Dimebon is by far the best news of this summer. Positive reports from Medivation Inc. and the Huntington Study Group (HSG) from their Phase 2 trial in Huntington's are encouraging. Indeed if degree of benefit seen in this Huntington's trial approaches that seen in Alzheimer's, this could be a very big advance.

In this article, we discuss results for both reported trials, and speculate on how soon this drug might reach people if further Phase 3 trials in the U.S. confirm effectiveness. We further suggest that a successful Dimebon trial could also lead to advances in biomarker discovery and validation.

The Genetic Information Nondiscrimination Act (GINA) was signed into Federal law on May 21, 2008, a full 17 years after the legislation was introduced. When more than a year from now it is enforceable and finally trickles down to Huntington's people, GINA will help prevent discrimination by employers and medical insurers -- but only for those without symptoms, and within certain limitations. It is important to know what GINA does --and does not do.

In a new publication from pharmaceutical giant Medtronics Inc., scientists describe a technique for RNA interference that decreases mutant huntingtin protein by 80% without impairing normal protein production. In theory, if this type of technique could be employed for all Huntington's individuals it might be an improvement on the ISIS Pharmaceuticals Inc. product which also can decrease mutant huntingtin production by 80%; but at least (as so far reported) also has the potentially unwanted side effect of decreasing normal huntingtin production.

Participation in good clinical studies and trials is the only way to get treatments for Huntington's disease. And efficient enrollment is the only way to get these treatments to Huntington people more quickly. HDTrials.org is a web effort intended to speed and improve communication about clinical trials for our community. By signing up on this site you can receive broadcasts and information about clinical trials in various geographic areas as soon as they begin. Thousands of participants will be needed. We have the opportunity and the power, through this and other efforts to speed enrollment in trials that can bring treatments to our families more quickly.

Recruiting has begun for 2CARE, a large PHASE 3 trial testing Coenzyme Q-10 in Huntington's disease. At its conclusion we will hopefully learn a definitive answer whether high doses of this supplement can slow the progression of Huntington's. The Huntington Study Group is recruiting for more than 600 participants for this trial that is slated to last as long as five years, but will be stopped earlier if benefit is shown during a shorter time.

On a less positive front, several recent trials of Coenzyme Q-10 for Parkinson's have not confirmed the benefit that an earlier trial had suggested . .

With his permission, I gratefully post an interview by my good friend Gene Veritas with Dr. C. Frank Bennett, Senior Vice President of Research at Isis Pharmaceutical. If all goes as planned, Dr. Bennett predicts 2010 as the date for first phase human clinical trials for Huntington's.

It sounds possible -- I can hope he's right.

Updated May 8, 2008

The European Huntington Disease Network (EHDN) has begun recruiting for a PHASE 3 study of ACR-16 in Europe that will involve about 420 patients. This clinical trial will test 2 different dosages of the drug and last for 6 months. We are more than a bit behind. Start dates for the U.S. counterpart, an earlier PHASE 2 trial that will last for 3 months have not been announced.

EHDN science writer Dr. Diana Raffelsbauer's interview with Dr. Arvid Carlsson, the nobel laureate who discovered the molecule is presented in EHDN's inaugural newsletter.

Dimebon is looking good for Alzheimer's (AD). A 2nd AD trial (this one a PHASE 3) confirmed benefit seen in an earlier PHASE 2 trial. Benefits in memory, thinking, and behavior persisted during the year of treatment; in fact 81% of of AD participants were better than when they started the study. Based on these results Medivation is planning a larger PHASE 3 trial later this year. If results are positive, the company aims to start the FDA approval process in 2010 and the drug could move to patients shortly after. What about Huntington's; why was this trial slow to recruit?

NBC correspondent Charles Sabine was an important highlight from the February 2008 CHDI conference , setting the tone for this meeting with his powerful opening presentation. Mr. Sabine-- who is known for his decades of news coverage in countries torn apart by war and disaster -- came out of the Huntington's closet. He spoke of his family's experience with the disease, and later about his own gene positive test. In his presentation he first offered heartfelt gratitude to all those who work for Huntington's; but he had other messages for CHDI and the assembled crowd: His was an eloquent plea to give hope and dignity to all families who sufffer from Huntington's. His video presentation from the HSDA website is linked below.

CHDI Inc. will be hosting the 3rd annual Huntington Disease Therapeutics Conference on February 4-7 in Palm Springs, California. Though they have have graciously extended an invitiation to Huntington families in the local area, this conference is quite technical, fast-paced, and intended mainly for researchers and drug company officials. If you would like to come, please contact Jerry Turner at This email address is being protected from spambots. You need JavaScript enabled to view it. so that CHDI can expect you as a visitor. The final day will be more relevant for Huntington families.