As range of possible therapies grows, CHDI expands efforts to defeat Huntington’s disease

his article is Part 1 of a two-part series.

CHDI Foundation, Inc., the largest nonprofit effort aimed at developing therapies for Huntington’s disease, has expanded its efforts and partnerships to accelerate the defeat of the deadly disorder.

That’s the message transmitted in a July 29 interview by Robert Pacifici, Ph.D., CHDI’s chief scientific officer, who has a very personal commitment to disease eradication, and in a July 13 public presentation by Douglas Macdonald, Ph.D., CHDI’s director for research operations and scientific alliances.

CHDI emerged in 2003 out of the Hereditary Disease Foundation, where it was known as “The Cure Huntington’s Disease Initiative.” Funded by donors who wish to remain anonymous, “CHDI” is no longer an acronym but simply part of the foundation’s name.

According to Dr. Pacifici, and as reported previously in this blog, CHDI continues to spend $100 million annually on HD research and programs. The number of staffers at its offices in Los Angeles, CA, New York, NY, and Princeton, NJ, has grown to 100, doubling in ten years.

A virtual biotech firm, CHDI has no labs. Instead, it partners with, funds, and outsources projects to contract research organizations (CROs), academic labs, and pharmaceutical and drug discovery companies like Ionis Pharmaceuticals, Inc., the developer of RG-6042, a gene-silencing drug now in a historic Phase 3 clinical trial run by Roche. Roche took over the license after an Ionis Phase 1/2a trial successfully and safely lowered the amount of huntingtin protein, normal and mutant, in trial volunteers’ cerebrospinal fluid (CSF). Roche is now evaluating whether this reduction of huntingtin protein leads to clinical benefit (efficacy) in the Phase 3 trial.

“On any given day, there are about 700 other people who are supported by CHDI that are working on various aspects of the drug discovery and development pipeline that we try and orchestrate and integrate and enable,” Dr. Pacifici told me at the Los Angeles office, which is strategically located three miles from the city’s international airport.

CHDI seeks to “push the field forward” towards effective treatments and other “therapies,” which could include approaches other than drugs, he explained.

However, unlike private for-profit firms, CHDI does not seek to grow or perpetuate itself: “We actually don’t want to build a big company. We’d like to dissolve CHDI, because our job is done.”

CHDI is motivated by “time, not money,” because it wants to “accelerate” the discovery of therapeutics, Dr. Macdonald said in his talk at the Fourth Annual Convention of the San Diego Chapter of the Huntington’s Disease Society of America (HDSA-San Diego).

“We don’t have any competitors,” he added. “We only have collaborators.”

Robert Pacifici, Ph.D. (photo by Gene Veritas, aka Kenneth P. Serbin)

Robert Pacifici, Ph.D. (photo by Gene Veritas, aka Kenneth P. Serbin)

An in-depth look

You can watch my interview with Dr. Pacifici, my recording of Dr. Macdonald’s presentation, and the 2019 CHDI research highlights report, Postcard from Palm Springs, in the videos at the end of this article.

I first met Dr. Pacifici in December 2007, when he spoke at a “Spotlight on Huntington’s Disease” that I organized at the University of California, Los Angeles, for the oversight board of the California Institute for Regenerative Medicine, the state’s $3 billion voter-approved stem cell initiative.

In 2009, I visited a CHDI office for the first time, in Los Angeles, to interview Dr. Pacifici and other scientists to learn more about the organization. Since then, I have done seven video interviews with Dr. Pacifici – during the foundation’s annual HD Therapeutics Conference in Palm Springs, CA – to obtain snapshots of the progress towards treatments. In 2011, I keynoted the sixth conference in a major step out of the terrible and lonely HD closet.

Our July 29 interview was our ninth overall and, at 82 minutes, our longest and most in-depth. I sought to gain perspective on CHDI and the overall efforts towards therapies. I also wanted Dr. Pacifici’s assessment of so-called natural and alternative remedies used by some in the HD community, such as CBD oil, and also of the potential role of repurposed drugs – the topic of Part 2 of this series.

I first came in touch with Dr. Macdonald because he was CHDI’s point person for collaborations with Ionis and other gene-silencing projects. His July talk in San Diego was the most comprehensive public presentation of CHDI’s activities in lay terms that I have seen. (Also see Dr. Pacifici’s June 28 overview of CHDI at the 34th Annual HDSA Convention in Boston.)

Recording these two scientists in July and exploring their ideas once again helped me cope with my status as an HD gene-carrier and, I hope, contributed mental stimulation to help delay the inevitable disease onset. (Click here to read more.)

July 2019 marked a personal milestone for me: not only tracking CHDI for a decade, but also living symptom-free.

…. READ MORE

Source: At Risk for Huntington’s Disease

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