How Are Funders Stepping Up in the Fight Against Still-Untreatable Neurodegenerative Diseases?



Nowhere is the need greater for new therapies than in the group of disorders known collectively as neurodegenerative diseases. Likewise, there are few areas where health philanthropy has more potential to make an outsized impact. As a new year begins, we take stock of some of the recent giving in this area of medical care and research.

Neurodegenerative diseases (NDs) afflict millions of people worldwide. They include ALS, multiple sclerosis, Alzheimer's, Parkinson's and Huntington's diseases. What these conditions have in common is a progressive degeneration and ultimately, the death of neurons. Neurons allow the brain and nervous system to communicate with each other; when these cells die, the patient loses the associated physical or cognitive function, while continued deterioration ultimately leads to death. These are terrible afflictions for which no effective therapies exist. But as we’ve reported extensively, funders are working hard to change this dismal situation. In fact, just in the past few months, we’ve seen over $125 million in collective grants to ND research.

Breaking Down Research Silos

Leading the pack is the Chan Zuckerberg Initiative (CZI), which announced $52 million in funding in December to launch its CZI Neurodegeneration Challenge Network, an interdisciplinary research effort studying the underlying causes of neurodegenerative disorders. The initiative is providing 17 awards of $2.5 million each to early-career investigators and nine awards of just over $1 million each to collaborative research teams. 

A focus of the Challenge Network is to “break down silos” to bring together researchers from different fields within the subject of neurodegeneration. Traditionally, neurodegenerative diseases have been studied as specific diseases, with specialized fields of research, funding, support and infrastructure. That specialization is hardly surprising, but medical science has been changing up these narrowly focused approaches. 

Much as cancer researchers are increasingly moving away from a specialized, “organ-centered” view of cancer (i.e., lung cancer, pancreatic cancer, etc.) toward an appreciation of cancers that share similar biology and mechanisms, the Challenge Network is also trying to move away from the disease-focused approaches in ND toward an understanding of these disorders as a class. Researchers hope that various ND conditions may respond to some of the same treatments. 

This funding is timely, as studies of NDs are increasingly benefiting from recent advances in genetics that have pointed to the role of the immune system and inflammation. One researcher awarded $2.5 million from the CZI is Ivan Marazzi, Ph.D., of Icahn School of Medicine at Mount Sinai in New York City. Marazzi showed that certain mutations that increase the risk for infections may also boost risk for NDs, including ALS. Another CZI grantee, Isaac Chiu of Harvard Medical School, will investigate the role that gut bacteria play in regulating neurodegeneration in ALS. (We reported last year on CZI support for research on the gut, in collaboration with the Helmsley Charitable Trust.)

Lots of Funding Will Go To Alzheimer’s

While ALS is relatively rare, Alzheimer’s disease is already a global health crisis, with estimates suggesting that 130 million people will be affected by 2050. Along with CZI, several funders are throwing in to develop effective therapies to treat the disease. Cox Enterprises and the James M. Cox Foundation is giving $23.7 million to Emory University in Atlanta toward establishment of a program for the study and treatment of early declines in memory. Mild cognitive impairment (MCI)—a distinct, early decline in memory and the ability to think—is often a precursor to Alzheimer’s disease. 

The Mild Cognitive Impairment Empowerment Program will focus on research, clinical care, caregiver support, and empowering patients to take an active role in their health and wellness. Emory will work in partnership with the Georgia Institute of Technology's Institute for People and Technology and the SimTigrate Design Lab in Georgia Tech's College of Design to improve quality of life for MCI sufferers and their families and develop more effective, evidence-based therapies.

Emory is also the beneficiary of a $25 million gift from the Goizueta Foundation toward its Alzheimer’s Disease Research Center (ADRC). The grant will fund the ADRC development of a clinical trials unit and to support the Neuroinflammation Discovery Unit. In the clinical trials unit, investigators will expand testing of new drug treatments, including industry-sponsored trials. In the Neuroinflammation Discovery Unit, investigators will study new approaches to combat neuroinflammation and disease progression.

Last year, we reported that the Paul G. Allen Frontiers Group and American Heart Association, through their American Heart Association-Allen Initiative in Brain Health and Cognitive Impairment, awarded a team at Salk Institute $19.2 million over eight years to investigate Alzheimer’s disease and age-related cognitive decline, and to seek new therapies.

Related: Head and Heart: An Unusual Partnership to Break New Ground in Biomedical Research

Much like CZI, the Salk team is taking a whole-body approach to the study of the disease. A multidisciplinary team of 10 Salk scientists representing the fields of metabolism, immunology and inflammation, genetics and epigenetics, and protein analysis, believe that Alzheimer’s and other age-related brain disorders are triggered not by a single event, but by a failure of interdependent biological systems that break down with age. Failure in any one system can cause a domino-like crash that results in devastating brain disorders like Alzheimer’s.

How About Parkinson’s?

Parkinson’s disease (PD) affects more than 10 million people worldwide. As we recently reported, there has been a lot of activity among researchers and funders in PD, including the Parkinson’s Foundation commitment of $6.2 million across 53 research grants and the Michael J. Fox Foundation for Parkinson's Research (MJFF) adding to its $800 million investment in translational and early clinical research. In late November, MJFF announced another 53 grant awards totaling more than $8 million.


Lastly, the foundation of UCLA alumni and donor Steven Gordon made a $25 million grant to his alma mater in late October to launch an initiative to research treatments and care for Parkinson’s. The award will establish the UCLA Laurie and Steven Gordon Commitment to Cure Parkinson’s Disease at the David Geffen School of Medicine. The money will fund basic research and endow five faculty chairs in fields related to Parkinson’s.

Making a Difference

We have often reported how critical philanthropy is to biomedical research, especially in backing riskier research and new ideas or younger investigators who often struggle to find support, as well as supporting work on diseases that affect comparatively few people. Public funding of biomedical research in the U.S. is dominated by one funding agency: The National Institutes of Health. The NIH annual budget is more than $30 billion—no other source of research funding in the world comes close to that figure. However, only a sliver of that funding goes to neurodegenerative diseases. NIH funding for Alzheimer’s has reached $1.9 billion annually, but still lags far behind money for cancer and HIV/AIDS (worthy causes though they are.) Compounding this is the highly competitive nature of securing a grant—the NIH funds only 5 to 7 percent of grant applications it receives.

Given this funding landscape, philanthropy’s role in backing ND research is quite meaningful. And a major strength of such grantmaking is the long time frame that funders often bring to their work, which is a must for backing biomedical research. For example, CZI’s founders, Mark Zuckerberg and Priscilla Chan, have talked about carrying on their battle against all disease through the end of the century.