Who can we trust?
As MPN patients, we are vulnerable.
Sometimes in pain or discomfort, always uncertain of future disease progression, we rely for relief on a medical establishment deeply indebted to a few large pharmaceutical companies for research funding. And since the approval of ruxolitinib nine years ago most of that funding has poured into JAK2 inhibitors that offer short term relief but no impact on the course of our MPN. Worse this class of drugs carries a heavy load of side effects subjecting us to a variety of serious health impacts.
We are the subjects of clinical trials of drugs that are heavily promoted… and almost always fail. We are provided powerful drugs by institutions and physicans who are often on the receiving end of substantial funding from corporations making and selling those drugs. The FDA lacks the capacity — and sometimes the will — to rigorously supervise approval of experimental drugs. Virtually all our nonprofit advocate supporters as well as the commercial MPN purveyors of information receive substantial funding from drug companies..
The result: Despite investment of hundreds of millions of dollars, participation by ten thousands of patients in clinical trial, and the best efforts of a small army of researchers, scientists and physicians, since 2011 only two drugs have been FDA approved for MPN treatment, both JAK inhibitors. And the interferons and hydroxyurea — the workhorse drugs we actually rely on — are only available to us off-label.
Interferon, the only drug to have a proven track record of knocking back the mutant clone, while widely accepted in Europe, is available to us only because it’s approved for other diseases… and it is only reluctantly prescribed in the United States. Our workhorse, inexpensive drug, hyroxyurea, is also not FDA approved and frequently passed over in favor of the outrageously priced alternatives. Do read Hans’ story in this issue. His is a voice we can trust.
Who can we trust?
That’s the ultimate question and some surprising answers are addressed in Requiem for a Dream. In Drs. Richard Silver (retired), Hans Hasselbalch, Ruben Mesa, Claire Harrison, Brady Stein and a handful of other specialists we have our Dr. Anthony Fauci. (And if you have not yet read Julie Libon’s amazing article on Dr. Silver, here’s your chance.)
We each need to find our own trusted physician. (If you’re still searching, The List may help.) And in the MPN Research Foundation, even as it reorganizes, we have a tradition of patient support, reliable management, scrupulous Board oversight and passionate employees. Watch their grants and program decisions over this next year to get a feel for its evolution.
With this issue, MPNforum is leaving the house. We talk about that a bit in Requiem. Essentially, our work is done. More than ever it is crucial that MPN patients and caregivers be educated and vigilant. Share hard won knowledge. And support one another. Good luck.
(Note: For answers to your MPN questions please visit our graphic Catalog .)
When it’s time to move on. The Last MPNforum.
The legendary Danish professor shares findings from new MPN clinical studies
The one site you need to check your MPN status instantly.
Along with her friend and partner Robert Rosen, Barbara spearheaded the major MPN science of the past decade
What happens when your MPN hits a speed bump in the road?
Fromthe MPN pioneer: What a long strange trip it has been
The lost trove of 50+ patient recommendations surfaced in time for inclusion in this edition of the Internet’s Most Popular web page
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