Posted March 2, 2010 by LaVonne Goodman M.D.

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.

Posted February 16, 2010 by LaVonne Goodman M.D.

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.

Posted November 3, 2009 by LaVonne Goodman

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.

Posted October 28 2009 by LaVonne Veatch Goodman

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?