Study will use miniature brains cultivated from stem cells to study protein mutations

CurePSP has awarded a $100,000 grant for a potentially breakthrough study that will employ 3D organoid models that have been shown to produce structures similar to those seen in the human brain to understand the effects of protein mutations that are associated with neurodegeneration.

The grant will assist the Tau Consortium Stem Cell Group, created and supported by the Rainwater Charitable Foundation, in helping to extend the scope of this multidisciplinary and multiyear study. The funding has been provided through generous donations from Frank Semcer, Sr., in honor of his wife Mary Jane Semcer; and from F. Jackson Phillips, in honor of his wife, Linda A. Phillips. Both women have received diagnoses of progressive supranuclear palsy (PSP), a rare form of neurodegeneration that involves pathological accumulation of the tau protein, a type of naturally occurring protein in the brain. CurePSPs grant was made under the auspices of the Prime of Life Brain Initiative, a collaborative venture with Rainwater to advance research into neurodegeneration.

The CurePSP grant will support research conducted by investigators of the Tau Consortium Stem Cell Group including Dr. Sally Temple of the Neural Stem Cell Institute, Dr. Alison Goate of the Ichan School of Medicine at Mount Sinai, Dr. Justin Ichida of the University of Southern California, Dr. Celeste Karch of Washington University, and Dr. Martin Kampmann of the University of California San Francisco.

Sometimes called "disease in a dish," 3D organoids are grown from skin-derived stem cells of patients and healthy individuals. Studies using these patient derived cell cultures aim to avoid some of the challenges and limitations of using animal models in research, including lack of construct validity of data (the degree to which data measures what it claims to be measuring) collected from animal studies when projected to humans. Since the first development of 3D neuronal cell cultures (organoids) less than 10 years ago, efforts have focused on the standardization and reproducibility of organoids protocols. Even more recently, genetic engineering techniques (CRISPR) and sequencing methods such as single-cell RNA sequencing have enabled greater robustness and a deeper understanding of these personalized human-derived disease models.

Dr. Kristophe J. Diaz, Vice President Scientific Affairs for CurePSP, said, "The use of human-derived disease models as opposed to reliance solely on animal models is a crucial step in the study of complex neurodegenerative disorders. We are thrilled to participate in this landmark study with the Tau Consortium and thank the Semcers and Phillips for their support."

Patrick Brannelly, Managing Director of the Tau Consortium, added, "PSP has become a key focus in the study of neurodegeneration for several of the worlds best academic institutions. CurePSPs collaboration in this study is greatly valued. We look forward to our continued partnership through the Prime of Life Brain Initiative."

CurePSP was an early supporter of the work of the Neural Stem Cell Institute with grants to Dr. David Butler for his work in developing therapeutic agents to prevent tau protein accumulation with novel antibody-based reagents called intrabodies and to Dr. Mo Liu for her pioneering work in developing PSP organoids.

About CurePSP

CurePSP is the nonprofit advocacy organization focused on progressive supranuclear palsy, corticobasal degeneration, and other prime of life neurodegenerative diseases, a spectrum of fatal brain disorders that often strike during a persons most productive and rewarding years. Currently, there is no effective treatment or cure for these diseases, which affect more than 150,000 people in the U.S. alone. Since it was founded in 1990, CurePSP has funded nearly 200 research studies and is the leading source of information and support for patients and their families, other caregivers, researchers and doctors, and allied healthcare professionals. CurePSP is based in New York City. Please visit http://www.curepsp.org for more information.

About the Rainwater Charitable Foundation

The Rainwater Charitable Foundation was created in the early 1990s by renowned investor and philanthropist Richard E. Rainwater. The foundation supports a range of different programs in K-12 education, medical research, and other worthy causes. In order to deliver on its mission to accelerate the development of new diagnostics and treatments for tau-related neurodegenerative disorders, the Rainwater Charitable Foundation Medical Research team manages the Tau Consortium and the Rainwater Prize Program for advances in neurodegenerative disease research. With more than $100 million invested to date, Rainwater support has helped to advance eight treatments into human trials. For more information, please visit http://www.tauconsortium.org.

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Contacts

David Kemp kemp@curepsp.org 802-734-1185

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