HD Lighthouse Contributing Editor's Comment: The efforts going on at CHDI are exciting. These people have a real sense of urgency about the research.
--Marsha L. Miller, Ph.D.
Posted to the HDL: 24 Jan 2006

LaVonne Veatch Goodman, M.D.

In mid December, I was invited to participate in a working group meeting at the offices of CHDI, Inc. (CHDI). What follows is my first hand report to Lighthouse readers and my impressions of this organization. But first, because both High Q Foundation (High Q) and CHDI are relatively new players in the Huntington’s community, a brief background of each will be given.

High Q Foundation

Though High Q has been working behind the scenes for several years, it has emerged as the major player for Huntington’s research. High Q founders, Ethan Signer and Allan Tobin are not new to the HD community and were leading forces with Nancy Wexler in the Hereditary Disease Foundation (HDF). For the last decade, HDF has coordinated excellent basic research which was relatively “open-ended”, with each researcher designing best individual paths in deciphering molecular biology. On the other hand, High Q Foundation is shifting and coordinating Huntington’s research with the specific goal of focusing its direction to a “translational” mode. In plain words, the goal is to narrow research by selectively promoting that which is most relevant to the development of treatments for Huntington’s people. CHDI is an integral part of the High Q plan, and was jump-started by a generous and special donor.

CHDI, Inc.

Headquartered in Los Angeles, CHDI is the not-for-profit biotech drug discovery company set up by the same people that support High Q Foundation. It is not affiliated with any other pharmaceutical or biotech company. CHDI is “virtual” in that it doesn’t do the laboratory work itself, but contracts out specific projects to other companies. This pattern of pharmaceutical work is practical and cost effective even for big pharmaceutical companies. CHDI’s director, Robert Pacifici, brings impressive pharmaceutical credentials and is leading a group of twelve scientists drawn from the pharmaceutical industry. They are working full time and exclusively for therapy development in Huntington’s.

Though they don’t comment on specific agent/drugs, CHDI does some work on those already available. However, most effort focuses on new drug development. This can include work on a totally new drug, or work on chemical modification of an existing drug. Drugs in each of these categories are new chemical entities and require the process of FDA approval. The industry standard for taking a “lead” compound (chosen from chemical or animal screens) forward to phase 1 (safety) human clinical trials takes a minimum of 5 years. Of course, CHDI may be able to shorten this time by selecting compounds from other biotechs that have already gone through some of the process.

Meetings

Because research in Huntington’s is split into categories depending on possible molecular mechanisms of HD, High Q and CHDI have been meeting with expert scientists in many fields. It seemed that the purpose of these meetings is not just to share information, but to narrow the playing field; by getting the experts’ opinion of “best” pharmaceutical target(s) within each system. High Q and CHDI were practical and direct. They ended the session with two important questions; one that focused on experiments which could narrow the field of targets and another that came right out and asked the scientists which available drug they thought would work best.

Take Home Thought

CHDI and High Q have an inclusive approach. They bring together scientists from multiple disciplines and seek input and ideas from academic, industrial, and biotech sectors. And importantly, they have included HD patient/family representation, who can give a patient/family perspective and can report back to you at Lighthouse.

Better; CHDI and High Q incorporate urgency into their work. CHDI’s stated mission is to “rapidly discover and develop drugs that prevent or slow the progression of Huntington’s disease.” They are realistic too. They don’t use the “cure” word as they work toward attainable treatments.

Best; CHDI and High Q gives the impression that they believe, and are working from the premise that Huntington’s is treatable. Unlike those of us “old” to Huntington’s; both our exhausted HD families, and our true but wearied researchers; this group hasn’t been worn down by long years of struggling with HD. They bring a “can do” attitude and welcomed new energy.

I hope they can deliver.

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