covered, either by your insurer or the trial sponsor.
  • What happens if I’m harmed by the trial – what treatment will I be entitled to?
    • Here’s another “it depends” situation. Addressing harm to trial participants is an ongoing ethics issue in the US. The key here is to review all trial enrollment documentation fully – with help from a medical ethicist or legal eagle who’s not involved with the trial, or your oncology team – and have any potential harm scenario fully spelled out, including who will address the remedy for harm, and how that remedy will be delivered.

Having solid family support is a key factor in managing cancer treatment, and in thriving as a cancer survivor. Getting your family involved in your care by talking through your options and decisions with them will give them a sense of involvement in your care, and its outcome. They can help you through the down days when side effects have you feeling punky, and celebrate the bright days with you when scans show progress against your cancer.

Curing cancer is a team sport. You, your family, and your oncologists are all on that team. Work together toward a win, which often includes unlocking the power of precision medicine via clinical trials – which can become a win for other cancer patients, too.

Talking to Your Oncologist About Clinical Trials

You’ve gotten a cancer diagnosis. You’ve selected an oncologist as your partner, working toward “No Evidence of Disease,” or NED (NED is every cancer patient’s very best friend). Your and your oncologist are working up a treatment plan, and you want to talk about clinical trials as part of that plan. Should you kick off that discussion, or wait until your onc brings it up?

YES, definitely bring up clinical trials yourself, if your oncologist hasn’t started that conversation. If you’re not sure how to kick off that discussion, here are some tips.

  • “Just do it.” Lace up your mental Nikes, and just ask the question. Have some resource links handy at your next oncology team visit, or start the conversation before the visit via your oncologist’s patient portal. Start with the information on the Patient Empowerment Network’s Health Centers and Programs hub, take a dive into gov, or check out the American Society of Clinical Oncology’s Cancer.Net trials site.
  • There’s an article in the Journal of Oncology Practice, “Identifying and Selecting a Clinical Trial for Your Practice,” that talks clinicians through the process of selecting clinical trials for their oncology practice. Reading through that can help you craft some great questions, and open a productive conversation with your treatment team about clinical trials for your cancer.
  • The National Institutes of Health has a great tip-sheet for oncologists on how to talk to their patients about clinical trials. You can use that to frame the conversation you’d like to have with your own oncologist about your clinical trial options. I’ve often found that reading articles and tip-sheets aimed at the clinical side of the equation have helped me accelerate discussions with my own clinical teams about treatment options, for cancer and for other medical conditions.

When you’re dealing with a cancer diagnosis, you want to have all your options on the table, and make the most informed decisions possible. Opening up a dialogue with your oncology team about clinical trials early in the treatment process will give you the information you need for those “most informed decisions.”

Another reason to open those discussions early is to gauge your oncologist’s response to shared decision making, and participatory medicine. If your oncologist doesn’t welcome self-advocacy on your part, it’s better to know early in the treatment process so you can shift to another, more participatory practice.

You are the focus of your cancer treatment team’s work. Lead the discussions, share your perspective, and participate fully in your treatment planning. Opening the discussing of clinical trials is a great way to get your team on your page about treatment and outcome preferences, and to unlock the power of precision medicine.

A How-To On Reading Scientific Papers

“Be skeptical. But when you get proof, accept proof.” – Michael Specter

That quote is from Denialism: How Irrational Thinking Hinders Scientific Progress, Harms the Planet, and Threatens Our Lives, where New Yorker staff writer Michael Specter examined the distrust of science that’s turned discussion of scientific topics into a potential minefield. Some good examples of that minefield are climate change, and childhood vaccinations.

Anyone interested in scientific progress – full disclosure, I’m in that group – needs to understand the ideas being explored in scientific papers, the dispatches from the front lines of scientific thinking and discovery. To arrive at that understanding, you have to be able to understand what you’re reading, and I’ll be the first to admit that isn’t easy.

Scientific papers are written by scientists, for scientists, and follow a set of rules and formal structures that can feel like they’re designed to prevent any understanding by the average Joe/Jane “just plain human.” In this post, my goal is to help anyone interested in, but not formally trained in, science tackle reading – and understanding! – an article in any scientific journal.

10 steps to scientific (article) understanding

  1. Check the source

    • What journal is publishing the article? Check Beall’s List, and if the journal appears there, you can stop reading – it’s a fake journal.
    • Who is the lead author, and what organization or institution is s/he affiliated with? If it’s an established university or research institute (University of Chicago or Scripps Institute, for example), keep reading.
  2. Read the introduction first, not the abstract

    • The introduction will reveal the Big Question, the one that the research project worked to reveal the answer to. For instance, an article in the Christmas 2017 issue of The BMJ reports on research into the effects of pet ownership on human biomarkers of ageing; the introduction clearly lays out the Big Question as “ we examined the prospective link between pet ownership and a selected range of objective biomarkers of ageing proposed for use in large scale population based studies of older people.”
  3. Write out your own summary of what the research was examining

    • This will give you a grasp of why the researchers wanted to ask the Big Question, and a framework for assessing what their answers to that question are.
  4. Identify the null hypothesis

    • The null hypothesis could really be better termed the “nullifiable” hypothesis, since the purpose of the research project is to nullify the hypothesis that there are no differences in possible answers to the Big Question.
    • An example of a null hypothesis is “the world is flat,” which is what Copernicus worked to scientifically disprove a while back. He was successful, but there are some people who still reject his conclusions. (Warning: opening that link might be hazardous to your sanity.)
  5. Look at the approach, and the methods, used in the research study or experiment(s)

    • What did the researchers do to answer the Big Question? What specific experiments did they run?
    • Sketch out diagrams of each experiment or data crunch.
  6. Read the results section of the article

    • Look at the written results, as well as all charts and figures related to those results.
    • What are the sample sizes? Really small sample sizes are a red flag.
    • What results are listed as “significant,” and what as “non-significant”? If you want to totally geek out on this topic, this post will make your geeky day.
  7. Do the results actually answer the Big Question?

    • Using your own judgment, do you think the study authors have answered the question asked in the introduction?
    • Do this before you read the paper’s conclusion.
  8. Does the conclusion make sense, in light of everything you’ve read and evaluated while going through the paper?

    • Do you agree with the conclusion?
    • Can you identify an alternative explanation for the results in the article?
    • What are the next steps the authors see emerging from their research?
  9. Read the abstract at the beginning of the paper

    • In light of the work you did in Steps 1 through 8, does the abstract line up with what the authors said their research purpose was?
    • Does it fit with your own interpretation of the paper?
  10. What are other scientists saying about the paper?

    • Have other scientists written about this paper?
    • What other research is referenced in the paper?
    • Have the authors of that research weighed in on the paper you’re evaluating?

Reading, and understanding, scientific papers takes practice. It’s also fun, if you’re a science nerd, or just interested in new scientific discoveries. And it’s work worth doing, because the more you know, the more likely it is that you yourself might make a discovery that makes a difference.

Paying It Forward: Volunteering for Clinical Trials

Editor’s Note: This blog and video is from the Alliance for Aging Research. The Alliance for Aging Research is dedicated to accelerating the pace of scientific discoveries and their application to vastly improve the universal human experience of aging and health.

Getting medical discoveries from the research lab to patients depends on clinical trials and the people who volunteer to participate in them.  Volunteering in a trial may help society at large by bringing new treatments one step closer to patients, and could help a loved one if you have a genetic disease or condition.  Volunteering may also give you access to a cutting-edge treatment and medical team that carefully monitors your health.  But clinical trials can’t happen without volunteers, and 37% of trials don’t enroll enough patients to move forward.  Clinical trials need volunteers like you so watch this short film to find out more about why they are important, how to get involved, and what it means to participate.