No drugs. No surprise.
For a very few of us, it happens in the beginning. For most, it’s a final option, an endpoint with hopes for a fresh start.
That’s what clinical trial and stem cell transplant have in common.
Headed into the Summer, now that reports are available from the big US and European hematology conferences we can see the naked truth.
We have no good drug options beyond, possibly for some, the interferons.
No FDA approved drugs at all except Jakafi for symptomatic relief of high risk myelofibrosis. Our basic meds are all off label. Their MPN use is not indicated on the professional label. Hydroxyurea, interferon…off label.
Pain or distress prevents many of us from considering why we don’t have good drugs.
Simple cause and effect. All those reports from hematology conferences on clinical trials, complete responses, promising results, etc. and the lack of meds to relieve and cure our myeloproliferative neoplasms are directly related.
No drugs. That’s the inevitable result of a clinical trial system that demands nearly $1 billion and several years of trial and error, testing a single blood cancer drug with uncertain outcomes. No brilliant graduate student, no small biotech need apply. This game is for the big dogs only.
For the past five years, while we suffer and die, drug companies have been chasing the same damned JAK2 inhibitor with lookalike drugs, clogging drug development channels and squandering resources – billions of dollars that might otherwise have been spent on real scientific exploration.
There is an answer. Until this ancient, flawed clinical trial system gets reformed, until genetics, information technology and biotechnology are wedded in a new medical research paradigm, we can take evasive action.\\
We can embrace a healthy lifestyle and off label drugs to the extent possible. We can avoid all dosage and toxicity trials if possible. We can’t afford the time or impact on our bodies. And we can determine to enter late stage trials only with some definite and specific anticipation of benefit. We are patients, not lab mice. Choking off the supply of willing subjects — if possible — is one way to bring about change.
Guiding us in this effort are our personal hematologists and trusted MPN specialists. Doctors Ruben Mesa, Claire Harrison, Serge Verstovsek – all featured in this Summer issue of the Forum — are workers in the tangled obscure vineyard of MPN. They are our allies and worthy of trust and respect.
As principal investigators into new drugs, however, they are also beneficiaries of funds from Novartis/Incyte and other drug companies.
We can trust them to report objectively and honestly about drugs produced by Novartis/Incyte and others. We can’t expect them to publicly criticize the companies or the clinical trial process in which they are embedded.
But we can. We can look at this stuff with an open mind, open heart and clear head, share our findings with each other, criticize when we must, and act together to change things.
Aren’t you sick and tired of being sick and tired?
Coming up TOMORROW in the Summertime MPNforum… Reviews of the PRM-151 and Ruxo/PV trials…Preview of the MQJ SCT discussion by panel of transplant experts with intro by David Steensma and Claire Harrison, perspective by Chris Harper. Stories from patients emerging from the clinical trial and SCT experiences, Harvey Gould, five days post transplant tells all…Voncille, Patient F in the PRM-151 trial tells how it is….and a major expansion of the List of Hematologists (thank you Ellen Jacquardt). See you all tomorrow.
Dr. Ruben Mesa needs us to step up and take his survey. We now have assurances of privacy and an arm’s length relationship with sponsor Incyte. Our reservations about Incyte’s use of marketing data from the survey are trumped by Mesa’s need for these data to complete his Fatigue research. Our fatigue research.
Think about it. No one has done more for us than Ruben Mesa, investigating MPNs, teaching, treating patients, counseling, We owe him a debt of gratitude and we owe him this one. Please. Click here to go to the survey. Thank you.
Comments on: "TSR – Summertime, 2014" (5)
Hi there I would have done the survey but when I went into the link it’s for US only sorry I’m in Canada. Just thought I would let you know that is why I am unable to participate. thank you
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Does anyone know who is behind the survey at http://www.mpnsurvey.com? I found out about it when I saw a display ad in the New York Times a few weeks ago. It is being administered by “ICF International.” When I emailed and asked who they were working for, and why, they would not say.
Hi, Leif…I had the same questions. Incyte is sponsoring the research which set off every alarm bell. ICF is distant corporate number cruncher far as I can tell from its website. I talked to Dr. Mesa who double checked the security of the study. While Incyte may well be interested in this survey for its marketing purposes, the over-riding issue for me is Ruben Mesa needs it for his research. I can understand — and share — your reluctance considering the covert presentation, but I think that was just an error. Ruben Mesa’s word is good enough for me. Thanks for the comment. We need to question everything.
Interestingly, even though when I first emailed they would not tell me the sponsor, once I actually started the survey a message came up saying it was Incyte.