Renowned Alzheimer’s Researcher and BrightFocus Contributor Wins “The Oscars of Science”

Martha Snyder Taggart, BrightFocus Editor, Science Communications
  • Science News
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University of Pennsylvania Professor Virginia M.Y. Lee, PhD, who helped develop and guide BrightFocus Foundation’s Alzheimer’s Disease Research program, has won a 2020 Breakthrough Prize in Life Sciences. The prize, known as the “The Oscars of Science,” recognizes her research leading to new avenues for potential drug discovery and development.

Now in its eighth year, the Breakthrough Prize annually honors achievements in the Life Sciences, Fundamental Physics, and Mathematics and is sponsored by Sergey Brin, Priscilla Chan and Mark Zuckerberg, Ma Huateng, Yuri and Julia Milner, and Anne Wojcicki. Dr. Lee, the John H. Ware 3rd Endowed Professor in Alzheimer's Research at the University of Pennsylvania’s Perelman School of Medicine, is one of four winners of the 2020 Life Sciences prizes, which will be presented at a November 3 ceremony in Los Angeles. The award provides $3 million to each scientist toward their future research.

Dr. Lee is being honored for her work in understanding how different types of two misfolded proteins (TDP43 and alpha-synuclein) can move in different cell types and lead to different types of neurodegenerative diseases and movement disorders, including frontotemporal dementia and amyotrophic lateral sclerosis for TDP43, and Parkinson’s disease and Multiple System Atrophy for alpha-synuclein. TDP43 and alpha-synuclein have also been shown to be involved with other dementias, like Alzheimer’s disease.

In 1991, Dr. Lee evolved the “tau hypothesis,” which posited that tau tangles inhibit the proper firing of neurons. She found similar entanglements associated with Parkinson’s and with ALS, and later uncovered how misfolded proteins could spread from cell-to-cell through the central nervous system. By working to replicate the pathological evolution of tau proteins, Lee invented a protein roadmap to neurodegenerative disorders and an elucidation of common mechanisms of degeneration.

“It is an honor to receive such a prestigious award, and this funding will help us as we continue to push for a greater understanding of these diseases that can ultimately help us find ways to help millions of these patients,” Dr. Lee told Penn Today, a university publication.

Dr. Lee is a past member of the Scientific Review Committee (SRC) for the BrightFocus Alzheimer’s Disease Research (ADR) program, where, for seven years in the 1990s, she was active in reviewing and rating ADR grant applications.  During that time, Dr. Lee was part of the advisory panel shaping the committee’s emphasis toward “high-risk, high-reward” projects, i.e., innovative research ideas that are non-doctrinaire, yet well-grounded and solidly planned.  Dr. Lee’s contributions to the ADR SRC helped ensure that the BrightFocus grant portfolio remained diversified, and that BrightFocus played a valuable role in funding some of the foundational work on protein misfolding and aggregation in neurodegeneration.   

Since then, she has been a part of ADR grants involving tau’s role in AD and other tauopathies, including being a mentor on a 2014-16 postdoctoral fellowship grant to Jing Guo, PhD, and collaborator on a 2018-21 grant to Zhuohao He, PhD,  both at the University of Pennsylvania.

In addition, she’s collaborated with her husband, John Trojanowski, MD, PhD, also at University of Pennsylvania, to investigate the involvement of the TDP-43 protein in AD. This has led to their characterizing a new brain disorder that can mimic AD or frontotemporal dementia called “limbic-predominant age-related TDP-43 encephalopathy” (LATE), which has been summarized in an article by Dr. Trojanowski posted on the BrightFocus website.

“Dr. Lee has supported the Alzheimer’s Disease Research program in innumerable ways, often behind the scenes,” said BrightFocus Vice President of Scientific Affairs, Diane Bovenkamp, PhD.   “We celebrate Dr. Lee’s achievements and applaud the Breakthrough Prize Board’s recognition of her impressive track record of research and leadership. Her groundbreaking science is lending critical insights into the root causes—and potential cures—of Alzheimer’s, related dementias, and other neurodegenerative diseases.”

Earlier Breakthrough Prize winners with Ties to BrightFocus Foundation

Another BrightFocus ADR SRC member, and past BrightFocus grantee, John Hardy, PhD, of University College London, received a 2016 Breakthrough Prize in Life Sciences for his discovery of mutations in the amyloid precursor protein gene (APP) that cause early-onset AD. Analyzing the genomes of members of a family with a high incidence of early-onset Alzheimer’s, he discovered that they shared the same APP mutation, which leads to a cascade of effects resulting in the buildup of fragments of protein to form amyloid.

In 2017, the 2016-19 BrightFocus ADR grantee Huda Y. Zoghbi, MD, of Baylor College of Medicine, won a Breakthrough Prize in Life Sciences for her discoveries, not directly related to her current ADR grant, of the genetic causes and biochemical mechanisms of two rare inherited neurodegenerative diseases, spinocerebellar ataxia and Rett syndrome.  These findings contributed insights into the pathogenesis of other neurodegenerative and neurological diseases, including Alzheimer’s, and led Dr. Zoghbi to pursue dementia research. Earlier this year, she was awarded the BrightFocus Pioneer in Genetics award.