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New Directions in Alzheimer’s Drug Research

Dr. Roberta Diaz Brinton joined other award-winning scientists on the front lines of discovery to discuss the future of Alzheimer’s research. The group gathered before the 2019 Alzheimer’s Drug Discovery Foundation’s annual luncheon of the Melvin R. Goodes Prize for Excellence in Alzheimer’s Drug Discovery.

An excerpt from the discussion…


Until recently, Alzheimer’s drug development has been largely focused on beta amyloid plaques and tau tangles in the brain, the classic hallmarks of Alzheimer’s disease. Unfortunately, these attempts have yielded less successful results than had been anticipated. Sharing the ADDF’s long-held belief that aging is the main cause of Alzheimer’s disease, these visionary scientists have taken the field in new directions—investigating drugs that impact multiple symptoms of aging, not just plaques and tangles. Their body of work represents some of the most novel, non-amyloid approaches to the discovery and development of drugs for Alzheimer’s and related dementias. It was exciting to hear each of the participants discuss what could become the “next big thing” in Alzheimer’s research.


Roberta Diaz Brinton, PhD (2017 Goodes Prize awardee), inaugural Director of the Center for Innovation in Brain Science at the University of Arizona, where she also serves as Professor of Pharmacology and Neurology in the College of Medicine, Tucson, is conducting research focusing on the bioenergetic and regenerative systems of the brain. For the past 40 years, Dr. Brinton has been working on a neuroactive metabolite of progesterone called allopregnanolone—a substance found in the body, that declines with age, with tremendous metabolic effects.

Early on the ADDF funded Dr. Brinton’s work, as she discovered that the allopregnanolone molecule seemed to stimulate the proliferation of stem cells and create new nerve cells. This process, called “neurogenesis,” has the potential to restore lost cognitive function in Alzheimer’s patients by essentially regenerating the degenerated Alzheimer’s brain.

Dr. Brinton explained that she is moving forward with a phase 2 clinical trial funded by a grant from the National Institute of Aging (NIA), built on ADDF support, including the Goodes Prize. “It appears that allopregnanolone is most beneficial in those who carry the risk factor gene for Alzheimer’s disease,” she explained. As such, she is looking at a precision medicine approach for therapeutic development of allopregnanolone for Alzheimer’s in this high-risk population. While the initial trial involved intravenous infusion, thanks to the Goodes Prize, Dr. Brinton hopes to be able to investigate and patent formulations of this molecule with alternate delivery systems.

To read the full article, click here.

September 24, 2019

The Center for Innovation in Brain Science (CIBS) at the University of Arizona is addressing the challenge that, in the 21st century, there is not a single cure for a single neurodegenerative disease and is focused on four age-associated neurodegenerative diseases: Alzheimer’s, Parkinson’s, Multiple Sclerosis and ALS.

© 2024 Center for Innovation Brain Science. University of Arizona Health Sciences.

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