Highlights of the HD Therapeutics Conference

HD Lighthouse Contributing Editor's Comment:

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Posted to the HDL: 19 Mar 2006

Dr. Pacifici

Sponsored by CHDI, Inc.

CHDI, Inc., the drug company that works solely for Huntington’s http://www.chdi-inc.org/ hosted its landmark HD Therapeutics Conference in early February. This meeting marked an important milestone. As Dr. Michael Hayden, a premier HD scientist who has contributed over two decades of research observed; this was the first conference with “HD” and “therapeutics” in its title. True to the therapeutics focus and CHDI goal to “rapidly discover and develop drugs that prevent or slow Huntington disease”, this conference focused solely on “translational science”. This is the complicated process that moves beyond basic scientific research through the stages of drug development.

In addition to the CHDI team, an international group of scientists from industry and academia participated. Speakers included leaders in HD basic research and the Huntington Study Group (HSG). Also on stage were Alzheimer’s scientists, and representatives from large and small pharmaceutical companies. Leaders from the High Q Foundation, the Huntington’s Disease Society of America (HDSA), the Hereditary Disease Foundation (HDF), and HD family advocates were present to represent the full range of stakeholders.

The CHDI Team

Dr. Robert Pacifici, chief scientific officer of CHDI, introduced the company’s team members. Each one brings practical expertise in specific areas and stages of drug development, gained by prior experience in the pharmaceutical industry. Because they come from many companies, the team can provide not only extensive “insider” knowledge, but also the potential for industry collaboration. Their job is to utilize the knowledge gained through basic research to choose the most promising molecules to develop into drugs, and take these molecules through the stages of drug development.

Assay Presentations Several presenters from academia and industry described different screening methods (assays) used to select best drug candidates of HD effectiveness. The “good news” take- home point from these sessions is that small companies are bringing forward drug candidates found in screens. One biotechnology company in Boston, EnVivo, has developed a screen that identified a drug currently moving (with HSG assistance) toward first phase human clinical trials. The more vexing take-home point is that although there are an impressive number of assay systems, no system (or combination of systems) has been shown better than others. As one pharmaceutical representative put it, “I came here to learn about the best assays to put my drug through, and learned that there aren’t any best (yet)”.

Clinical Trial Measures

Presenters Drs. S. Tabrizi and J. Stout described the difficulties involved for measuring disease progression in Huntington’s. They stressed that better measures are needed to shorten the time necessary to complete clinical trials. Preliminary results from PREDICT-HD (http://www.huntington-study-group.org) were presented. Researchers are working hard to analyze results so that presymptomatic individuals can soon enter clinical trials.

Clinical Trial Plans

Drs. R. Ferrante, I. Shoulson, and S. Hersch presented results from early human trials on CoQ10 and creatine, and discussed planned Phase III clinical trials. Both of these drug molecules target the same system (bioenergetics) involved in HD. Each drug will be tested in separate trials that will cost millions of dollars and long years of time. At the conclusion of these trials, it will not be known if these drugs are better when used in combination.

Clinical Comments and Commentary

A very important question was posed by Dr. Frank Longo, a revered Alzheimer’s scientist from Stanford, who presented exciting work on drug molecules that mimic the action of brain-derived neurotrophic factor (BDNF). Following the presentations of Drs Hersch and Shoulson, Dr. Longo (who is also a neurologist) asked how he should answer his HD patients’ questions about whether to take creatine or CoQ10. Dr. Hersch gave his opinion about the potential advantages of creatine, and Dr. Shoulson his opinion regarding CoQ10. Both went on to say that they do not discourage their own patients from taking these supplements. The same message was expressed by other research neurologists in private conversation.

This clinical approach represents a paradigm shift in the HD research community. In contrast to the message from the HDSA 2005 Conference, leaders are giving (with cautious optimism) permission for HD people to take supplements. More than a few of the researchers present at the conference went further, expressing that supplement usage made good sense, but stressing the need to use high quality (at least USP: US Pharmacopeia-approved) preparations to limit potential toxicity of high-dose usage of either of these agents.

Commentary

Before CHDI, HD researchers had to do it all. They were responsible for doing the research, finding drug candidates, and taking them all the way through clinical trials. This process is the common plight of many orphan diseases because big pharmaceutical companies aren’t interested in treatments for small populations of patients. HD researchers have had to do what the pharmaceutical industry wouldn’t.

But now, in an organization that is the first of its kind, HD has CHDI. This organization is geared up and funded to take the wealth of HD research knowledge to an efficient drug development phase. CHDI is collaborating with academia and industry to bring forward the best new drug candidates, and to improve on existing drugs.

Though much of the CHDI effort won’t bear fruit in less than five years, the team is also working on projects that could greatly impact HD people now. CHDI has requested information from HSG’s observational database and will search through these data for clues about whether the use of various supplements and approved drugs might benefit HD people more immediately.

CHDI is a timely addition to other “already strong” HD organizations. It is exciting to witness this team powering up and flexing muscle for its part in the fight for HD lives. Whether coach, cheerleader or spectator in the audience, it’s time to cheer them on.

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