MPNforum Magazine

Clinical trial – Why we have no drugs

Zhen

The clinical trial.  For a very few of us, it’s an option. For most, it’s a  desperate, last-ditch attempt with hopes for relief, a cure or remission. .

Headed into the Summer, after the big  US and European hematology conferences, we can see the naked truth.

We have no good drug options beyond, 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 and hope for relief prevents many of us from considering why we don’t have good drugs.  All those reports from hematology conferences on “promising” clinical trials?    Where are the drugs?

No drugs. That’s the inevitable result of a clinical trial system that demands hundreds of millions of dollars and several years of trial and error to test a single blood cancer drug with uncertain outcomes. No brilliant graduate student, no small biotech need apply. This clinical trial game is for the big dogs only.

Consider this.  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 use 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 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 clinical trial principal investigators featured in this 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.

Experience-based medicine – the feel-good term to justify the billions spent in the clinical trial industry – is little more than trial and error wedded to statistics and money. Money is the bloodstream of the clinical trial.   We belly up to take our shots, our pills, our infusions in hope of relief.  The trial and error takes place in our bodies while capital flows into the clinical trial in hopes of profits flowing into corporate accounts.

Our experience-based judgment is this clinical trial system is lousy at producing new, effective meds for MPN patients and impressive at generating revenues and profits for the medical-academic complex.

 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.