Lifting Huntington’s families’ spirits with the promise of clinical trials

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Perhaps nothing lifts the spirits of struggling Huntington’s disease families more than news about research breakthroughs.

With people emitting oohs and ahs about some of the key developments, you could feel the excitement in the room Monday evening, September 24, as Jody Corey-Bloom, M.D., Ph.D., presented her annual research update to the support group of the San Diego Chapter of the Huntington’s Disease Society of America (HDSA).

The director of the HDSA Center of Excellence for Family Services and Research at the University of California, San Diego (UC San Diego), Dr. Corey-Bloom kicked off the update with the hottest topic in the HD research world: gene silencing techniques aimed at attacking the disease at its roots.

“This is a big thing,” Dr. Corey-Bloom stated. “There are actually two methods to accomplish this that are under way in research labs around the world…. We want to switch off the gene that is responsible for Huntington’s disease. A lot of people feel very good about these techniques.”

Before a room packed with some 50 support group participants, Dr. Corey-Bloom proceeded to outline the efforts to set up clinical trials to test RNA interference (click here to read more) and antisense oligonucleotides (ASO) (click here to read more) as ways to block the negative effects of the HD gene, which produces a harmful protein.

“If we can get rid of this harmful protein, there’s a good chance that we could slow down the disease or even prevent it,” Dr. Corey-Bloom explained.

Dr. Jody Corey-Bloom at a 2010 HD research meeting (photo by Gene Veritas)

A trial in 12 months?

 “It’s likely that we’ll see both approaches being tried,” Dr. Corey-Bloom said, noting, that, given the current state of the science, it appears that the ASO approach will enter a trial first.

Dr. Donald Cleveland of UCSD and Isis Pharmaceuticals, Inc., of Carlsbad, CA are nearly “ready to go” with a clinical trial, perhaps as soon as within 12 months, she observed.

“The critical thing to say here is that we really are edging closer to human trials,” Dr. Corey-Bloom emphasized.

The trial site has not yet been determined.

Dr. Cleveland, UCSD’s Ludwig Institute, and Isis will be honored at HDSA-San Diego’s 12th Annual Celebration of Hope Gala on November 16, 2012.

In the coming weeks, I will report in detail on the UC San Diego/Isis project, which is mainly underwritten by the CHDI Foundation, Inc., the multi-million-dollar, non-profit biomedical research foundation dedicated exclusively to accelerating Huntington’s drug discovery and development.

You can watch Dr. Corey-Bloom’s presentation in the video below.

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Source: At Risk for Huntington’s Disease



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