EXOME

all the information, none of the junk | biotech • healthcare • life sciences

Rainwater Foundation Deploys Millions Toward Rare Brain Disease, PSP

Xconomy Texas — 

Dallas — A foundation started by a Texas billionaire announced Tuesday it will offer millions of dollars in awards to scientists who research neurodegenerative diseases such as Alzheimer’s and progressive supranuclear palsy.

The Rainwater Charitable Foundation, which was founded in the 1990s by billionaire investor Richard Rainwater, announced it will start to offer in 2019 an annual award of $250,000 to an investigator that contributes to the understanding of diseases that may be impacted by tau, a protein that has been implicated in some neurodegenerative diseases. It is believed abnormal clumping of tau in the brain’s nerve cells can cause cell damage related to a variety of diseases, including Progressive supranuclear palsy (PSP), a rare disorder that Rainwater died from in 2015. The foundation also announced another $18 million in awards for bigger discoveries—even cures—related primarily to PSP (more on that below).

And this is isn’t the first foundation to offer this kind of cash prize for brain research this week. On Nov. 5, National Instruments co-founder James Truchard announced a $5 million donation to the University of Texas at San Antonio in order to found the Oskar Fischer Project, which will give out $4 million to seven individuals who present promising ideas about the cause of Alzheimer’s disease and say they’ll work to prove it.

Notably, The Oskar Fischer Project isn’t requiring a scientific background from its applicants, instead seeking out a “genius” from any walk of life who may be able to make a difference by connecting previously unnoticed dots.

The Rainwater Charitable Foundation, meanwhile, is requiring applications for its prizes to have made previous “significant contributions to research in Tau-related diseases,” according to Amy Rommel, the manager of the Rainwater Prize Program. Most of the money from the Rainwater Foundation, which is based in Fort Worth, is being targeted toward making progress in PSP, a disease that causes problems walking and thinking and leads to other significant health problems, such as falling.

The exact cause of PSP isn’t definitively known, and there aren’t currently any effective treatments. But it is being studied by some major drug developers, including Biogen (NASDAQ: BIIB), of Cambridge, MA, which acquired an experimental drug (BMS-986168) from Bristol-Myers Squibb in 2017. And in February, Chicago-based AbbVie (NYSE: ABBV) signed a deal with Voyager Therapeutics (NASDAQ: VYGR), also of Cambridge, to develop gene therapy tools called adeno-associated viruses for Alzheimer’s and other neurological diseases, including PSP.

The Rainwater Prize Program will offer multi-million dollar awards for researchers whose treatments for PSP achieve varying levels of regulatory approval:
—$2 million for an FDA-approved treatment that “meaningfully extends better quality of life” for patients with PSP;
—$4 million for an FDA-approved treatment that cures PSP early in its progression;
—and $10 million for an FDA-approved treatment that prevents PSP or reverses damage done by the disease.

It’s of course a tall order to gain regulatory approval for any drug. The foundation is also offering prizes of up to $2 million for researchers whose work addresses gaps in the technology or knowledge of tau-related diseases, including PSP, Alzheimer’s, and Chronic Traumatic Encephalopathy (CTE). The amount awarded depends on the milestone that’s been achieved, Rommel wrote in an e-mail. (That’s in addition to the aforementioned $250,000 annual award the foundation plans to hand out.)

To be eligible, a person must have published, peer-reviewed tau-related research, which the foundation’s committee of scientific leaders will look over when considering who to pick for the awards. People can’t nominate themselves, though, and must get a third party to submit them as a candidate. The application process is open until April 26, and the foundation plans to begin judging for finalists in July, according to Rommel.

Richard Rainwater gained notoriety while investing the fortune of the Bass family of Texas, multiplying it from $50 million to $5 billion over 16 years, according to Fortune. He started his own investment company, Rainwater Inc., in 1986, according to the magazine.

Rainwater’s diagnosis of PSP came in 2009, when few resources were available for the condition. The disease is caused by damage to certain parts of the brain—again, tau is viewed as the culprit—which can cause stiffness and clumsiness that may seem similar to what patients of Parkinson’s disease experience, according to the National Institute of Health. PSP is more rapidly progressive than Parkinson’s, however, and problems with PSP patient’s eyes are common, from rapid eye movement to blurred vision, the NIH says. Before his death in 2015, Rainwater assembled a working group of scientists to study PSP, calling it the Tau Consortium, and dedicated the funds that would be used to start the Rainwater Prize Program.

In addition to its work in medical research, the Rainwater Charitable Foundation also supports work in education. The Rainwater family has invested more than $100 million to date on research into neurodegenerative disorders, which has helped advance eight experimental treatments into human trials, according to a news release.

That the Rainwater Foundation happened to announce its multi-million dollar award program the same week as the Oskar Fischer Prize program happens to be purely coincidental, Rommel says, adding that her group was unaware of Truchard’s announcement.

“Both foundations and individuals play a critical role in advancing options for treatment—and eventually, a cure,” Rommel wrote.