In a recent article by Gene Veritas at curehdblogspot.org, Jonathan Monkemeyer suggested that "advocates must actively participate in the search for treatments, perhaps even trying drugs and substances approved for other purposes in their own off-label studies, seeking advice from researchers on dosing, and having people reporting their observations via a website". While I agree with the first part of the statement, I have some concerns about the remainder.
No question that we need strong HD family advocates like Jonathan to increase HD awareness and bring new ideas. However . .
It is problematic to suggest that HD individuals ask their doctors to prescribe off-label drugs solely for the purpose of running their own studies. What does off-label mean? This is the prescribing of FDA-approved drugs for unapproved indications; for example the use of an antipsychotic like Zyprexa® to treat chorea in HD. In fact Xenazine® is the only FDA approved drug specifically for use in HD. There is no question that off-label prescribing is vitally important in the treatment of symptoms.
What about patient websites for off-label drug study? This is an idea already brought to practice. PatientsLikeMe is an example. This successful "for profit" social networking website launched in 2006 for patients with Amyotrophic Lateral Sclerosis (ALS). Members share health information including drugs they are taking and self-reported measures of breathing capacity (a clinical marker of disease progression for ALS). Using this information, investigators can observe whether patients on certain medications (lithium for example) appear to be doing better than those not on this drug. Note they did this study ONLY after an early phase clinical trial for dose safety in ALS was completed. The site now has many other diseases -- including HD -- added into their system. This site also includes social networking and matching of patients to clinical trials (this is the "for profit" part). However, even this most successful site -- with more than 30,000 patients followed for up to 6 years -- has yet to make a clinically meaningful breakthrough in any disease.
What about HD? Enroll-HD, a scientifically better alternative: Though Enroll has many goals, one is to correlate drug usage for HD symptoms (antipsychotics. etc.) and for other conditions (like statins for cholesterol) with disease progression using expert measures of disease progression. This is a more scientific way to study off-label symptom benefit and relationship to disease progression than the self-reported measures used at PatientsLikeMe. Further, Enroll will have excellent capability to identify and match participants to appropriate clinical studies and trials.
What about self-study of off-label drugs that have potential neuroprotective effect for HD? Why not try available drugs that have shown some benefit in mouse model study? This is an idea similar to the HDDW trials that used finger tapping and cognitive web measures in an observational IRB-approved study of supplements -- those that had had previous safety trials. A reasonable idea -- but no definitive results other than teaching me some of the difficulties in studies for HD.
However, Jonathan's suggestion for testing drugs, with doses based simply on mouse studies is not reasonable, it is hazardous. There are very good reasons to be skeptical about translating mouse studies to people -- even if they are done by our favorite scientists. At least so far, there is no evidence that drug studies in mice will translate to people. And worse there is some evidence that harm can be done. Remember minocycline? A decade ago this FDA approved antibiotic looked like it might be helpful in model mice; but it proved to be harmful in ALS patients. There is no "safe dose" that can be calculated for people from mouse studies, only educated guesses. The reasons to be skeptical about translating mouse studies to people are similar to those used in my recent HDDW article on supplements .
Author's Proactive Advice: Exercise, maintain a healthy diet and sleep cycle, seek adequate treatment for HD symptoms from an HD knowledgeable physician, and run to sign up for Enroll-HD. PatientsLikeMe has the advantage of social networking, and if their numbers of HD members increase (only 63 so far), it may supply some useful information on self-reported benefit of available drugs. The best proactive work for new drugs? Join a clinical trial and/or find and encourage others to do the same. Therein is our patient-power -- and our best hope for speeding the progress of new drugs for HD.