No imagination needed: Huntington's people are living this scene. We know that brain damage is happening, and we know that several promising treatments have passed early tests for safety. We also know that more brain damage will happen during the time it takes for phase 3 clinical trials to be completed and for the FDA approval process to run its course. What's a person to do? Go down with the ship, or try the lifeboat?
Huntington Study Group authors note that only a small minority of PHAROS participants reported taking creatine, coenzyme Q-10, minocycline, or omega 3 fatty acids. The HSG authors suggest that this is an "educated" decision, although it was clear that average doses taken were far below what HSG believes to be potentially therapeutic dosage. What this really suggests is that HSG doctors (on average) aren't educating HD people about the specifics of research findings, so that an informed choice can be made.
Clinical Trial Option: Some will participate in clinical trials. Usually this option is available only for those who fit specific criteria: early to mid stage disease, physically independent, free of psychiatric symptoms, and access to an academic medical center. In early open label trials, most if not all participants will be given active drug. In later phase 3 trials, while the majority will receive active drug, some will receive inactive placebo.
Clinical trials are vitally important for gaining knowledge and getting treatments to Huntington's people. Further, participants have a reasonable chance of getting active drug with potential for personal benefit. And if the trial is successful and short, those who receive placebo will have minimal brain damage before they too, have access to active drug. If the trial is several years long, the placebo participants will suffer greater brain damage.
Lifeboat Option: Available Supplements and Drugs Some will use available lifeboats that have successfully passed first human tests. These include creatine, omega 3 fatty acids, and coenzyme Q-10. Trehalose and SSRI antidepressants probably fall into this category but have not had a "champion" to bring them to clinical trial. Those using these agents know that they don't come with instructions, leaving users on their own to search for clues gained from early test results.
New Drug Options: Dimebon and ACR-16 are experimental new drugs soon to enter clinical trials for Huntington's in the United States. Besides participating in these trials, there may be other options.Compassionate Care Option:For experimental drugs that aren't available to the general public, the FDA permits "treatment use" of drugs still in clinical trial for serious conditions in patients for whom no satisfactory alternative drug is available. Though the present process for applying for such use is difficult, we are told the FDA is working on standardizing the process and encouraging pharmaceutical companies to participate.
Expanded Access Program
Option: This program provides wide access to a drug that has
completed clinical trials but is not yet FDA approved. This is
often used for cancer drugs. Accelerated Conditional Marketing
Option:This program could be useful if you are European. The
European Medicines Agency (EMEA), the counterpart of the FDA in the
U.S., goes a step further and has specific regulations that permit
accelerated conditional marketing approval for treatments of
seriously debilitating diseases. This allows even wider patient
access to drugs still in phase 3 clinical trials.
Cancer and AIDS patients have long used these options to obtain experimental drugs before FDA approval. As more experimental drugs come to clinical trial for Huntington's, it's time to learn more about how these programs can be useful for us.