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In 2009, Richard Rainwater was diagnosed with Progressive Supranuclear Palsy (PSP), a rare neurodegenerative disease that causes serious problems with walking, balance, and vision. PSP is one of several neurodegenerative diseases classified as a tauopathy due to the accumulation of tau protein in the brain. Although there’s no cure for PSP, treatments are available to manage the signs and symptoms of the disease. Determined to help others suffering from PSP, Rainwater assembled a team of experts and deployed significant resources to accelerate research into the neurodegenerative disease.
Shortly before his passing in 2015, Rainwater established the Rainwater Prize Program. The Rainwater Prize Program recognizes scientific progress with a goal of increasing awareness of critical gaps in neurodegenerative research, attracting and retaining researchers to work on tauopathies and rewarding achievements that can lead to effective treatments for patients. The program consists of over $10 million in prizes broken out into four categories – two of which are awarded annually and two upon the achievement of specific milestones and breakthroughs.
“My father’s brilliance in his legendary investing career played through in how he invested in PSP research,” said Todd Rainwater, Richard Rainwater’s eldest son and a trustee of the Rainwater Charitable Foundation. “While he knew the discoveries emerging from his support may not come soon enough to benefit him, he believed effective treatments were on the way and wanted to be able to reward breakthrough scientists for their efforts.”
On April 27th, 2023, this year’s Rainwater Prizes for Outstanding Innovation Prize and for Innovative Early-Career Scientist – totaling $600,000 – were awarded at the Eurotau Conference in Lille, France.
The Outstanding Innovation Prize was awarded to a team: C. Frank Bennett, PhD, Ionis Pharmaceuticals; Don W. Cleveland, PhD, University of California San Diego; and Timothy M. Miller, MD, PhD, Washington University School of Medicine in St. Louis. Collaborators Bennett, Cleveland and Miller were recognized for their work over the past 20 years advancing antisense oligonucleotide (ASO) technology for the treatment of neurodegenerative diseases including amyotrophic lateral sclerosis (ALS), Huntington’s disease, Alzheimer’s disease and tauopathies.
Two days prior to the Rainwater Prize Ceremony, the FDA granted accelerated approval to Biogen/Ionis’ QALSODYTM (tofersen) for the treatment of SOD1-ALS patients using the ASO technology developed in part by this team. The results give hope that an effective treatment using this therapeutic approach for other neurodegenerative diseases, many of which are already in clinical trials, may be in sight for patients and their families.
In addition, Susanne Wegmann, PhD, DZNE (the German Center for Neurodegenerative Diseases) was awarded the Rainwater Prize for Innovative Early-Career Scientist. This award celebrates exceptionally productive scientists within their first 10 years of their professional appointment. Wegmann’s research program focuses on the interactions of the tau protein in cellular processes implicated in tauopathies like Alzheimer’s disease. Her research utilizes different experimental models and methodologies to discover Tau’s normal function and misfunction in the brain. She exemplifies the pillars of the prize: significant contributions to the field, leadership, and mentorship.
Previous prize recipients include Michel Goedert, Medical Research Council Laboratory of Molecular Biology; Patrick Hsu, University of California, Berkeley; David Holtzman and Celeste Karch, Washington University School of Medicine in St. Louis; Alison Goate, Icahn School of Medicine at Mount Sinai; and Martin Kampmann, University of California, San Francisco.
Nominations for the 2024 Prize Program are now open. To learn more and to nominate a deserving researcher, visit www.rainwaterprize.org.