RCF Announces Inaugural Awards for Treating Neurodegenerative Disease

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

The Rainwater Prize Program was created to encourage and reward scientific progress toward new treatments for neurodegenerative diseases related to the accumulation of tau protein in the brain.

RCFs medical research program seeks to accelerate the development of treatments and eventual cures for tau-related neurodegenerative diseases. These tauopathies affect more than 50 million patients who have been diagnosed with Alzheimer's disease, chronic traumatic encephalopathy (CTE), progressive supranuclear palsy (PSP), and many other forms of neurodegenerative disease.

The Rainwater Prize Program aims to accelerate scientific progress by encouraging additional research into neurodegenerative diseases, attracting new researchers to the tauopathy field, and awarding scientific achievements that lead to new, effective treatments. The Rainwater Prize Program is divided into three prize categories:

1. The Rainwater Breakthrough Prize for Effective Treatments in PSP
2. The Rainwater Milestone Prize for Advances in Tauopathy Research
3. The Rainwater Prize for Outstanding Innovation in Neurodegenerative Research

Renowned investor and philanthropist, Richard Rainwater, was diagnosed with PSP in 2009. In addition to starting the Tau Consortium, a highly collaborative working group of world-class scientists, he also dedicated funds for the Rainwater Prize Program with the goal of accelerating milestone achievements and breakthroughs in neurodegenerative disease research.

The foundation is offering up to $10 million for groundbreaking discoveries, making it the largest prize program for brain research ever created.

"This is a significant milestone for the Rainwater Charitable Foundation, and we are thrilled to collaborate with both of the inaugural honorees," said Todd Rainwater, trustee at RCF. "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.”

Dr. Goedert, programme leader at the Medical Research Council Laboratory of Molecular Biology, initially detected that tau is an integral component of the paired helical filaments of Alzheimer's disease and discovered the six tau isoforms that are expressed in the human brain. 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 (FTD) 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 Dr. 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. During his graduate training at Harvard University, Dr. 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 FTD. At Berkeley, he is focusing on expanding the capabilities of RNA-based CRISPR systems and studying 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 Dr. 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.”

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 Dr. Goedert and Dr. Hsu were chosen based on their research, leadership, mentorship, and overall contributions to the scientific community.

Dr. Goedert and Dr. Hsu 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.

About the Rainwater Charitable Foundation's Medical Research

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. With nearly $100 million invested to date, the Rainwater family has helped to advance eight treatments into human trials. For more information, visit https://tauconsortium.org/.