The Huntington Study Group Annual Meeting for clinical trials took place on the days preceding the symposium. Dr. Marsha Miller has posted an excellent summary of the concluding symposium events on the HDSA website, so my focus is on the HSG meetings that led up to the symposium, and my general impressions of the whole process.

This type of meeting differs from other Huntington's meetings because it focuses solely on clinical trials and projects for people. What was most impressive about these meetings is the sense of renewed clinical trial energy, increasing collaborative effort with members from the European Huntington Disease Network (EHDN) and CHDI, Inc., and the reports of several new drugs in development approaching clinical trial stages. It is clear that there is a lot of old and new clinical trial action.

Clinical Trial Reports:

Working Group Highlights: Working groups are subsets of investigators who work on different research areas that impact clinical trials. There are several groups, which include individual focus on biomarkers, genetic correlates, clinical correlates, clinical trial initiatives, and neuropsychology. By far, the recurring theme in most of the discussions centered on finding best measurements for motor, cognitive, behavioral, and functional components of disease progression that could be used for successful clinical trials.

Education Session Highlights: These discussions focused on several steps in drug development that could heighten the chances of bringing success in HD clinical trials.

Another Highlight: There was significant interest for clinical trials of drugs that address Huntington's symptoms. This arose in the context of two surveys of Huntington doctors (one from HSG, and one from EHDN) about drug usage for the many symptoms of HD. Most notable about these surveys is that they found no consistent pattern of drugs prescribed for chorea, anxiety, irritability, and other psychiatric symptoms even among the experts. This pointed out the huge unmet need for symptom-based clinical trials.

Determination of best drugs for use in each symptom would establish a standard of care that should result in improved care for all Huntington patients. Hopefully discussions begun at these meetings will result in more of this type of clinical trial in the U.S. and Europe.

Summary: I came away from these meetings and the following symposium with optimism for better things to come in 2008. There is renewed enthusiasm for clinical trial efforts, recognition of the needs to make them better, and a push toward greater collaboration in efforts here and abroad. It was also very good to hear news of CHDI drug candidates that are getting closer to testing in people. I can hardly wait for a more detailed CHDI update coming in early February at their annual meeting. It's clear that they are gearing up for clinical trials too.

It has been eight long years from positive studies of creatine and Co Q-10 in model mice to advance to initiation of phase III trials in people. It is clear we need to develop better and more efficient systems to speed the clinical trial process as anticipated new drugs come down the pipeline.