For comparison: The National Institute of Health (NIH) budget for Huntington's research is $47 million for 2007. The HDSA budget dedicates about $5 million per year to HD research. The Hereditary Disease Foundation (HDF) research contribution, though significant, is not available for comparison.
What is High Q/CHDI? High Q was first established in 2002 to provide financial support for Huntington's disease research. In the last 5 years High Q has adopted a more active approach and added two sister organizations; CHDI Inc. and HP Therapeutics. These foundations have kept such a low profile that many in the HD community are not aware of them or what they do. I think it is good to learn more about these efforts because it brings us greater hope for the timely development of treatments.
All three groups are managed in a coordinated manner, and function more like business organizations than traditional medical research foundations. Instead of open ended grants, the foundations award contracts for specific projects. By using a contract system, they direct resources to those areas they believe will be most productive for finding treatments over the shortest time period.
Funding and Management: At the top, generous anonymous donors provide the funding for this foundation. Robi Blumenstein, a lawyer with experience in business, is the day-to-day manager of foundation operations. He also serves on the Advisory Committee to NINDS, the NIH institute that oversees most of the federal funding for Huntington's research and clinical trials.
High Q: Alan Tobin and Ethan Signer are senior scientific advisors to High Q and CHDI who oversee High Q research operations. Each of these scientists were former long term advisors to Nancy Wexler's Hereditary Disease Foundation (HDF). Though most of High Q's work relates to laboratory research, it has provided clinical trial support for the Huntington Study Group, the Huntington Project, SET-HD and COHORT.
CHDI: Robert Pacifici leads CHDI. This foundation, which started work in late 2004, starts with the research results produced by scientists world-wide and carries out the complicated work of drug development. CHDI contracts with biotech and pharmaceutical companies, as well as academic research sites, to develop drugs. Time is important, and as reported at the recent CHDI and HDSA meetings this year, this organization hopes to deliver several drug candidates on schedule.
HP Therapeutics: Daniel van Kammen is the new official leader of this part of the foundation. HP Therapeutics will increase clinical trial efforts in the U.S. In Europe, HP Therapeutics has already helped to establish the European Huntington's Disease Network (EHDN). This effort alone has doubled the HD clinical trial base, and EHDN is already at work on several studies to make all clinical trials more efficient.
Editorial Comments: I'll use the words of Paul Enderson, our new HDDW guest editor: "Speaking as somebody with a vested personal interest in the work that High Q and CHDI are doing, I find it reassuring and encouraging to know that there are people with money magnanimously pushing research in the right direction".