The Rainwater Charitable Foundation, one of the largest independent funders of neurodegenerative disease research, announced Oct. 22 Dr. Michel Goedert (Medical Research Council Laboratory of Molecular Biology, Cambridge, UK) and Dr. Patrick Hsu (University of California, Berkeley) as the first recipients of the Rainwater Prize for Outstanding Innovation in Neurodegenerative Research and the Rainwater Prize for Innovative Early-Career Scientists.

Launched in November 2018, the Rainwater Prize Program encourages and rewards scientific progress toward new treatments – and eventual cures – for neurodegenerative diseases related to the accumulation of tau protein in the brain, the foundation said in a news release.

The Rainwater Charitable Foundation was created in the early 1990s by the late 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.

With nearly $100 million invested to date, the Rainwater family has helped to advance eight treatments into human trials.

Dr. Goedert, Programme Leader at the Medical Research Council Laboratory of Molecular Biology, first made a name for himself by showing that tau is an integral component of the paired helical filaments of Alzheimer's disease and discovering the six tau isoforms that are expressed in the human brain, the news release said.

He has since helped establish the idea that the abnormal assembly of tau protein is central to tauopathies, and along with his colleagues, identified one of the first mutations in MAPT (the gene that encodes tau) that causes inherited frontotemporal dementia in humans.

"It is an honor to be recognized by a group of experts who understand the critical role played by the tau protein in many neurodegenerative diseases," said Goedert. "With this prize, my goal is to encourage other researchers to join us in further exploring the root causes of these diseases and eventually to partner in developing novel methods for prevention of disease."

Dr. Hsu, assistant professor in the Department of Bioengineering at UC Berkeley, is a rising star in the field of genome engineering, the Rainwater Foundation said.

During his graduate training at Harvard University, Hsu performed some of the earliest studies with CRISPR-Cas9, a technology that enables geneticists and medical researchers to edit parts of the genome by altering its DNA sequence.

At the Salk Institute, his lab discovered RNA-targeting CRISPR systems that enabled recognition and control over RNAs in living cells. He used his new tool to target MAPT RNAs to correct splicing imbalances associated with frontotemporal dementia.

At Berkeley, he plans to focus on expanding the capabilities of RNA-based CRISPR systems and study genetic defects that can cause neurodegenerative risk.

"The Rainwater Prize is a special honor that inspires my team to continue creating new technologies that could impact brain disorders," said Hsu. "The devastation of neurodegenerative disease became apparent at an early age when I witnessed my grandfather suffer from mild cognitive impairment followed by Alzheimer's disease – an experience that ultimately inspired me to dedicate my career to science.”

The Prize Program promotes four main prize categories, including the Outstanding Innovation ($250,000) and Early-Career ($150,000) Prizes to be awarded at the Tau 2020 Global Conference.

The third prize, the Rainwater Milestone Prize for Advances in Tauopathy Research, will award up to $2 million for investigators whose work significantly contributes to the understanding of tau-related diseases – by addressing critical gaps in technology and disease knowledge – that will help the scientific community to develop effective treatments.

The final and largest prize, the Rainwater Breakthrough Prize for Effective Treatments in Progressive Supranuclear Palsy (PSP), will award up to $10 million for FDA-approved treatments that extend good quality of life for patients, cure PSP early in progression, or prevent and/or reverse disease damage from PSP.

"This is a significant milestone for the Rainwater Charitable Foundation, and we are thrilled to collaborate with both of the inaugural honorees," Todd Rainwater, Trustee at the Rainwater Charitable Foundation, said in the news release.

"It is inspiring to work with individuals who share our commitment to advance cures and treatments for neurodegenerative diseases that are impacting millions of patients and families – like mine – across the globe. We are looking forward to continuing the legacy of our founder and my father, Richard Rainwater, by awarding researchers annually for their exceptional efforts in the field," Todd Rainwater said.

Colleagues and peers nominated the awardees based on published and peer-reviewed research related to tau and were selected by a committee of international scientific leaders from a wide range of fields and backgrounds.

Both Goedert and Hsu were chosen based on their research, leadership, mentorship, and overall contributions to the scientific community. They will present their findings in February at the Tau 2020 Global Conference, which is co-sponsored by the Rainwater Charitable Foundation, Alzheimer's Association and CurePSP.

Representatives from academia, industry, government, and the philanthropic sector are encouraged to attend.

More information on public registration can be found at: https://alz.org/tau2020/overview.asp

https://rainwatercharitablefoundation.org

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