MPNforum Magazine

MAGIC..Myelofibrosis Risk Assessment Tool + The 15 Top MPN Mutations…

Finally we have a simple, powerful tool to convert our basic clinical data into a clear graphic signal to highlight our status . Clinically validated and produced with the help of an international consortium of scientists and hematologists. Pop in your numbers and press the button! MAGIC is free and it’s here.

It’s not your Daddy’s MPN Research Foundation…

A new generation has taken the reins of  the MPN world’s main independent source of research funding. They’re busting out with new projects…and that’s just the beginning. Here are the people and programs of the new MPNRF.

How I treat PV…

“First, do no harm.”  Dr. Jerry Spivak, Johns Hopkins professor and MPN guru , shares his surprising  insights on diagnosing and  treating polycythemia vera. Whatever you think your PV treatment options may be, this paper might expand your perspectives and improve both your quality of life and longevity. You can read it here.

The Santa Syndrome — or The Pharma in the Dell

 I’m naturally cynical. I never believed in Santa Claus and it still shocks me to discover how many of my contemporaries do. Not so much the fat guy in a red suit and big white beard, but belief in finding a gift under the tree if you’ve been good.  A gift placed there by a generous intruder sliding down the chimney. Since this is MPNforum,  I’m talking about myeloproliferative neoplasms.   And the great mass of MPN patients who have been.,,,. (More)

Who pays your Doc?….The 2018 Sunshine data base, the national healthcare transparency program, is now available. The Center for Medicare and Medicaid Services.. has once again assembled and published its tool that permits easy tracking of payments made by drug companies to physicians and institutions.  Both research payments and “general” payments covering consulting, travel, honoraria, speaking fees, meals, lodging, meetingsm etc.. You can type in your doc’s name and get the details here. 

Drug drought and no cure in sight?  Got an MPN?  Dealing with it means getting help managing symptoms, the bread and butter of the massive pharmaceutical industry.  Fundamental scientific research is the job of scientists in labs and universities.  Applied research, producing symptom-relieving drugs that can then be tested and sold to patients, that’s the home territory of corporations.   Understandably, we spend billions on palliation, anything to relieve our symptoms, but only trivial amounts on the hard science that explores our underlying MPN disease.   We reach for relief with the massive encouragement of drug companies,       and that often means JAK inhibitors.  Beyond Incyte’s efforts to extend its reach from MF into the chronic MPNs, the great bulk of MPN clinical trials mounted by Big Pharma  are focused on lookalike Jakafis,  Thousands of MPN patients have been enrolled in dozens of large scale JAK inhibitor trials for the same old short term “fix” without much to show for it.  .If not for the handful of meds available years earlier —  the interferons, hydroxyurea, anagrelide — we would leave the pharmacy empty handed. Meantime, out of Europe,some very cool and fundamental work, was recently published in NEJM and Blood, work that digs deeply into the genomic roots of our MPNs. . No billion dollar payday awaits these scientists. . But for us maybe there is something even more valuable, shining a light on the pathway to a cure.  Like refinement of gene editing and CAR-T targets.   When you get a little free time Google the current work by Vainchenker and Kralovics,  or Grinfeld, Nangala et al., some of which is summarized in Notes on MPN Mutations.

The JAK inhibitor derby is far from dead and gone.  Six years after we reported on the JAK Derby, they’re still at it. There’s Momelotinib (CYT-387).  By any other name this well traveled ancient molecule may well, finally, become available after Cytopia sold it to YM Biosciences which sold it to Gilead which sold it to Sierra Oncology (run by Nick Glover, former YMI CEO).. .And, there’s another contender, resurrected from the junk heap,  Fedratinib, the briefly disgraced drug that was prematurely abandoned by Sanofi — — along with thousands of patients tossed out in the cold with no notice after a “successful” Phase III trial and FDA challenge — is making a comeback after John Hood and Catriona Jamieson picked it up and then flipped it over to Celgene. You can’t make up stuff like this game of musical chairs  . It would be funny if thousands of MPN patients hadn’t put their lives on the line while these corporate manipulators sorted out their best, most profitable, options. What’s the Severe Adverse Effect of delay and corporate shuffling?.

It’s a beginning but did The MPN Patient Bill of Rights get it wrong? There’s a mix of good intentions and missed opportunities in the MPN Patient Bill of Rights produced by a handful of well-meaning patient advocates and nonprofits.  In some respects the new MPN Patient Bill of Rights misses the point…. Even if it had teeth (it doesn’t) none of its provisions would have spared us Sanofi’s brutal boondoggle of a Fedratinib blow-up or revealed Jakafi’s hidden dangers unmasked by the FDA….The “MPN Bill of Rights” basically grants us the right to be informed, to be an educated consumer of medical services.  Yes it does note our rights to privacy, to get a second opinion, the right to seek financial aid, to access information and bring family and friends to consultations.  All good stuff but still  pretty passive.  And.in an outright sour note, the document itself, printing of which is sponsored by Incyte Corporation, also grants us the right to access clinical trials and participate when we’re eligible..

What’s not granted is: Our right to be included as an active and equal participant in exploration of our treatment options… Our right to be alerted to the secret dangers of prescription drugs…Our right to know the hidden risks and personal costs of clinical trial participation…And most desperately needed, the right to have a patient advocate on every MPN clinical trial. (something for which over 1000 of us petitioned the FDA and were granted.)…What the MPN Bill of Rights basically doesn’t grant us is an equal, dignified and respected say in our own treatment,.

List of 300 Patient-Recommended Hematologists from 27 Nations & 47 States

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

Stem cell transplant — Six front-line stories and some vital links.

THE ARCHIVES:  A Visual Catalog of MPNforum Articles.

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