🔒Funding research: While UMass Chan celebrated an historic donation and cemented its position as a leader in ALS research, the school raised red flags about planned federal cuts

In 2014, it was hard to log into any social media platform and not see your neighbors and Hollywood starlets alike pouring gallons of frigid water over their heads, taking part in the viral Ice Bucket Challenge.

The trend in which participants tagged each other in videos, daring them to either donate to ALS research or dump a jug of ice water over their heads, generated an unprecedented $115 million for research of the progressive neurodegenerative disease, according to Virginia-based research and advocacy nonprofit The ALS Association.

Eleven years later, UMass Chan Medical School received a similarly staggering donation when the Florida-based Paul J. DiMare Foundation gifted the Worcester school $35 million for the research of neurodegenerative and genetic diseases, particularly ALS. The donation was the third largest in the history of the entire University of Massachusetts system.

The gift emphasizes the great need for funding for the disease, said Dr. Robert Brown, UMass Chan professor of neurology and director of the Program in Neurotherapeutics.

And these private donations may soon become significantly more important as the federal government is weighing funding cuts to health research: cuts so large that a single donation like the one from the DiMare Foundation can’t make up for the loss.

$35M from a patient

The DiMare Foundation is named after the late DiMare family patriarch who, while he was ill, was keenly focused on assuring the acceleration of discovery of ALS, said UMass Chan Chancellor Dr. Michael Collins.

“Many patients, when they get a difficult diagnosis, they want to be sure that people are doing everything they possibly can to try to find a cure or a therapy for a disease. I don't think he was any different than that, from what I'm told,” said Collins.

While in treatment, DiMare became a patient of Brown, a pioneer in the world of ALS research.

In 1993, Brown led a team of researchers who discovered the first gene linked to the inherited form of ALS. Since then, more than 50 ALS genes have been identified, each offering researchers yet another option for treatment, said Brown.

“Each defines a step or a pathway in the process of motor neuron death in the disease, and therefore, each one also defines potential targets for treatment,” he said.

Thirty years after the gene’s discovery, the first therapy for ALS linked to the gene was approved by the U.S. Food & Drug Administration, a timeline embodying the long journey from concept to consumer.

While the pipeline for developing ALS therapy is full, researchers need definitive proof for each potential therapy, said Brown.

The difficulty in curing ALS

One of the greatest obstacles in ALS research is that no one knows what causes the disease, said Kuldip Dave, senior vice president of research at The ALS Association.

“When the biology is not known, you can't really model it really well in a lab,” Dave said.

In addition, researchers have to navigate the lack of good preclinical models. The cell and mice models used in research today are not necessarily good representations of what is happening in the disease with humans.

More than 95% of clinical trials fail, due to this lack of reliable biomarkers and the unknown cause, he said.

Researchers are working on three elements of ALS research, he said: discovering the disease’s cause, drug development, and clinical trials; capacity building and data and sample collection; and creating therapies to help people today.

All of this research costs millions upon millions of dollars, and that’s where the beauty of donations like the $35 million from the DiMare Foundation come into play, said Collins.

“Having funds like this with which faculty members can access without having to participate in that competitive grant process is really wonderful because they can work on ideas that are novel, and perhaps haven't already been … proven to work” he said.

With the proper investments, the UMass Chan endowment fueled by these donations will grow and further its reach.

“Hopefully someday it'll be a billion, and that spins off monies that the campus will have forever,” said Collins.

Trump’s planned cuts

Grants and donations go to a wide range of costs that both directly and indirectly make research possible, said Dave.

Direct costs are defined as those that can be identified specifically with a particular sponsored project, according to the National Institutes of Health. Examples of these costs include hiring research technicians, purchasing test tubes, and acquiring animal models, said Dave. These funds are generally granted directly to a scientist for the intended research.

While exactly how the DiMare donation will be delegated is still to be determined, Brown said it would allow UMass Chan to support a number of direct costs, including expanding its faculty and investigators in neurodegenerative disease. Funding can also support the hiring of those doing basic work, also known as fundamental or pure work, including geneticists and scientists.

Without those researchers who devise the technology for discovery of disease, finding the first ALS gene would not have been possible, said Brown.

“Part of the whole enterprise is making sure that the basic underpinning is indeed supported,” he said.

On the other hand, indirect costs, or overhead costs, are those that cannot be readily identified with a project. These can range from building maintenance and utilities to purchasing computers, said Dave.

Conversations around direct and indirect costs have become part of everyday lexicon since early February when the President Donald Trump Administration announced it would cap NIH funding for indirect costs to 15% for biomedical and public health research.

UMass Chan expects the Trump cuts to have a significant impact on its operations and expects to lose about $50 million in annual revenue. While a federal judge temporarily blocked the cuts, universities and research institutions are anxiously waiting in limbo on next steps.

“These types of massive cuts and attacks on universities, it's going to have an impact on the science for many decades to come,” said Dave.

The federal government is the largest funder of ALS research in the U.S., he said; all major discoveries in ALS happened because someone had an NIH grant.

The void the cuts would create would be impossible to fill by any single donation, including the likes of the DiMare donation.

As they await news of the Trump cuts, Brown and his colleagues at UMass Chan are taking research from petri dish to mouse models, to hopefully in the near future reach human trials.

Brown’s long-term goal holds steadfast: to cure the disease.

“It’s a bold ambition to be sure, but an important one,” he said. “We would like our grandchildren to ask in 20 years, ‘What was that disease?’”

Mica Kanner-Mascolo is a staff writer at Worcester Business Journal, who primarily covers the healthcare and diversity, equity, and inclusion industries.