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Huntington's Disease Drug Works
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THOUGHTS ON CLINICAL TRIALS IN HUNTINGTON'S

A CORRECTION

I was wrong and wish to correct a statement in my recent Hereditary Disease Foundation update written for the HD Lighthouse. I said that the Huntington Study Group (HSG) had "outlawed" clinical trial situations other than the placebo-controlled trial. The correct statement is that they support "controlled" trials which is later defined (clinical trial section) as placebo-controlled (phase III) trials. I believe it is fair to say that they are not considering any other type of clinical trial. But my descriptive phrase "outlawed" was my frustrated exaggeration.

WHY FRUSTRATED?

I'm frustrated because the Hereditary Disease Foundation conference underscored just how remarkably wide the gap is between extraordinary science and anything useful for HD people. Research has identified a multitude of molecular targets and scores of drug candidates. And more come on each list every week.

But so far, there has not been a single treatment identified. That's really why I'm frustrated.

THE CLINICAL TRIAL SITUATION

The only HD clinical trials approaching success for HD are being done by drug companies: Prestwick is seeking FDA approval for Tetrabenazine and Amarin for Miraxion. These clinical trials situations are delivering.

However, the Huntington Study Group (HSG) trials aren't working as well. Despite its extensive network (over 250 clinical researchers and 70 academic centers), no efficacy trials for people are even started. They are in the planning stages.

So far HSG trials have shown that creatine and minocycline are safe for HD people in the short term. Because the short term safety profiles of these drugs were established decades ago, from the HD patient perspective we have learned no more than we knew five years ago: Creatine and minocycline help the mouse; and are safe in people, at least for the short-term.

I am being hard on HSG because their clinical trial system is so slow: They are great at research, but they are slow in getting this great research to people through their clinical trial system. I mean no disrespect to these scientists: But I think it is fair to say that there is something terribly inefficient about this system that has more clinical researchers and more drug candidates, than HD people in therapeutic clinical trials.

HSG ALTERNATIVE?

I may be "burned at the stake" for saying it, but from the HD patient-advocate perspective, HSG's clinical trial system isn't working. Perhaps it can be fixed; perhaps it will get fired up. But meanwhile this generation waits.

Should we consider another approach to the HD clinical trial? One that could be utilized (in addition to the HSG academic clinical trial) for those candidate drugs that have no pharmaceutical company sponsor?

HERETIC SUGGESTION

Might it be time to create a non-profit professional HD Clinical Trial Company, (advised by HD scientists and patient advocates) whose full-time and only job is to run efficient HD clinical trials? HSG academic clinicians are smart people, and should continue with HSG trials. But because they have a multitude of other responsibilities: clinical practice, research, teaching, clinical trials are often not top priority.

Isn't it a good time for a system whose only priority is clinical trials in HD?

Time could be saved in a system based on a clinical trial "company" that took on the work of clinical trials for HD full-time. Further, such a company could be required to report clinical trial results more efficiently. It isn't in the best interest of HD people to depend solely on the HSG system that can take two years between trial completion and reporting of trial results (as occurred in the HSG minocycline safety trial).

Money could be saved by a private clinical trial company because all money spent would go directly for support of clinical trials. Academic institutions take 50% or more of grant money for "institutional" overhead, so only 50% granted money actually goes to the clinical trial.

Time and money could be saved by a clinical trial company that combined safety and efficacy phases of clinical trials; as has been proposed by the FDA. Time and money would be saved in the Institutional Review Board (IRB) approval process. IRB approval must be done before any and every clinical trial with people. Academic multi-center trials require separate IRB approvals from each participating institution for the same trial. Take a guess at how long that process takes. A private clinical trial company needs only one institutional review board approval. A single approval could include several agents.

ALTERNATIVE?

Imagine a professional HD clinical trial company: One that would do clinical trials for HD full-time, and one that could form a "joint venture" with "for profit" drug companies. Many pharmaceutical companies are unwilling to support HD clinical trials, but would likely be willing to pursue FDA approval if such trials were done "for" them. In such situations, there would be pre-negotiated profit sharing for positive outcomes. Any such profit acquired would be returned to the company for the support of further clinical trials.

Of course, there is no perfect clinical trial or clinical trial system. But might this (or similar) model be a good addition to the HD clinical trial situation?