To Crack the Puzzles of Alzheimer's, a Foundation Takes a Laser Approach

photo: Juan Gaertner/shutterstock

photo: Juan Gaertner/shutterstock

As Alzheimer’s disease inflicts an ever-rising toll on aging populations in the U.S. and overseas, the race is on for research breakthroughs that can slow the devastating march of this and other neurodegenerative diseases. The latest good news on this front is that the Rainwater Charitable Foundation, one of the largest independent funders of neurodegenerative disease research, recently announced that it is launching the Rainwater Prize Program. The foundation is offering up to $10 million for ground-breaking discoveries, making it the largest prize program for brain research ever created.

A Laser Focus

While there are other foundations working in the space, such as the Alzheimer's Association and the BrightFocus Foundation, what sets Rainwater apart is its laser-focus on tau proteins, which are abundant in nerve cells and perform the function of stabilizing microtubules. Neurodegenerative diseases such as Alzheimer’s disease may be prevented by stopping tau from “turning rogue.” The Rainwater Prize Program is designed to “encourage and reward scientific progress toward new treatments for neurodegenerative diseases related to the accumulation of tau protein in the brain.”

Tau is also found in other diseases, collectively known as tauopathies, which affect more than 50 million patients and include Alzheimer's disease, chronic traumatic encephalopathy (CTE), progressive supranuclear palsy (PSP), and many other forms of neurodegenerative disease.

The Rainwater Charitable Foundation was created in the early 1990s by investor and philanthropist Richard E. Rainwater. In 2009, Rainwater was diagnosed with PSP, which has no known cause, treatment or cure. As a result of this diagnosis, the foundation expanded its focus to include the emerging field of PSP medical research. Mr. Rainwater assembled a team of over 30 principal investigators, named the Tau Consortium, to research the causes, progression and potential treatments of PSP. Since founding the Tau Consortium in 2009, the foundation has committed over $100 million to the program and has helped to put eight treatments into human trials.

The new Rainwater Prize Program is divided into three prize categories (with any one individual or team eligible to win up to $10 million):

  • The Rainwater Breakthrough Prize for Effective Treatments in PSP will be awarded when a breakthrough treatment is advanced to clinical trial according to the following eligibility: $2 million will be awarded to an FDA-approved treatment that meaningfully extends better quality of life for patients; $4 million will be awarded to an FDA-approved treatment that cures PSP early in progression; and $10 million will be awarded to an FDA-approved treatment that prevents PSP and/or reverses disease damage.

  • The Rainwater Milestone Prize for Advances in Tauopathy Research awards up to $2 million to investigator(s) who provide a significant contribution to the understanding of tau-related diseases by addressing specific critical gaps in technology and disease knowledge that will help the scientific community develop effective treatments. 

  • The Rainwater Prize for Outstanding Innovation in Neurodegenerative Research recognizes investigator(s) whose work is considered a significant contribution to the understanding of tau-related diseases. This $250,000 award will be given on an annual basis starting in 2019.

A Pivotal Time For This Research

This funding has become available on the heels of new research. A recent study by researchers from Drexel University College of Medicine suggests an alternate view of the role of tau.  The current understanding of tau is that it works much like a railway. Tau is mostly present in neuronal axons and is attached to microtubules. In this analogy, the microtubules are the railroad tracks and the ties are tau. Tau and microtubules work together to move the cargo down the long distances of axons. The Drexel research suggests that tau's actual role in the neuron is to allow microtubules to grow and remain dynamic.

"The popular theory suggests that patients with neurodegenerative diseases are losing microtubules because they are becoming less stable. What our study suggests is that, with the depletion of tau, patients are, in fact, losing the dynamic regions of microtubule," said Peter Baas, PhD, a professor in the Department of Neurobiology and Anatomy at Drexel College of Medicine and the study's principal investigator. This new discovery “suggests that microtubule-stabilizing drugs currently in clinical trials may not be effective in treating Alzheimer's and other tau-based neurodegenerative diseases,” said Baas.

Reasons for Hope

With an estimated 5.7 million Americans afflicted with Alzheimer's dementia, a population projected t0 grow to 13.8 million by mid-century, this research is imperative. Perhaps the most encouraging news in neurodegenerative disease research is the speed at which new information is being gathered. For example, Alzheimer’s disease was first identified in 1906, but it wasn’t until 1986 that tangles of tau were linked to the disease. Now, due to advances in technology and techniques, knowledge about these diseases has grown exponentially over the last few decades.

Dr. Richard Carmona, former U.S. surgeon general and the chair of the Rainwater Prize Program, says, “It is hard to fathom that we have such inadequate treatment options for the millions of patients diagnosed with a tauopathy every year. We need more funding and creative initiatives such as the Rainwater Prize Program to increase the amount of research and accelerate scientific advances. These millions of patients need hope for a better life.”