RNA interference (RNAi) therapy was an important theme at the 2010 HD Therapeutics Conference. If this type of therapy can be fully mastered by scientists and drug developers it could be very promising because it will treat HD at its source by reducing the amount of defective huntingtin protein that is produced in cells. RNA interference works by introducing into cells short pieces of RNA that recognize and then destroy the means to produce huntingtin protein. While there were many posters detailing research in different types of RNAi, the major presentations centered on small molecule RNAi.

In another exciting presentation at the CHDI meetings, Guy Miller, MD, PhD the CEO of Edison Pharmaceuticals gave information on EPI 743, a compound that has been developed for rare childhood mitochondrial diseases. The Huntington's community first heard about Edison's CoQ10 analogues, which were described as more potent and much more highly absorbed into brain than naturally occurring CoQ10. Now one of these compounds has been tested and shown benefit in children with 2 different types of genetic mitochondrial diseases, suggesting that EPI-743 function may be more broad than CoQ10.

EPI-743 may be much more than just a potent CoQ10. It appears that this drug either works at more than one part of the electron transport chain -- or that it might act as a beneficial "parallel circuit" around several defects in this mitochondrial energy system.

In an important highlight from the 2010 CHDI meetings, Dr. Joakim Tedroff from NeuroSearch presented very encouraging preliminary results from the Phase 3 ACR-16 (Huntexil) trial in Europe. This trial will almost certainly bring forward a new drug treatment for Huntington's (HD) that significantly improves, not just stabilizes motor ability. Trial results also showed a trend toward cognitive improvement, and very importantly that there were no significant drug side effects. This drug is not only effective, it has safety features to allow its use in all those with HD. And in other promising news outside of the trial, NeuroSearch has shown that metabolic improvement occurs in the the brains of Huntington's patients who have taken the drug.

Just before New Years, Kimberly Gibson an enthusiastic gene positive young woman from Atlanta, Georgia sent this message on behalf of a group working for Huntington's: "We have a few questions. How realistic is a cure within ten years? What kind of treatment do you predict us having in five? What would it take to make it happen?"

What kinds of treatment are realistic in 5 or 10 years is more than I can know, or guess. But I am pretty sure I know what it will take and who will "make it happen". It will be people who work with passion -- just like her.

Who has the right to decide how couples at risk for Huntington's should have children? Or more specific to the debate now occurring in Europe: What rights do couples have when requesting reproductive procedures (PGD) for the purpose of having a gene negative child? What are the issues in this debate -- and who has the right to decide?

In case you can be in Baltimore, Maryland on Saturday November 21, don't miss this one day symposium with many exiting presentations about clinical research. And for the first time this year a workshop on clinical trials will be given for Huntington families. Sign up today. It will be worth the trip.

And in a very special part of this event, Judy Roberson from Northern California will be a featured speaker.

We at HDDW are saddened by the death of Marie Portillo, who lived half of her too short life physically dragged down by juvenile onset Huntington's. But according to our good friend Malcolm Casale, Marie's godfather "She had quality of life 'til the end, as she was always cheerful and would join us in singing and readings. Part of this was due to her foundational belief that the cure was becoming available, and that all she had to do was hang on a little while longer."


We need 60 more good Huntington's people to get the ACR16 trial done in North America. Do you know that 90% of the more than 400 Huntington's participants from the European trial have petitioned centers so that they may continue ACR16 compassionate use after the trial? This remarkably high number suggests that Huntington families in Europe believe that ACR-16 may be doing something very good.

Why are we in the U.S. and Canada risking our chance to get this drug? Do you know that slow enrollment is the reason 2 out of every 3 clinical trials in the U.S must close before enough information is gathered to bring a new drug forward to the FDA -- because the drug company runs out of money. Each day that we don't enroll is adding to the cost of this multimillion dollar trial.

If we don't fire up 60 more people to join this trial, we may be risking our chance to get this drug.

Huntington's care provided by Huntington Study Group (HSG) professionals and Huntington's Disease Society of America (HDSA) is almost exclusively limited to specialty centers. This is exactly what our fund-raising and federal research dollars support: Expert medical care and social service support given by Centers of Excellence. This is all well and good, but . .

What about families -- who probably represent the majority of Huntington's patients worldwide -- who don't have a center within geographic reach? What about those families whose medical care and support services are all too often poor to non-existent? Do our Huntington's organizations believe that responsibility for care ends at the center's door?

Today after three-pronged efforts that included a few good folks from the HD family community, clinical investigators in the HART ACR16 clinical trial and the sponsoring drug company, Neurosearch is moving forward in its application to the FDA to provide drug to North American participants after they have completed the placebo controlled trial. It goes to show that the HD World can be moved -- if we all push together.