Huntington's Disease Drug Works (HDDW) founded by LaVonne and Nathan Goodman in 2004 has evolved over the years. At present the focus is providing information on present treatment options for Huntington's disease that include:
- Drugs that are presently available and used (with variable success) for treatment of symptoms that include chorea, depression, anxiety, irritability, agitation, psychosis, apathy and rigidity.
- Supplements and life-style modifications that are available that may be useful treatments for neuroprotection.
We also provide information on drug development and clinical trials:
- Reports on drug candidates and their sponsoring companies.
- Updates on progress in finding biomarkers.
- Clinical trial information and education.
The Huntington Train to Glory, Connecting HD people to HD Treatment
After the completion of HDDW trials in 2007, we extended the goal of this site to provide information on treatments for Huntington's; both those for symptoms and those that may slow down disease progression. We have also added more information on drugs in development and clinical trials.
The path from research to effective treatment for Huntington's disease is like a train on a track. Scientists built this train, with support from hard-working Huntington's organizations along with the sweat and tears of many HD families. Borrowing from Woody Guthrie's eloquent lyrics, this Huntington's train is truly bound for glory. The research engine has been fired up with energy as more is learned about the biology of this disease in several model systems. This train has gathered speed as first one, then several drugs and agents have been found which partially treat the disease in mouse models. Scientists continue to believe that drugs which are therapeutic in the mouse have the highest chance of success in people. And though the train was slowed with recent clinical trial failure of Miraxion, we remain optimistic that treatments will come.
We'll endeavor to provide information on treatments that are available, and on those that are still in the development process.
HDDW Individual Therapeutic Trials It is known that many people with symptomatic Huntington's disease as well as many asymptomatic individuals "at risk" are taking drugs and over-the-counter agents that have been shown beneficial in genetic models of this disease. HDDW's primary objective in these trials was to follow the course of symptomatic Huntington's people at all stages of disease while taking a cocktail of agents which includes creatine, omega-3 fatty acids, trehalose, coenzyme Q-10, and blueberry concentrate.
Results: First participants began in 2004, and have been followed for three years by tests that are performed from their home computer. These tests measure motor and cognitive function on a weekly basis and track results over time. In this small group, results showed stabilization or improvement in several participants with early or midstage disease who took agents consistently. Those with late disease did less well, and showed continued progression of their disease. These results suggest that the combination (or a part thereof) may beneficial for up to three years, and best if treatment is started early and the agents are taken consistently.
Comments: It is important to remember that HDDW individual trials were observational, and as such can not be generalized to the larger population. Only large research-quality clinical trials can give definitive results.