MPN Treatments and Clinical Trials Archives

When it comes to treatment, MPN patients and their care partners have much to consider. There are often many options available, each with advantages and disadvantages. Some people may seek clinical trials, others may have few feasible options. Understanding treatment options, goals, and what to expect are vital to achieving the best possible outcome for you.

More resources for Myeloproliferative Neoplasms (MPN) Treatments and Clinical Trials from Patient Empowerment Network

Clinical Studies and Research Opportunities For Patients

Essential Thrombocythemia (ET)

Background 

QuintilesIMS Global Services, a worldwide consulting firm, is conducting a research study to better understand the experience of ET patients. QuintilesIMS plans to interview patients who have been diagnosed with ET one‐on‐one over the phone. QuintilesIMS is looking for interested people to share what they have gone through since being diagnosed with ET.

Purpose

The overall purpose of the study is to learn which symptoms of ET are the most common, and how these symptoms typically affect the lives of patients like you who live with the disease. This understanding may help improve how the patient’s experience is accounted for in clinical trials that measure the effect of disease treatments. It also may help patients and their families by highlighting the consequences of the disease that most need greater attention, possibly leading to the development of new programs and support services.

Your Involvement

If you qualify, you will be invited to participate in a 75‐minute telephone discussion with one of QuintilesIMS’ healthcare researchers. You will not need to do anything to prepare, other than being ready to describe what your life has been like while living with ET. At no point before, during, or after the interview will you be contacted to purchase anything or be asked to take any medication. You will be one of 20 patients participating in the study and your information will be kept confidential by removing any personal identifiers.

To qualify, patients must have the following criteria:

1. Patient is ≥18 years of age

2. Patient has a current diagnosis of Essential Thrombocythemia (ET) and fulfills one of the following:

· Patient has received hydroxyurea treatment in the past and discontinued it for any reason

OR

· Patient could not receive hydroxyurea because it was contraindicated for the patient

3. Patient has some self‐reported disease related symptom burden

4. Patient is physically and mentally able to participate in an 75 minute interview in English to discuss signs, symptoms, and impacts related to ET

5. Patient is not currently participating in a research study where an investigational agent is being administered

6. Patient has no prior treatment with any oral JAK inhibitor

Compensation

In return for your voluntary participation in the interview, your time and effort will be compensated with a $125 Visa gift card.

Next Steps

To participate, please call Michael Posey from QuintilesIMS, at +1 412‐973‐3162 (Monday‐Friday, 9:00am ‐ 5:00pm EDT), or write to michael.posey@QuintilesIMS.com. Michael will ask for your consent to participate in the study, see if you qualify, then schedule your interview.

Essential Thrombocythemia (ET)

A Double-Blind, Double-Dummy Phase 2 Randomized Study to Evaluate the Efficacy and Safety of Ruxolitinib Versus Anagrelide in Subjects With Essential Thrombocythemia Who Are Resistant to or Intolerant of Hydroxyurea (RESET-272)

The purpose of this study is to evaluate the efficacy and safety of ruxolitinib versus anagrelide in subjects with essential thrombocythemia who are resistant to or intolerant of hydroxyurea.

Talk to your doctor if you are interested in participating in this study.

To find a study center near you, call , then select option 1.

You can also visit clinicaltrials.gov and enter NCT03123588 to learn more.

Multiple Myeloma

Help Us Better Understand Multiple Myeloma Treatment Experiences

Pinpoint Patient Recruiting is seeking individuals with Multiple Myeloma to participate in a market research study about patients’ experiences with treatment decisions for Multiple Myeloma. The feedback will be used to help future patients and their caregivers.

If you are currently being treated for Multiple Myeloma and are receiving your second, third or fourth course of drug therapy (because previous courses of therapy either did not work, stopped working or you experienced a relapse), you may qualify to participate.

If you qualify and complete the online survey, you will receive an honorarium of $75 for your time and participation. All information and responses will remain confidential.

To see if you qualify or to get more information visit: https://www.pinpointpatientrecruiting.com/multiple-myeloma-research-pen

You may also contact Kim Slusher via email at kim@pinpointpatientrecruiting.com.


Myelofibrosis

A Phase 2 Study of the Safety, Tolerability, and Efficacy of INCB050465 in Combination With Ruxolitinib in Subjects With Myelofibrosis

The purpose of this study is to evaluate the safety, tolerability, and efficacy of the combination of INCB050465 and ruxolitinib in subjects with myelofibrosis

Talk to your doctor if you are interested in participating in this study.

To find a study center near you, call , then select option 1.

You can also visit clinicaltrials.gov and enter NCT02718300 to learn more.

Understanding Itching and Night Sweats With MPN

From the Understanding Myeloproliferative Neoplasms (MPNs) Town Meeting, a panel of experts explains why MPN patients have to deal with itching and night sweats and what they can do to treat those side effects. The panel includes:

  • Olatoyosi Odenike, MD, Associate Professor of Medicine at The University of Chicago Medical Center
  • Julie Huynh-Lu, PA-C, Physician Assistant, Department of Leukemia at The University of Texas MD Anderson Cancer Center
  • Srdan Verstovsek, MD, PhD, Professor, Department of Leukemia, Division of Cancer Medicine at The University of Texas MD Anderson Cancer Center
  • Carmelita Escalante, MD, FACP, Professor and Chair, Department of General Internal Medicine at The University of Texas MD Anderson Cancer Center

Please check out the full video below to hear from the experts.

Understanding Itching and Night Sweats With MPN from Patient Empowerment Network on Vimeo.

How Do You Find Out About Clinical Trials?

Interview with Larry Anderson, Jr., MD, PhD, Assistant Professor, Department of Internal Medicine, Division of Hematology/Oncology University of Texas Southwestern Medical Center, Patient Advocate, Lynette, and Robert Orlowski, MD, PhD, Director of Myeloma and Professor in the Departments of Lymphoma/Myeloma and Experimental Therapeutics The University of Texas MD Anderson Cancer Center

From  the Virtual Town Meeting: Understanding the New World of Myeloma Treatment, Andrew Schorr first ask Dr. Anderson about how patients can find out about clinical trials, whether that be a governmental website, advocacy groups, or each institution’s individual website. Later he gets Lynette and Dr. Orlowski’s opinion on the matter. Check out the full video below to hear from three myeloma experts.

How Do You Find Out About Clinical Trials? from Patient Empowerment Network on Vimeo.

What Can Help Fight Fatigue With MPNs?

Interview with Carmelita P. Escalante, MD, FACP, Professor and Chair Department of General Internal Medicine The University of Texas MD Anderson Cancer Center

From the October 2016 MPN Town Meeting, the question, “Is there any drugs or maybe even vitamin B-12 that can be used to combat the fatigue associated with an MPN?” Check out the full video below to see how MPN expert, Dr. Carmelita Escalante, answers.

What Can Help Fight Fatigue With MPNs? from Patient Empowerment Network on Vimeo.

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Jeff Folloder: I have a question for Dr. Escalante. Mark from the internet wants to know if there’s any drugs or maybe even vitamin B-12 that can be used to combat the fatigue associated with an MPN.

Dr. Carmelita Escalante: For fatigue – specifically fatigue we look at other conditions. So, we try to treat those conditions, whether they’re depression, etc. Specifically, for fatigue, we would not use B-12 unless your B-12 deficient. There’s not literature to say that B-12 supplements are helpful unless you’re low on B-12.

The drugs that we have used and there is mixed evidence, is stimulants. Stimulants are drugs like methylphenidate, which is Ritalin, which has been used in children with ADHD, attenuation deficit disorders. It’s probably the most studied of all the stimulants. We’ve also used provigil and nuvigil, which is modafinil and armodafinil. They are approved for sleep dysfunctions; such as sleep apnea.

There’s been a scattering of others that have been used, like Adderall. The data is mixed. There is probably more negative trials than positive. But, in severe fatigue, there is a small – there’s some data that shows improvement.

I specifically try to get the behavioral and exercise treatment going, but for some patients the stimulants can be very helpful. And we use them in fairly small doses. There’s very negligible side effects. So, I think it’s a win if I use it and the patient feels better and can do more.

There is a big placebo effect. So, when we do trials with it we always have to have a placebo on. But, the bottom line is if it’s helpful and the patient is tolerating it and doing the other things that we’ve prescribed to try to improve the fatigue and we have everything controlled that we can, and it helps, I think it’s a good thing. Especially, I’ve noticed if there are cognitive deficits, which is – which methylphenidate has been used for in kids to try to get them to focus, it can be very helpful.

We use it a lot in our brain tumor patients. I’ve used it especially inpatients that have jobs where they really have to focus and they tell me I just can’t do my work because I can’t keep focus, and it can be extremely helpful for them.

Andrew Schorr: Okay, what about the obvious one? I’m drinking a lot of coffee. Coffee’s okay?

Dr. Carmelita Escalante: Coffee’s okay. It’s a fairly week stimulant. I think if you have very mild fatigue and it helps you. I’d be careful in those energy drinks because sometimes, especially if you’re on other medications or if you have heart conditions, you need to be very careful about drinking several a day – may not be in your best interest, especially if you feel your heart racing or you feel jittery.

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Nothing About Us Without Us: Patient Involvement in Research

Until recently, patient participation in research was limited to their involvement as subjects enrolled in research studies, but there is a shift occurring as funding bodies increasingly look for evidence of patient and public involvement (PPI) in research proposals. The rationale for this is increasing evidence that PPI in the provision of healthcare leads to improved outcomes and better quality of care.

Assumptions are made every day about patients; assumptions which may lead to a failure to deliver optimum care. When these assumptions extend to research, quite often there is a mismatch between the questions that patients want answers to and the ones that researchers are investigating. As an example, the research priorities of patients with osteoarthritis of the knee, and the clinicians looking after them, were shown in a study to favor more rigorous evaluation of physiotherapy and surgery, and assessment of educational and coping strategies. Only 9% of patients wanted more research on drugs, yet over 80% of randomized controlled trials in patients with osteoarthritis of the knee were drug evaluations. PPI recognizes that patients bring a unique perspective and experience to the decision-making process in research. It is paternalistic and patronizing to rely on speculation about patient experience. By considering the actual experience of patients, researchers can make more informed research decisions. Involving patients is an important step in ensuring that the real life experiences of patients are considered when it comes to setting research priorities. This in turn will increase the relevance of research to patients and improve research quality and outcomes.

As an advocate you may be asked to become involved in a research project, so it is important to have a clear understanding of what PPI is – and what it isn’t. PPI is not about being recruited as a participant in a clinical trial or other research project, donating sample material for research, answering questionnaires or providing opinions. PPI describes a variety of ways that researchers engage with people for whom their research holds relevance. It spans a spectrum of involvement which may include any of the following:

  • Being involved in defining the research question
  • Being a co-applicant in a research proposal
  • Working with funders to review patient-focused section of applications
  • Being an active member of a steering group for a research study
  • Providing your input into a study’s conception and design
  • Contributing to/proofing of documentation
  • Assisting in the implementation and dissemination of research outcomes
  • Improving access to patients via peer networks and accessing difficult-to-reach patients and groups

Effective PPI transforms the traditional research hierarchy in which studies are done to, on, or for participants into a partnership model in which research is carried out with or by patients.  PPI should always involve meaningful patient participation and avoid tokenism. The Canadian Institutes of Health Research Strategy for Patient-Oriented Research (SPOR) describes PPI as fostering a climate in which researchers, health care providers, decision-makers and policy-makers understand the value of patient involvement and patients see the value of these interactions. Underpinning this framework are the following guiding principles for integrating patient engagement into research:

  • Inclusiveness:Patient engagement in research integrates a diversity of patient perspectives and research is reflective of their contribution.
  • Support:Adequate support and flexibility are provided to patient participants to ensure that they can contribute fully to discussions and decisions. This implies creating safe environments that promote honest interactions, cultural competence, training, and education. Support also implies financial compensation for their involvement.
  • Mutual Respect:Researchers, practitioners and patients acknowledge and value each other’s expertise and experiential knowledge.
  • Co-Build:Patients, researchers and practitioners work together from the beginning to identify problems and gaps, set priorities for research and work together to produce and implement solutions.

Derek Stewart, a patient advocate and Associate Director for Patient and Public Involvement at NIHR Clinical Research Network, sees a growing momentum of actively involving patients and public in research gathering pace worldwide. “It is really pleasing to hear researchers saying how valuable it has been to involve patients and the public in their work”, he says. “It has equally improved the quality of the research and enriched their own thinking and understanding.”

Earlier this year, PCORnet, the National Patient-Centered Clinical Research Network, announced its first demonstration study which reflects PCORnet’s aims of patient engagement and open science. ADAPTABLE (Aspirin Dosing: A Patient-centric Trial Assessing Benefits and Long-Term Effectiveness) will compare the effect of two different aspirin doses given to prevent heart attacks and strokes in high-risk patients with a history of heart disease. Seeking input at every critical step, from consent design and protocol development, through dissemination of final study results, the project represents a new research paradigm. Unprecedented in the design of clinical trials, the final consent form and protocol were shaped with input from patients, local institutional review boards, physicians, and study coordinators.

Another noteworthy example of PPI can be found in the Metastatic Breast Cancer Project a direct-to-patients initiative launched at the Broad Institute of MIT and Harvard last October. Corrie Painter, an angiosarcoma patient and Associate Director of Operations and Scientific Outreach at Broad Institute, explains that “the project seeks to greatly accelerate the pace of biomedical research by empowering patients to directly contribute to research and was built in lock step from design to consent language with dozens of patients.”

To what extent you may wish to be involved in PPI will depend on several factors. Do you have professional experience (e.g. project management, clinical experience, etc.) which would be useful? Are you happy to work as part of a team? Or would you prefer to work on your own? You should also take into consideration your other work or family commitments. For instance will you need to take time off work to attend meetings? Consider also at what point you are in your own health journey. Will participation in research place an added burden on your treatment or recovery? In making the decision to become involved in research, you should always balance your own health needs with the desire to be supportive of research and the research process.

 

Useful links

PCORI www.pcori.org

PCORnet www.pcornet.org

Metastatic Breast Cancer Project www.mbcproject.org

#WhyWeDoResearch www.whywedoresearch.weebly.com

Who Is Eligible and How Can I Learn More About Clinical Trials?

From the Lung Cancer Town Meeting in September 2016, Janet Freeman-Daily interviews a panel of lung cancer experts about who is eligible for clinical trials and how you can learn more about them. The panel includes the following experts:

  • Nisha Monhindra, MD Assistant Professor of Medicine, Hematology/Oncology Division, Feinberg School of Medicine Robert H. Lurie Comprehensive Cancer Center of Northwestern University
  • D. Ross Camidge, MD, PhD, Director Thoracic Oncology Clinical and Clinical Research Programs University of Colorado Denver
  • David D. Odell, MD, MMSc, Assistant Professor, Thoracic Surgery Robert H. Lurie Comprehensive Cancer Center of Northwestern University
  • Timothy J. Kruser, MD, Assistant Professor, Radiation Oncology Robert H. Lurie Comprehensive Cancer Center of Northwestern University

Check out the full video below to hear all of the lung cancer experts advice.

Who is Eligible and How Can I Learn More About Clinical Trials? from Patient Empowerment Network on Vimeo.

How to Read Beyond the Headline: 9 Essential Questions to Evaluate Medical News

Ben Goldacre writing in Bad Science classified science reporting as falling into three categories – wacky stories, scare stories and breakthrough stories; the last of which he views as ”a more subtly destructive category of science story”. Whether you get your news through digital or traditional means, you can’t fail to notice the regularity with which journalists report on the latest medical breakthroughs. Some of these reports are sensationalist (“coffee causes cancer”) and fairly easy to dismiss; but do you know how to separate fact from fiction when it comes to less sensationalist headlines?

The foundation of empowered patient-hood is built on reliable health information. This means not only knowing where to find medical information, but being able to evaluate it and knowing how it can be applied to your own, or your loved-ones’ particular circumstances. Headlines often mislead people into thinking a certain substance or activity will prevent or cure chronic disease. As patient advocates we must learn to read beyond the headlines to filter out the good, the bad, and the questionable. The following questions are designed to help sort the signal from the noise next time you read the latest news story heralding a medical breakthrough.

1. Does the article support its claims with scientific research?

Your first concern should be the research behind the news article. If an article contains no link to scientific research to support its claims, then be very wary about treating those claims as scientifically credible.

2. What is the original source of the article?

If the article cites scientific research you should still treat the findings with caution. Always consider the source. Find out where the study was done. Who paid for and conducted the study? Is there a potential conflict of interest?

3. Does the article contain expert commentary to back up claims?

Look for expert independent commentary from doctors or other healthcare providers to explain the findings (there should be an independent expert source quoted – someone not directly connected with the research).

4. Is this a conference presentation?

Journalists frequently report on research presented at large scientific meetings. It’s important to realize that this research may only be at a preliminary stage and may not fulfill its early promise.

5. What kind of clinical trial is being reported on?

If the news relates to results from a clinical trial, it’s important you understand how, or even if, the results apply to you. Quite often, news publications report on trials which have not yet been conducted on humans. Many drugs that show promising results in animals don’t work in humans. Cancer.Net and American Cancer Society have useful guides to understanding the format of cancer research studies.

6. What stage is the trial at?

Research studies must go through several phases before a treatment can be considered safe and effective; but many times journalists report on early phase trials as if these hold all the answers. The testing process in humans is divided into several phases:

  •  Phase I trials: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
  • Phase II trials: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
  • Phase III trials: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.

Source: ClinicalTrials.gov

7. How many people did the research study include?

In general, the larger a study the more you can trust its results. Small studies may miss important differences because they lack statistical power.

8. Did the study include a control group?

A control group allows researchers to compare outcomes in those who receive a treatment with those who don’t. The gold standard is a “randomised controlled trial”, a study in which participants are randomly allocated to receive (or not receive) a particular intervention (e.g. a treatment or a placebo).

9. What are the study’s limitations?

Many news stories fail to point out the limitations of the evidence. The limitations of a study are the shortcomings, conditions or influences that cannot be controlled by the researcher. Any limitations that might influence the results should be mentioned in the study’s findings, so always read the original study where possible.

Useful Resources

  • Gary Schweitzer’s Health News Review website provides many useful resources to help you determine the trustworthiness of medical news. To date, it has reviewed more than 1,000 news stories concerning claims made for treatments, tests, products and procedures.
  • Sense about Science works with scientists and members of the public to equip people to make sense of science and evidence. It responds to hundreds of requests for independent advice and questions on scientific evidence each year.
  • Trust It or Trash is a tool to help you think critically about the quality of health information (including websites, handouts, booklets, etc.).
  • Understanding Health Research (UHR) is a free service created with the intention of helping people better understand health research in context. It gives clear and understandable explanations of important considerations like sampling, bias, uncertainty and replicability.

MyLifeLine: Learn About Clinical Trials

Editor’s Note: This post was originally published here on MyLifeLine.org. The mission of MyLifeLine.org is to empower cancer patients and caregivers to build an online support community of family and friends to foster connection, inspiration, and healing through free, personalized websites.

Learn About Clinical Trials

MLL ACT

Why consider a cancer clinical trial?

What clinical trials can offer, from the care you receive to the impact you can make.

Clinical trials offer a chance to receive investigational medicines or procedures that experts think might improve the treatment of cancer. This important option is not limited to people who have run out of choices. In fact, there may be clinical trials for every stage of disease in dozens of cancer types. In this video, patients and doctors share their perspectives on why joining a clinical trial may be an option worth considering.


“To have the opportunity to go on a clinical trial for a patient is extremely exciting.” —Sandra Swain, MD; oncologist


Screen Shot 2016-05-18 at 10.23.06 AM
Concern:
I don’t want to be a guinea pig for an experimental treatment.
The Truth:
Cancer clinical trials are developed with high medical and ethical standards, and participants are treated with care and with respect for their rights.

Concern:
I’m afraid i might receive a sugar pill or no treatment at all.
 The Truth:Cancer clinical trials rarely use placebo alone if an effective treatment is available; doing so is unethical.

Concern:
Cancer clinical trials are only for people with no other treatment options.
 The Truth:Trials can study everything from prevention to early- and late-stage treatment, and they may be an option at any point after your diagnosis.

Concern:
I’m worried that I won’t receive quality care in a cancer clinical trial.
 The Truth:Many procedures are in place to help you receive quality care in a cancer clinical trial.

Concern:
People might access private information about me if I participate.
 The Truth:In nearly all cancer clinical trials, patients are identified by codes so that their privacy is protected throughout and after the study.

Concern:
I’m afraid that my health insurance will not help with the costs of a cancer clinical trial.
 The Truth:
Many costs are covered by insurance companies and the study sponsor, and financial support is often available to help with other expenses; talk to your doctor to understand what costs you could be responsible for.

Concern:
Informed consent only protects researchers and doctors, not patients.
 The Truth:
Informed consent is a full explanation of the trial that includes a statement that the study involves research and is voluntary, and explanations of the possible risks, the possible benefits, how your medical information may be used, and more. Informed consent does not require you to give up your right to protection if the medical team is negligent or does something wrong.

Concern:
I’m afraid that once i join a cancer clinical trial, there’s no way out.
 The Truth:
You have the right to refuse treatment in a cancer clinical trial or to stop treatment at any time without penalty

How to know if a cancer clinical trial is right for you.

There are many factors to keep in mind when considering a cancer clinical trial.

As with any important decision, it’s a good idea to think about the risks and benefits of joining a cancer clinical trial. This video encourages you to ask your medical team about all of your treatment options, including cancer clinical trials. Trial participants, doctors, and patient advocates explain the factors you’ll want to keep in mind as you consider your treatment plan.


“I’ve always advised patients…when the circumstances weren’t urgent, to take time to understand their disease and to evaluate the alternatives.”  —Sandra Horning, MD; oncologist and chief medical officer


What to ask your doctor(s)

Asking The Right Questions Keeps You Involved In Your Care

A cancer diagnosis is often overwhelming, and it’s sometimes hard to gather your thoughts and know the right questions to ask. This video talks you through some of the questions it will be helpful to ask about your cancer, your treatment options, your doctor, and about whether participating in a cancer clinical trial is right for you.


“Talk to your doctor and say, ‘Tell me my full options.’ Raise questions. Be a pain in the neck. That’s what the doctor is there for.” —Arthur Caplan, PhD; medical ethicist


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Rules And Procedures Are In Place So That You Will Receive High-Quality Care

Before a single patient can join a trial, many different experts must approve every detail of the study—from why it’s being done to how often patients should be monitored. Once the trial begins, more unbiased experts provide oversight to check that the rules of the trial are being followed and patients’ rights are protected. This video features doctors and patient rights advocates explaining the high standards by which trials are developed and run.


“I explain…that when they’re on a clinical trial, they’re going to be followed very closely by…specific guidelines.” —Daniel P. McKellar, MD; surgeon and Commission on Cancer chairman


Informed Consent Describes The Study Process, Potential Risks And Benefits, And Your Rights As A Participant

If you are eligible and decide to join a trial, you will be required to review and sign the informed consent forms. This can be an overwhelming process, but it is how you will learn all the details of the trial, including the potential benefits and the possible risks, and give your permission to be treated. This video features patients, doctors, and patient rights advocates who offer tips and insights to help you navigate the process of informed consent.


“When I received the stack of papers…it made me realize this is really serious. But then…it was actually a good feeling to know that this was not something that was being done lightly.” —Rose Gerber; trial participant


Information And Support Are Close At Hand

Because so many people have been affected by cancer, there are many reliable and helpful resources to help you through your cancer journey. In this video, trial participants and doctors help you find the people and resources that may be helpful in educating you about cancer clinical trials.


“The first thing is to hold on tight and be optimistic and to get very engaged and educated about your cancer.” —Jack Whelan; trial participant


Reliable Resources To Help Along The Way

First, talk to your doctor

Your healthcare team is the best source for information about your treatment options, including cancer clinical trials. There are many questions you’ll want to ask your healthcare team when you’re ready to discuss treatment options. Print this helpful Discussion Guide and bring it to your next appointment so that you don’t forget anything important. Record your answers on the form and keep it handy for future reference.


Where to find information about cancer clinical trials

These clinical trial resources will help you find trials that might be right for you.


Support services

These trustworthy sources provide assistance with trial-related costs, which may not always be covered by insurance.

Practical support

Financial support

Additional nationwide support organizations


Don’t go it alone

There are millions of people just like you who are ready to ACT against cancer. These organizations provide advocacy, information, awareness, fundraising opportunities, and a community of like-minded people touched by cancer.

Heading Off Cancer Growth on the Cellular Level

Cancer cells are like all the cells in our body, in that they need certain basic building blocks – amino acids – in order to reproduce. There are 20 amino acids found in nature. The amino acid serine is often found in abundance in patients with certain types of breast cancer, lung cancer, and melanoma. The overproduction of this amino acid is often required for the rapid and unregulated growth characteristic of cancer.

Scientists at the Scripps Research Institute (TSRI) wondered if there was a way to take advantage of the relationship between cancer cell proliferation and serine. Amy GrayThey examined a large library of molecules -numbering 800,000 – to find an enzyme that inhibited serine production. After much research, the group found 408 contenders that could possibly work. This list was again narrowed down to a smaller set of seven, ending with one promising candidate. This molecule, 3-phosphoglycerate dehydrogenase (PHGDH), seemed to inhibit the first step in a cancer cell’s use of serine to reproduce itself.

Luke L. Lairson, assistant professor of chemistry at TSRI and principal investigator of cell biology at the California Institute for Biomedical Research remarked, “In addition to discovering an inhibitor that targets cancer metabolism, we also now have a tool to help answer interesting questions about serine metabolism.”

What does this mean for cancer patients in the future?

Discovering an enzyme that inhibits serine production means that a key process in cancer cell proliferation can be slowed down or even stopped.   Interfering with cancer cell metabolism could be a pathway to treatment. Potentially, adding the molecule PHGDH to cancer cells disturbs the basic need of cancer cells to divide and reproduce rapidly. Obviously this finding points to years of further research and drug development. But discovering this key relationship between serine over-production and a molecule that slows it down could be a model for new cancer treatments in the future.

 

References:

http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3989988/

http://medicalxpress.com/news/2016-03-team-approach-curbing-cancer-cell.html

Persevering Through Clinical Trials

Andrew Schorr talks with patient advocate, Voncille “Peppe” Fryou about her journey with cancer and clinical trials. She begins by explaining that after 10 years without an accurate diagnosis, Peppe finally found out why she was tired all the time…Primary Myelofibrosis (PMF). To beat this disease, she has been enrolled in four different clinical trials, and is now currently participating in the PRM-151 trial. One thing she has learned from her experience is that patients need to be active participants in their studies. Watch the video below for Peppe’s full story and advice on clinical trials.

Persevering Through Clinical Trials from Patient Empowerment Network on Vimeo.

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Andrew:    

Peppe, you’re from where in Louisiana?

Peppe:    

Lafayette, Louisiana, which is on the Gulf Coast.

Andrew:     

And you were diagnosed with primary myelofibrosis just like me right off the bat.

Peppe:

Um-hmm.

Andrew:     

How did it present itself?

Peppe:   

I was tired all the time. For years and years, I was tired. And we did all sorts of tests, and they would come back negative. The only thing they ever came back with was Epstein Barr virus, which they said I had chronic fatigue syndrome.

Andrew:  

So you went how many years before you got an accurate diagnosis?

Peppe:  

Let’s see, probably 10 years.

Andrew:

So it can happen. So you have this diagnosis, and you were very sick.

Peppe:

Yeah.

Andrew:

You eventually got over here to MD Anderson, Dr. Verstovsek. And you’ve been in a number of clinical trials.

Peppe:

Yes.

Andrew:

And you’ve had highs and lows with those. It’s not always been perfect at all.

Peppe:

Exactly. Sometimes, particularly me, I started off well, but didn’t end out very well. I sort of started off good, and then I just started going downhill. And so Dr. V says we’re going to try something else. We’re going to try something else. Are you for that? Yeah. We made a pact. He said as long as he’s being a doctor that I have to stay here and be his patient. So I stayed on his butt all the time. You got to keep me alive, doc. Get busy. And he did.

Andrew:

So you’re on one of these trials we mentioned, the PRM-151 trial.

Peppe:     

Yes.

Andrew:  

What does that involve?

Peppe:     

I get an IV. I go through my clinicals. I get an IV. And I go home. No side effects, nothing.

Andrew: 

Is this an infused medicine?

Peppe:  

Yes. Several years ago, I had a port put in because of some of the medications that would make my counts drop. I needed blood transfusions. And the vein thing got old real fast because it was years I needed the transfusions. So I had a port put in. The doctor did a botched job with that. I had another port put in. He did a terrible job with that. So I came to MD Anderson and said put me a port in. And now, I have an excellent port, and everything works fine. And it’s just hook me up, and –

Andrew:    

One last thing is so you’ve had quite an adventure with myelofibrosis. What’s your view of today and the future right now?

Peppe:     

Oh, we’ve come so far. I mean, like I said, this is my fourth study. Plus, I was on the medication Jakafi. It really wasn’t – he may disagree with me. It really wasn’t a good drug for me. And I put on 50 pounds. And I’m just too little for that. But I’ve gotten that weight off. But I think that it’s important that patients stay active in the studies. I’ll tell you why I agreed to team up with Dr. V. I had a friend years ago that had Hep C. And she got in on a study someplace in New Orleans, Tulane or some place. And she stayed on that study through hell and back. She fell asleep at her desk. But if she missed days of work, they were going to back off on her medication. And her best survival rate was taking it full force. So she did it. She did it. She’s still alive today. And so that’s my hope and my dream for all of us is that our experts are going to find what it is that we need to be cured or control it.

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Barriers to Clinical Trial Accrual

Interview With Dr. Michael Thompson (@MTMDPhD), Medical Director, Early Phase Cancer Research Program, University of Wisconsin

9 1/2 year CLL patient Carol Preston interviews Dr. Michael Thompson about some of the barriers to clinical trial accrual and how healthcare professionals might overcome them. Today, there is only about 3-5% of adult patients enrolled in clinical trials versus the 60% in pediatrics. Why is that? Watch the video below to find out.

Barriers to Clinical Trial Accrual from Patient Empowerment Network on Vimeo.

Improving Clinical Trial Design

Interview With Dr. Michael Thompson (@MTMDPhD), Medical Director, Early Phase Cancer Research Program, University of Wisconsin

Carol Preston’s interview with Dr. Michael Thompson discusses issues related to clinical trial design. The obstacles to good patient-centric clinical trial design are numerous. The informed consent is a text-only legal-type document containing difficult to understand verbiage. Instead, it should be more patient-friendly, with images, graphics and easy-to-understand instructions.

Patients are becoming more empowered and more aware of the issues and are speaking out and willing to participate to make clinical trial design more patient-friendly and effective. More patients are aware of trials and want to educate and inform others. Clinical trial design and informed consent need to be updated and made more efficient so that more patients will be able to easily consider clinical trial enrollment.

Check out the full conversation and the rest of Dr. Thompson’s ideas in the video below:

Improving Clinical Trial Design from Patient Empowerment Network on Vimeo.

Why So Few Adults in Clinical Trials?

Interview With Dr. Michael Thompson (@MTMDPhD), Medical Director, Early Phase Cancer Research Program, University of Wisconsin

In Carol Preston’s interview with Dr. Michael Thompson, he states that about 60% of children participate in clinical trials, but only 3-5% of adults do. So, the question becomes, why are there so few adults participating in clinical trials? What are the adults afraid of? Dr. Thompson goes through some of the reasons why adults do not participate as much in clinical trials as much as children:

  • Patients do not qualify for the available trial
  • Patients believe trials require more testing, resulting in more travel and higher costs
  • Patients think trials may require too much effort on their part
  • Many patients distrust clinical trials
  • Patients don’t believe in the drugs
  • Many patients are not even aware of available clinical trials
  • Patients are afraid of receiving a placebo
  • Patients are afraid of having adverse side effects from the medication

Check out the full video below as Dr. Thompson further discusses this topic and how trial enrollment statistics could change through patient education and engagement. If cancer patients are informed about what clinical trials could offer them, or if they are able to ask questions, more adult patients may be more willing to participant in these trials.

Why are Patients Afraid of Enrolling in Clinical Trials- from Patient Empowerment Network on Vimeo.

Current and Future Treatment Options for Polycythemia Vera

In this video clip from The Conversation: Polycythemia Vera, the participants discuss current and future treatment options. Dr. Verstovsek, Dr. Heaney, and Bob Rosen all agree that the future holds promise for PV. Research is increasing and new treatment options are on the horizon.

Current and Future Treatment Options for PV from Patient Empowerment Network on Vimeo.

This site provides general information. Please talk to your own doctor to make healthcare decisions that are right for you.

The Importance of Self-Advocacy

Interview of V.K. Gadi, MD, PhD Associate Member, Clinical Research Division, Fred Hutchinson Cancer Research Center

Dr. Gadi is interviewed on the importance of self-advocacy by cancer patients. He explains that historically, the doctor/patient relationship has been paternalistic, but such is not the case anymore. Now, Dr. Gadi learns just as much from his patients as from other sources. When patients are empowered with knowledge about their disease, they will be better equipped to carry on an intelligent conversation with their medical team and better understand the rationale for their treatment plan.

Dr. Gadi encourages patients to learn and to self-advocate in order to better understand their treatment options and help choose the best care available to them.

The Importance of Patient Self-Advocacy from Patient Empowerment Network on Vimeo.