Payoff: Years of Investments in Parkinson’s Research is Yielding New Breakthroughs and Momentum

Photo: Ocskay Mark/Shutterstock

Photo: Ocskay Mark/Shutterstock

It’s not often that you hear the words “good news” when it comes to Parkinson’s disease. But recently, there’s been a lot on the positive side to report—much of it thanks to private philanthropy.

First, researchers from Johns Hopkins announced they have developed an experimental drug similar to compounds used to treat diabetes that’s been shown to slow the progression of Parkinson's and its symptoms in mice. Even better news, the drug is expected to move to human clinical trials this year.

Also on the therapeutic front, a Japanese research team announced that after a successful round of animal trials, it will start human clinical trials for a new Parkinson’s disease treatment involving stem cells.

There’s more good news. Due to the tireless advocacy of regular folks and funders alike, the federal government is finally bumping up its funding for PD. Throughout 2018, the Parkinson's community made a strong push for their funding priorities. Thousands of people with Parkinson's and their loved ones sent lawmakers more than 46,000 emails advocating funding for the PD research database and the National Institutes of Health (NIH). Their efforts were rewarded when President Trump signed into a law a provision that includes a $2 billion funding increase for the NIH, which will include more money for Parkinson’s. A previous NIH funding increase last year also included greater support for PD research.

Meanwhile, we recently reported on the Parkinson’s Foundation commitment of $6.2 million across 53 research grants. And not too long ago, the Michael J. Fox Foundation for Parkinson's Research (MJFF) added to its $800 million investment in translational and early clinical research with 41 new grant awards totaling more than $6 million.

It’s hard to overstate the importance of private funding in the battle against Parkinson’s. MJFF funding has often been behind gains made by researchers against the disease. For example, MJFF funded part of the study on the drug foliglurax, which eases tremor, slowness and mobility problems associated with PD. MJFF also funded development of a glutamate therapy at Vanderbilt University—the brain chemical glutamate is significantly affected by Parkinson’s, and addressing this dysregulation could lead to major improvements in function for the estimated 1 million Americans with PD.


Hopefully, all this is just the beginning: Researchers backed by the latest round of grants are hot on the trail of other potential breakthroughs.

The MJFF research strategy is based on a four-pronged approach: understanding, measurement, development of treatments, and tools. For example, through the understanding portfolio, the foundation funded OccamzRazor, which “will use its artificial intelligence platform to analyze the wealth of knowledge in decades of Parkinson's research data. The project's aim is to look at previously unknown or undervalued data that could lead to breakthroughs.

In the measurement area, a joint team from the University of Toronto and Yale PET Center will be developing an imaging agent for use in PET scans. The agent will “detect changes in the connections between brain cells (i.e., synapses), which could be used to diagnose brain diseases such as Parkinson’s and determine the effectiveness of therapies targeting synapses.”

An example of a treatment grant involves McGill University scientists, who are conducting preclinical tests to determine if the drug bitopertin may be an effective treatment for one of the most troubling symptoms of Parkinson’s, dyskinesia.

Lastly, MJFF is funding new tools to enable the development of drugs for important targets. (A full list of all the grants can be found here.)

Private philanthropy has always played a major role in advancing drug discovery and advocacy for PD. Now, with the federal government bringing even more research firepower to the table, there’s a growing sense of momentum and more talk that a cure for PD may be achievable sooner rather than later. Some 60,000 Americans are diagnosed with Parkinson’s each year. Ten million people worldwide are living with PD.

The PD story may be a useful lesson for other disease-focused funders: Commit for the long haul, keep moving the research forward, and when enough promising results bring significant improvements into view, lobby the government to redouble efforts to cross the finish line.