As a physician caring for Huntington's disease (HD) patients, I am often asked about supplements that have shown benefit in animal models of HD. Other HD patients use these supplements without guidance or supervision by a doctor. And even when the doctor is aware of supplement use, it is difficult to monitor the effect. How would we know they are helping? In an effort to address this situation Huntington's Disease Drug Works (HDDW) was established to follow the course of 30 people taking supplements. Now at the three-year mark, we are pleased to share results. A similar summary was reported in the HDSA November edition of The Marker.

Supplements won't be the cure; but they are available now. I believe that HDDW results lend support for supplement use in HD if used before late disease, the earlier the better. It is my opinion that with a little luck and the continued pace of drug development, one or a combination of supplements just might keep us around long enough to take advantage of better treatments that will come.

The Results: The majority of those with early or middle stage disease who consistently took the combination of supplements did well, showing either improvement or stabilization of test measures and functional capacity during the duration of the trial. Those with late disease, those at any stage of disease who were noncompliant (i.e., who did not consistently take the supplements), and those who experienced significant psychiatric or medical illness showed decline.

Simply Put: The majority of those with earlier stage symptoms improved or stabilized if they consistantly took supplements.

Trial Description: Our method was described in the May 7, 2004, Science magazine [Couzin J 2004]. HDDW trials are not like traditional research trials. Instead, they are similar to the process in your doctor's office when a medication is prescribed and individual patient response is observed. HDDW trials were not placebo-controlled or blinded, which means that every participant received a known supplement combination. Participants were allowed to take additional supplements or medications. All of the participants were on antidepressants.

The progress of each participant was followed by finger-tapping (motor) and cognitive (thinking) tests performed on a home computers. The computer-based tests, which were completed several times per month, were relayed over the internet and analyzed at HDDW. Functional status was independently assessed by interview with each patient and/or family mentor. Each participant was supervised by their local doctor.

Supplements: Care was taken to supply quality supplements, when possible from companies whose product was approved for use in FDA sanctioned clinical trials. Supplements included creatine, coenzyme Q-10, an omega-3 combination of EPA and DHA, trehalose, and blueberry extract.

Supplement Dose Source
Creatine 5 grams 2 times/day Avicena
Coenzyme Q-10 300 mg 2 times/day
(=1200 mg/day Vitaline)
Trehalose 25 grams 3 times/day Brooklyn Corp.
Omega 3 1 gram EPA 2 times/day
300 mg DHA 2 times/day
Omega Brite
Blueberry Extract = 1 to cup of blueberries Brownwood Acres

Participants: Thirty participants were enrolled over the three years. Twenty-three have been followed for between 2 and 3 years (depending on individual start date). Of the remaining 7 participants, there has been 1 death (due to lung cancer), 3 nursing home placements, and 3 withdrawals (associated with psychiatric events). Participants included men and women between the ages of 34 and 75 from all areas of the country, and unlike traditional clinical trials, late stage participants were not excluded.

Results: Compliant early to middle stage participants: Six out of 9 early to midstage participants who have taken all supplements rigorously have shown improved or stabilized test measures of cognitive and motor function and functional capacity. Two other participants in this group who did not take trehalose had either motor or cognitive worsening. The final participant in this group demonstrated stability with all test measures until psychiatric events occurred. Thereafter, motor skills were stable while cognitive skills declined.

Noncompliant early to middle stage participants: Six early to middle stage participants were noncompliant, or did not take supplements as directed. In these cases, participants admitted noncompliance and pharmacy records confirmed lesser usage. All participants in this group declined in motor, cognitive, or both test measures.

Advanced stage participants: The remaining 8 participants had advanced disease or could not function independently at the beginning of the trial. Despite compliance maintained with the help of dedicated caregivers, 6 out of 8 of these participants have declined. Three compliant participants were stable, then began decline (going from walking to wheelchair) at the time of medical illness or surgery. Both noncompliant patients worsened.


We acknowledge that HDDW observational trials have limitations that include small numbers, no placebo controls, no blinding, and possible compliance bias (compliant patients often have healthier life-styles than noncompliant patients). These limitations prevent generalizing our results to the broader population of patients.

However, despite these limitations, we believe there are useful observations. First, there were no significant side effects due to the supplement cocktail at any stage of disease. Second, motor and cognitive skills either improved or showed 2-3 year stabilization (or lack of progression) for the majority of compliant early and middle stage participants. This is much better than the predicted natural course of the disease. Third, participants who were noncompliant or stopped supplements during the trial showed worsening. Fourth, greater benefit correlated with stage of disease: those with earliest stage improved, middle stage stabilized, and late stage worsened. Though there may be other explanations, all of these observations fit the pattern that would be expected if all or part of the treatment cocktail gives benefit.

Conclusions: And finally, as a physician treating HD patients, and as editor of this treatment website, how do I use these results? I give information about HDDW results and as well as completed research clinical trials. I also emphasize that more certain answers are unknown until other definitive clinical trials are completed. If a patient decides to use supplements, I give guidance and information on safest products, recommend most probable therapeutic dosing, and monitor for side effects. Some choose to use supplements, and some don't. It is, and should be, the patient's (or reader's) choice.


Couzin J. Huntington's disease. Unorthodox clinical trials meld science and care. Science. 2004 May 7;304(5672):816-7. PubMed abstract