‘It’s really getting real’: payoffs in the effort to treat Huntington’s disease

Schermata 2014-03-13 alle 21.22.41
The path to treating Huntington’s disease – a potential major breakthrough in the history of science and medicine – is becoming clearer.
 That was the takeaway message from the Ninth Annual HD Therapeutics Conference, organized by the CHDI Foundation, Inc. and held February 24-27 at the Parker hotel in Palm Springs, CA. Spending tens of millions of dollars annually, CHDI is a non-profit, virtual biotech founded solely to discover HD treatments. Some 300 participants from academia, the pharmaceutical industry, and biotech firms took part, as well as a number of patient advocates, including Olympic rowing medalist Sarah Winckless, who delivered the keynote address.
 “The tagline would have to be ‘it’s really getting real,’” said Robert Pacifici, Ph.D., the chief scientific officer for CHDI Management, Inc., in an interview with me at the conference. “What I’m seeing at this conference already is the culmination of very large, very long-term efforts – things that have taken years and thousands of person hours, patients’, caregivers’, researchers’, and physicians’ – finally coming together in ways that are really conclusive and really helpful.”
All that work has involved numerous questions about the disease and potential ways to treat it, Dr. Pacifici explained.
“All of those things sadly have an incredibly high attrition rate,” he observed. “The fact that we’re getting answers is the thing that makes me the most excited. Sadly, sometimes we don’t like the answer. Sometimes the answer is: ‘That doesn’t work.’ But that’s still very useful for researchers.”
 Winnowing out the useless approaches allows researchers to “refocus our resources on something that we feel has a better chance of bearing fruit,” Dr. Pacifici said.
 Sitting one evening with a group of CHDI researchers, I expressed the natural concern of the HD community – a concern sometimes tinged with impatience and frustration: could the rapidly expanding knowledge about HD result in an endless search for treatments fueled by questions that simply produce new questions rather than treatments?
 They answered with an emphatic no. Echoing Dr. Pacifici, they said that real solutions were in the works.
 The conference did seem more coherent in comparison with the previous three I had attended. Indeed, as one senior CHDI advisor observed in response to my observation, Huntington’s researchers now have an understandable “story to tell” about the disease and the research.
 You can watch my interview with Dr. Pacifici in the video below. Just below the Pacifici interview, Portuguese speakers can watch my interview about the conference with Dr. Mônica Haddad of Brazil.

Copyright: All rights of any text or trademarks mentioned in the article are reserved to their respective owners.