Science & Medicine

Summer, 2017


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Is there a Stem Cell Transplant in your future?... Everything you ever wanted to know about stem cell transplants. With yet another MPN drug bombing this month (see story in TSR) our attention once again turns to our only curative therapy: stem cell transplant  (SCT). Who’s a candidate? What does  it takes to come out the other side cured?  What are the physical, emotional and financial costs?  What are the odds of success?  

Six survivors and an MF patient waiting in the wings — CHRIS, DOLF, MARINA, ANGELA, BEATRICE, MARTY and JEREMY  —  weigh in with front-line SCT experience… Plus top docs spell out the risk/benefits of transplant…and How to know when it’s time to pull the trigger. (Full story here.

Mesa to Texas… Dr. Ruben Mesa to head up a University of Texas Cancer Center, maintain ties to patients and the MPN community..His letter to the MPN community is here.


See Jane run .. Jane Frantz’ journey from ET to MF to remission. “It was with hope, some doubt and a sense of adventure that I had my first acupuncture appointment.  Dr. Yingzhe Li asked me many questions about why I was there; as usual I had to explain a little about myelofibrosis. Then she looked at my tongue, felt my pulse and went to work,,, “(read more)


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Silver – When to treat with Interferon…molecular study yields compelling data

It was a small trial, 16 women and 14 men, but it will have a big impact.   The result of the study, “The Effect of Initial Molecular Profile on Response to Recombinant Interferon-a Treatment in Early Myelofibrosis will be published  in  the upcoming  American Cancer Society’s journal, Cancer.  Lead author is interferon pioneer Dr. Richard T. Silver with an international crew of co-investigators. (Dr. Hans Hasselbalch appended a detailed analysis of the report in the issue, Dr. Nick Cross is one of the co-authors.)  

Data from the study adds another strong molecular-based argument in favor of early use of interferon in treating MPN patients. “The use of low-dose rIFNa in “early” PMF, post-ET MF,and post-PV MF…resulted in bone marrow reversion, regression of splenomegaly, and disease stabilization with tolerable toxicity in 73% of patients.”  The cohort —  median treatment duration 5.6 years —  reflected the early MPN population: median age, 58 years, 22 were classified as low risk, and 8 were classified as intermediate-1 risk.  The takeaway message:  Molecular profiling at the time of diagnosis may predict prognosis and treatment response.

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 Contest: Name that project. Meds for MPN fatigue? …  Drugs and alternatives for MPN fatigue? … Sick and tired of being sick and tired.  Can you help Dr. Ruben Mesa and his team name the combined scientific effort that is moving the MPN Fatigue Project  to a full clinical trial of pharmacologic and alternative treatments?  Ruben’s Raiders — a large group of scientists, specialists and patient advocates —  are driving this new study. Currently they are seeking funding from the federal Patient-Centered Outcomes Research Institute (PCORI). And for that, they need a name. Any ideas?   Make a bit of history and email your candidate names to directly to Dr. Holly Geyer and the Scottsdale team here.

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 Can we finally lay momelotinib to rest?  It looks like Gilead won’t get another shot a fielding an overpriced drug in the MPN marketplace.  A Phase 2 study of momelotinib that demonstrated not quite enough efficacy in patients with primary and secondary myelofibrosis to move it along the drug pipeline got another shot at life. Could this well traveled Jak inhibitor have a role in treating PV and ET?  A Phase 2, open-label, randomized study in the capable hands of Srdan Verstovsek, Ruben Mesa,  and other luminaries concluded with disappointing results: “… momelotinib treatment has very limited activity as therapy for PV or ET.” Could be why Gilead isn’t taking the drug into Phase 3 MF trial.   The full report will be published in Leukemia Research

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 Genetic factors in the rise and treatment of MPNs…  Newly published in the New England Journal of Medicine, Dr. Jerry Spivak traces key mutations and therapies in the new era of gene expression profiling. “There are two challenges in future therapy for myeloproliferative neoplasms: accurate genetic, as opposed to phenotypic, identification of patients at risk for disease transformation, and eradication of neoplastic hematopoietic stem cells to prevent leukemic transformation. With regard to both challenges, since few oncogenes are recurrently mutated in these disorders and other mechanisms, including cytogenetic and epigenetic abnormalities, are involved in transformation, gene-expression profiling is likely to be the most informative approach for defining risk and identifying molecular pathways for targeted therapy.”

As Dr. Michael Goldstein observes, “This is a very dense detailed update, focusing on genetic factors in the development and course of MPNs. The entire article reads like an synopsis. It is light on treatment recommendations, however. Dr. Spivak is cautious when it comes to pharmacologic interventions, citing the harms associated with “chemotherapy” of MPNs (e.g, HU). Genetic profiling may eventually lead to to more precise application of pharmacologic, genomic and immunologic therapies. The bottom line – be patient….MPNs are chronic conditions and future advances are around the corner. ” The NEJM has made available access to full article... Simply go to the site, register for a 14 day free trial and navigate to the current issue.

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In case you haven’t kept up with fast-moving developments at MPN Advocacy and Education, head for their web page for an eye-opening refresher course. The big news is their expanded high-octane, low cost seminar events now stretching Coast-to-Coast and into Australia.  Closer to home, here’s the Fall Schedule:  September 29 – Los Angeles, CA/Woman&MPN October 26 – Atlanta, GA…  November 10 – Washington, DC   And if you missed some recent events there are a couple of videos worth watching.  Check it out here.


June, 2017 UpdateList of  262 Patient-Recommended Hematologists from 20 Nations

The MPNclinics – 302 answers to 141 Patient questions… with Index

THE ARCHIVES:  A Visual Catalog of MPNforum Articles. ..

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