International MPN News, Science & Opinion

Spring, 2016 — An Introduction

An introduction to the Spring 2016 issue of MPNforum 

 Why we have no good new drugs… and what we can do about it right now.

Zhen

Zhenya Senyak, Editor

The FDA’s recent abortion of an MPN clinical trial focused our attention both on the Agency and the clinical trial process it monitors.  What we discovered was a badly broken system that affects our health.  We have options to make things much better.  In this issue you’ll find….

 The FDA Report Card, a record of dismal MPN performance…

Clinical trials, and how money and power make them dangerous to our health…

HikeMF, a major fundraiser for our Foundation takes off again…

The Small Donation Program, this time funding the New MPNforum as we expand into direct action…

Posters of the Year Past…and Letters to the Editor, On Medicare failing to cover a SCT and what to do about it …On How it feels to be be booted off a clinical trial. 

(A link to the  MPNforum issue  is at the bottom of this page…or just skip the intro  go here.)

What our main stories have in common? Money and Power.  Theirs…and ours.

Theirs:  Last week CTI refused FDA’s offer to share data on its pacritinib clinical hold because “Publicly disclosing unblinded data could jeopardise a $30-$40 million study.”…Then there’s the new FDA chief, Dr. Robert Califf, confirmed by Congress last week.  A respected scientist, he was co-director of the tainted Xaralto clinical trial under investigation by the FDA . While at Duke he claimed “no more than half his salary” was paid by Big Pharma including Janssen,  the company that would eventually market Xaralto, a drug  that racked up $1.5+ billion in sales last year and is defending against multiple related class action lawsuits.

Ours:  Julie Libon raised $25,000 for the MPN Research Foundation last year and has organized another walk in the woods to beat that total, taking us along. This is clean, unattached money, an exercise in patient power. No special interest, no conflict of interest, just patients ponying up a few bucks to help fund independent basic scientific research.

Why the New MPNforum

This is the last issue of the old MPNforum Magazine. New focus, redesigned format coming up. Five years ago we started MPNforum to provide a window through which patients and caregivers could see each other and our doctors.  A place to report news, exchange opinions, views, share concerns. Back  in the dark ages of 2011 our world was mostly black and white, emails and bulletin boards.   Our doctors were just names and not faces.  We needed MPNforum to see the landscape, to meet each other.

And we did.  A half million of us came together to read MPNforum stories, comments, reports produced by hundreds of patients and caregivers. 

And then a strange thing happened, totally unexpected.

We couldn’t help but notice the absence of patient participation in scientific and regulatory circles. Patients and caregivers were missing from major MPN medical meetings.   Along with scientific breakthroughs, MPNforum found irregularities in research designs, extreme claims for drug effectiveness, rapacious drug pricing,  broken clinical trials that harmed us without explanation from drug company sponsors, the FDA, or our own physicians.

MPNforum, originally a social project, rapidly became a political and investigative journalism enterprise.

We felt a terrible lack of effective drugs, feeling the pangs of MPNs, watching friends suffer and die,  while all around us we saw a noisy, expensive feeding frenzy as a billion dollar look-alike derby of JAK inhibitors yapped at the heels of Incyte’s grossly overpriced Jakafi.

Over all our MPN village looms the shadow of a green and greasy storm-cloud of money – billions in dollars, pounds and euros corrupting science, the economy, political processes and medical care alike… even as Big Pharma masks its commercial motives in a benevolent, caring corporate face.

It hasn’t been all gloom and doom. The MPN Research Foundation expanded its investments in game changing research, funding major discoveries and exploring new possibilities. Dr. Richard Silver and his colleagues in Europe, mostly, carried the torch for off label interferon , the only demonstrably effective medicine to control MPNs in appropriate circumstances.  Dr. Ruben Mesa took up the MPN fatigue cause and coordinated multiple online research efforts to find solutions.  Among her other support activities, Dr. Claire Harrison runs open meetings and workshops for patients and caregivers. Robert Rosen, Chris Harper, Bonnie Evans, Diane Blackstock, Antje Hjerpe, Marina Peed and many other patients volunteer time and energy to support other patients.

But without money, without power we have not been able to advance the progress of an MPN  cure. Even the Foundation’s $11 million in research grants  pales before Big Pharma’s multi-billion dollar investment in hopeless clinical trials of lookalike MPN drugs.  Worse, those drug billions are poured into medical institutions paying a good portion of the salaries and overhead of the very people caring for us.

That is the genesis of the New MPNforum. We need Direct Action. Public attention. Access to legislators. 

We need more than show and tell.  We need to act to bring about change.   Who else can we look to? It is only us, the MPN patients and caregivers who can speak out, who can act, effectively. We need to reach legislators, media, the public. We need to monitor the regulators, the drug companies. We need to support scientists, researchers, and each other. We need to lobby and raise real funds. And we need to do it now.  

finger pointing small  To get involved send an email to ourMPNforum@gmail.com and simply type  MPN Action as the Subject.  Together, we’ll figure it out.

Together, we can be a powerful force for a cure.

What alternative do we have? If  we do not speak out for those who cannot, if we do not act for those too sick to act, who shall do it for us?

The full MPNforum 2016 Spring Issue is here.

Comments on: "Spring, 2016 — An Introduction" (1)

  1. Bryan Cronk said:

    Like it or not, my wife Linda might not / would not, be here today without (MPN) Clinical Trials. After being given 27 months unless there was treatment, there were 30 successful months on a Momelotinib / CYT387 Trial followed by 28 successful months, so far, on Ruxolitinib / Jakafi / Jakavi which was approved by Health Canada literally a week or two after Momelotinib stopped working. Then there are the Clinical Trials for Anti Epilepsy drugs leading to Linda’s off label use of Topiramate for several years now to control very severe migraine headaches, and the Clinical Trials for Hepatitis C and B and the off label use by Linda of Pegasys which has controlled her blasts and who knows what else since July 2014. Then there is Eprex / Procrit (red blood booster), Losartan (heart problems) which were approved through Clinical Trials which may be On Label or Off Label, not sure etc. Does not mean there is not room for improvement, and I can name a few, but it may not take a revolution.

    Many MPN patients, by the nature of the disease with every patient being different, will run out of all other lower risk options other than participating in a Clinical Trial (and yes some may decide, for whatever reason, that Clinical Trials are less risky than a Stem Cell Transplant), so let’s not take that away from them, through creating doubts and raising fears.

    Bryan Cronk

    Take care, Bryan & Linda, Barrie Ontario Canada, Linda is 68 yrs – Post ET(2004) – MF(High Risk / Advanced 2011 Feb), CYT387 2011-2013(failed), Jakavi Nov 2013, Pegasys July 2014, Jakavi 20+20 mg daily(spleen dose) + Pegasys 90 mcg weekly(High WBC dose) + Eprex 40,000 IU weekly(Mar 2015 4-10 weeks between transfusions due to Jakavi dose changes for spleen vs. 3-4 weeks) + Hydroxyurea 1,000 g daily(Lower WBC-down 40 points the last 3 weeks, Shrink spleen?) + Allopurinol

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