PHAROS is an observational trial of people at risk for Huntington's. Patients are tested but not treated. Dr. LaVonne Veatch Goodman comments on the first PHAROS publication and argues that this tired old study should be stopped.

Researchers from the Max Delbrueck Center in Germay report promising results for a nutritional supplement derived from green tea. Summary by Dr. LaVonne Veatch Goodman.

Dr. LaVonne Veatch Goodman explains what endpoints are in clinical trials and why different endpoints are used in different HD drug trials.

After looking over the schedule of the upcoming CHDI conference Dr. LaVonne Veatch Goodman draws an analogy between the drug development process and a symphonic production.

As drugs enter clinical trials for Huntington's, it's time to learn about and get involved in the process that will promote early access (compassionate use) for patients who desire access to drugs before the FDA approval process is complete. In this article, Dr. LaVonne Veatch Goodman reports on a new FDA plan to allow early access to drugs and reviews the history of early access in other neurodegenerative diseases.

Researchers discover important new clues about how trehalose protects cells from stress, adding further support for trehalose as a potential treatment for Huntington's Disease. Summary by Malcolm S. Casale, Ph.D.

Every six months we review data gathered from HDDW trial participants. While doing this review I also read a book about Admiral James Stockdale who had been a Vietnam prisoner of war, and I drew a connection between the experiences of HDDW participants and the wisdom that has come to be called the Stockdale Paradox.

The research measure of disease progression utilized in Huntington's patients is the Unified Huntington's Disease Rating Scale (UHDRS). Because frequent UHDRS testing is not possible in the usual office setting, another secondary objective of HDDW therapy trials is to define an appropriate set of measures that can be used in the primary care setting and correlate as best as possible with UHDRS. For this purpose, HDDW will provide each trial participant (who must also have a dedicated helper) with web-based and centrally organized tests for motor, cognitive, behavioral and functional capacity components. Serial results will then be supplied (in readable form) to each participating physician, who may in turn supply this information to the individual. It is the HDDW goal that these measures can reproducibly be used in the medical office setting to assess individual patient response. It will be the individual participant's rate of disease progression, and specifically not the average of the group population that is to be the measured and reported to the treating physician.

The primary objective of the "HDDW Individual Therapeutic Trials" (ITT) is to facilitate a medically supervised and personal best treatment plan for the person with Huntington's disease. 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 the ITT endeavor is to bring both of these HD groups, asymptomatic and symptomatic, to observed and supervised care while taking these drugs.

The HDDW goal is to provide information on treatments for Huntington's; both those for symptoms and those that may slow down disease progression.

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 explosive 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 believe that drugs which are therapeutic in the mouse have the highest chance of success in people And in May 2007 we'll likely have proof of this concept with FDA approval of Miraxion (omega-3 EPA), one of the first supplements shown to benefit the mouse.

We'll endeavor to provide information on treatments that are available, and on those that are still in the development process.