Researcher with Huntington's gene joins $400 million brain disease initiative

A scientist who carries the gene mutation for Huntington's disease is participating in a new international research program focused on neurodegenerative conditions. The scientist's grandmother and mother both died from the inherited disorder. Colleagues advised against centering a career on the disease, but the researcher chose to pursue it anyway.

Initiative details The Brain Health accelerator was announced Tuesday.

It is based at the Allen Institute and includes researchers from multiple countries. The program received $400 million in funding. Sources include $200 million from the Allen Institute, $100 million from the family of Amazon founder Jeff Bezos, and $100 million from Amazon Web Services, the National Institutes of Health, and EverythingALS.

Research approach The accelerator is initially studying Alzheimer's, Parkinson's, Huntington's, Lewy body dementia, and ALS. Researchers plan to emphasize human tissue studies over animal models. A mentor at the University of British Columbia noted that Huntington's disease can have severe effects on families.

The university's Centre for Molecular Medicine and Therapeutics maintains a biobank with more than 300 postmortem Huntington's brain samples available for study.

Timeline and challenges The scientist underwent preimplantation genetic diagnosis with a spouse. Twins born in 2005 were the first successful commercial PGD births in British Columbia and did not inherit the mutation. Researchers aim to begin human trials by 2031.

The mentor cautioned that moving from target identification to regulatory approval can take 10 to 15 years and cost up to a billion dollars.