‘None of us are free until we are all free’: science and solidarity at the 10th Annual Huntington’s Disease Therapeutics Conference

Riding the emotion of a keynote speech by a young scientist at risk for Huntington’s disease, and seeking treatments with the immense help of a non-profit foundation, the participants at a historic research conference this week witnessed the fusion of science and human solidarity ultimately necessary for defeating HD.

On the evening of February 23, I and the approximately 300 attendees at the 10th Annual HD Therapeutics Conference, sponsored by the CHDI Foundation, Inc., listened as Jeff Carroll, Ph.D., recounted his mother’s demise from HD, his positive test for the HD genetic mutation, and his decision to pursue a career in science to save himself and others from HD.

“He’s an interesting combination of things in terms of being an advocate in the community, in terms of being someone from an HD family, in terms of being a top-flight researcher in the HD community, in terms of being a great communicator – he and his partner Ed Wild – in establishingHDBuzz, which is just a tremendously useful model of how to communicate results out to the rest of the community,” Robert Pacifici, Ph.D., CHDI’s chief scientific officer, said in introducing Dr. Carroll at the conference in Palm Springs, CA.

As an HD researcher-advocate who has attended all ten therapeutics conferences since 2006, Dr. Carroll offered a uniquely qualified, candid assessment of the progress towards treatments and CHDI’s role in the process.

“Every year, I come home revitalized and energized by the site of so many smart people working so hard on this problem,” Dr. Carroll, 37, told the audience in the main ballroom at the Parker Palm Springs hotel. He expressed his profound gratitude to CHDI, which has funded his and numerous other scientists’ research.

Painful progress toward success

However, success depends on the “efficient and timely completion of well-designed Phase III trials with HD drugs,” Dr. Carroll continued.

“A few weeks ago I attended a meeting at the Princeton CHDI office that included attendees from major pharmaceutical companies currently running HD clinical trials,” he said. “They are deeply concerned about something that would never have occurred to me to worry about, which is poor recruitment for trials of Huntington’s disease drugs.

“On reflection, it makes sense that the HD community may be wary of the way we have been speaking to them. Participating in the first clinical trial of a new molecule might be exciting, but participants of the third could be excused for having some questions.”

Trial administrators put participants through a daunting number of tests, he observed, which may discourage people from participating in more than one trial. Because trials are extremely expensive, sponsors often try to maximize the findings in Phase II, but not enough trials are reaching Phase III, he added.

“It must be said the scale of what is possible here must be unique in human history,” Dr. Carroll said of the efforts by CHDI, which has put more than $700 million towards treatments. “Resources on the scale being deployed by CHDI have been spent on common diseases, but never before have they been spent on such a focused attempt to ameliorate a rare disease.”

The HD community will achieve “something never done before” or “fail majestically,” he quipped with irony.

He added: “We might actually be watching the painful progress toward success.”

You can watch Dr. Carroll’s speech in the video below.


Source: At Risk for Huntington’s Disease

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