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How I Advocated for a Clinical Trial

How I Advocated for a Clinical Trial
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Published on March 2, 2020

I am 76 and am living with primary myelofibrosis (PMF). I am retired from a full-time job but enjoy consulting with pharma and medical devices companies and volunteering.

In September 2018, I had a routine checkup with my physician, during which I mentioned a few symptoms of fatigue and weight loss. The doctor ordered some blood tests which all were normal except for being anemic and a high EPO.  As my hemoglobin dropped further, I started having additional symptoms of itching, night sweats and early satiety, in addition to the fatigue. In November 2018, I was scheduled to see a hematologist and had a bone marrow biopsy. The biopsy indicated PMF and showed several of the associated mutations including JAK2-617F. I have two other risk factors that have been associated with this cancer (Ashkenazi Jewish heritage and exposure to benzene). The hematologist suggested watch and wait with no other specific treatment at that time.

UCLA team

Two of my healthcare team at UCLA Medical Center, hematology/Oncology: Nicole Alimi, NP and Bruck Habtemariam, Clinical Research Study Coordinator. They have been fantastic in helping me through the clinical trial!

I decided to have a second opinion at the Huntsman Cancer Center in Salt Lake City and once again confirmed I had PMF. I was told the only “cure” was a bone marrow transplant but given the mortality rate with the procedure, I chose not to pursue it. I conferred with my doctors about therapeutic alternatives and did my own extensive research, through reliable sources, on the internet. The treatments I found were focused on the constitutional symptoms but did not treat the underlying pathology. In January 2019, I found a reference to the MANIFEST trial (Constellation Pharmaceuticals, Inc.) on MPN websites and Clinicaltrials.gov and I brought it to my doctors’ attention. Although only early results were available, it showed the potential to reduce the fibrotic burden and transfusion dependence in some patients. Given my background and attention to detail, I made a spreadsheet of all of my options for therapy and discussed it in detail, and we decided the best option was to try to enroll in this trial. After two months of correspondence and an exhaustive medical evaluation at the UCLA Medical Center, (the closest clinical site to my home in northern Utah), I was accepted for the 24-month trial.

The MANIFEST trial is the only treatment regimen I have tried to date. Since I was ruxolitinib-naïve (Jakafi), I was placed in the cohort to receive that JAK2 inhibitor in dual therapy with the study drug, CPI-0601. To date, I have not had any serious side effects or surgeries except further drops in my hemoglobin and platelets. I started transfusions of packed red cells every 3-1/2 to 4 weeks and after 5 cycles (15 weeks) in the trial my spleen volume was reduced by over 53 percent and all the constitutional symptoms were gone or significantly diminished. Now, I get blood draws one or two times a week to check hemoglobin and platelet levels and now can have the transfusions performed near my home in Logan, Utah.

In order to participate in this trial, I make a 2-3-day trip to UCLA every 3 weeks for tests and to get the next 3-week supply of test drug for the first 15 cycles. Now that I seem to have stabilized, I am on a 6-week cycle to UCLA with additional blood tests done locally.

blood draw gauze

Some of the many gauze strips used after blood draws to check my hemoglobin levels and other clinical values

In September 2019, I participated in the MPN Foundation Sponsored Patient Focused FDA Meeting in Bethesda, MD. It was my first chance to meet other MPN patients. Most were ET and PV sufferers, but I did get to learn a lot from the experience. I have joined the MPN Impact Council and am writing this blog to help others in navigating the complicated course of this disease and the treatment options. As is often stated in Patient Power, just keep asking questions of your healthcare team until you find the right course for your therapy! 

With a PhD in organic chemistry and a career in pharmaceuticals and medical device development, I have a good understanding of the clinical trial process and keep exceedingly careful notes of all of my self-observations. In some ways, I have an “out of body” experience when looking at my own data and analyzing it from the perspective of a researcher rather than a patient. I am hopeful that new treatments and therapies will be developed soon to help in my own treatment.

~Ned Weinshenker


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

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