The Role of Genetics in Alzheimer Drug Development: Margaret Pericak-Vance, PhD


The director of the John P. Hussman Institute for Human Genomics at the University of Miami provided insight on the outlook on genetics and gene-based therapies for Alzheimer disease. [WATCH TIME: 3 minutes]

WATCH TIME: 3 minutes

"It has taken a while to be able to pull the participants and samples necessary to do this research. It’s also very expensive. But it’s truly, I think, the key for the future. You’ll see more and more [research being conducted]."

Alzheimer disease (AD) is a complex neurodegenerative disorder with a pronounced genetic component; its estimated heritability is 60% to 80%. Recently, the National Institute on Aging awarded a $46 million grant to the John P. Hussman Institute for Human Genomics (HIHG) of the University of Miami Miller School of Medicine to fund a new 5-year international genetic study of Alzheimer disease (AD) in individuals of Hispanic and African ancestry.1

Through the recruitment, assessment, and genetic analysis of a significantly large cohort of participants of Hispanic/Latinx and African ancestries, clinical, phenotypic, and genetic data will be collected along with social determinants of health factors to create a large genomic study resource. The cohort will include 5000 individuals from various African countries, 4000 African American individuals, and 4000 Hispanic/Latinx individuals.

Improving therapeutics through more targeted gene-related approaches has become more widely discussed throughout neurology, but has yet to be fully translated in AD. To learn more about the current role of genetics in AD drug development, NeurologyLive® sat down with Margaret Pericak-Vance, PhD, one of the co-primary investigators of the genetic study. Pericak-Vance, director of the HIH and Dr. John T. Macdonald Foundation Professor of Human Genetics, University of Miami Miller School of Medicine, stressed the need for large-scale studies containing homogenous sets of patients to fully understand the impact of targeting specific at-risk genes.

1. John P. Hussman Institute to lead international genetic study of Alzheimer’s disease in people of Hispanic and African ancestry. News release. Miami Miller School of Medicine. July 11, 2022. Accessed July 14, 2022.
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