Inaugural Rainwater Prize Winners Focus on Alzheimer’s and Other Brain Research

An eminent Alzheimer’s disease (AD) scientist, Michel Goedert, and an early career genomic engineering investigator, Patrick Hsu, are inaugural winners of the Rainwater Prize Program that acknowledges leading work in brain research.

Such work includes groundbreaking efforts to advance new treatments for neurodegenerative disorders, including Alzheimer’s, that are associated with tau protein accumulation in the brain. Managed by the Rainwater Charitable Foundation, the newly established prize is also designed to attract researchers to the tauopathy field.

Program leader at the Medical Research Council Laboratory of Molecular Biology in the United Kingdom, Goedert received the Rainwater Prize for Outstanding Innovation in Neurodegenerative Research. That honor nets him $250,000 for his contribution to the understanding of tau-related diseases.

“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,” Goedert said in a news release. “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.”

Goedert received wide acclaim in his field by demonstrating that tau is an important part of the paired helical filaments of Alzheimer’s, and uncovering the tau isoforms expressed in the human brain. Since then, he has helped establish the centrality of the abnormal collection of tau protein to tauopathies. He and his team also identified one of the first mutations in the gene that encodes tau — MAPT — that causes inherited frontotemporal dementia.

As it does to some extent in other neurodegenerative disorders, tau figures prominently in Alzheimer’s, a disease characterized by the protein plaques and tangles thought to be responsible for brain cell death and tissue loss. Tangles are twisted (misfolded) fibers of tau, which normally plays a role transporting nutrients through nerve cells. The tangled tau proteins disrupt that transport, ultimately starving and killing cells. There are several experimental compounds being developed in AD that target tau protein tangles.

Hsu, the other inaugural winner, is an assistant professor of bioengineering at the University of California, Berkeley, with a specialty in the field of genome engineering. Awarded the Rainwater Prize for Innovative Early-Career Scientists, Hsu was awarded $150,000 to support his work.

During graduate training at Harvard University, Hsu conducted some of the earliest investigations using CRISPR-Cas9, a technology that enables genome editing through DNA sequence alterations. His Salk Institute lab discovered ribonucleic acid-targeting CRISPR systems, tools that Hsu used to ultimately target mutations in MAPT related to frontotemporal dementia. He hopes to enhance the capabilities of CRISPR technology, focusing on genetic defects that can put individuals at risk for neurodegenerative disease.

“The Rainwater Prize is a special honor that inspires my team to continue creating new technologies that could impact brain disorders,” Hsu said. “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.”

Goedert and Hsu will present their findings in February at the Tau 2020 Global Conference in Washington, D.C.

In addition to the categories won by the two scientists, the program includes the Rainwater Milestone Prize for Advances in Tauopathy Research, which awards up to $2 million for work contributing to the understanding of tau-related diseases. The largest award — up to $10 million — is the Rainwater Breakthrough Prize for Effective Treatments in Progressive Supranuclear Palsy.