MPNforum Magazine

No drugs. No surprise.

Zhen

Clinical trials:  For a very few of us, it’s an option.  For most, it’s a last-ditch attempt at relief, remission or a cure. ..

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? Looking to clinical trials for the emergence of a Magic Pill?

Clinical trials may well be the gold standard for experience-based medicine. But our experience-based conclusions are this multi-billion dollar industry urgently requires change.

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.