A Funding Leader Lays Out Key Priorities in Neuromuscular Disease Research

If I had to pick which disease research funder has gotten its name out in front of the most IP readers in their lifetimes, the Muscular Dystrophy Association (MDA) would be near the top of the list. While not terribly large — it had $63 million in total revenue, according to its most recent Form 990 — the organization’s name was splashed on Americans’ television screens for over 40 years thanks to Jerry Lewis’ star-studded Labor Day telethons.

The Chicago-based MDA is “committed to transforming the lives of people affected by muscular dystrophy, amyotrophic lateral sclerosis (ALS), and related neuromuscular diseases,” and has committed more than $1 billion to accelerate the discovery of therapies and cures since its inception in 1950. It disbursed $14 million in grants for the 2021 calendar year, with support mostly flowing to organizations for research and “medical diagnosis,” and it lists open funding opportunities on its site. The association also provides clinical care and support to families living with neuromuscular disease.

In 2010, the final year with Lewis as host, the MDA’s 22-hour telethon raised $59 million. He passed away seven years later. In 2020, actor and comedian Kevin Hart picked up where Lewis left off, hosting a streamed telethon that reached over 100 million viewers and raised $10.5 million in just two hours. As one would hope, this steady stream of support has enabled the researchers to bring promising treatments to market in the past decade.

I recently reached out to Dr. Sharon Hesterlee, the MDA’s chief research officer, for a forthcoming State of American Philanthropy white paper on funding for neurological research organizations, and asked her what challenges keep her up at night. “The first is a good problem to have,” she said. Clinicians in the neuromuscular disease field “who have not previously had any treatment options for patients are now, due to the approval of 12 new treatments in the last 10 years, having to navigate complex administration and reimbursement scenarios.”

One issue, Hesterlee said, is that researchers’ efforts to conduct post-approval studies are hampered by the lack of an “infrastructure to do rigorous data collection in a consistently global way.” Lacking a common infrastructure, organizations develop their own databases “in ways that are duplicative, expensive and ultimately produce data silos.”

Hesterlee joined the association in 2019 with over 20 years of experience in the pharma, biotech and nonprofit sectors, including a previous 10-year stint with MDA’s research and venture philanthropy teams. Prior to rejoining MDA, she served as the executive vice president for portfolio development and head of neuromuscular programs at gene therapy company Asklepios Biopharmaceuticals, Inc.

Given her resume, Hesterlee is well-positioned to comment on how pandemic-era innovations are pushing the field forward. Arguably the biggest innovation involves researchers’ pivot to video or activity monitoring devices. Normally, patients would have to show up to a site in-person to participate in studies, but during the pandemic, researchers were able to collect data remotely from wearable devices. “The field of remote data collection has really started to mature, and investigators and sponsors have become more comfortable with this mode of collection,” she said.

This approach also advances funders’ goal of diversifying ethnic and racial demographic representation in clinical trials. According to Hesterlee, researchers’ pandemic-era “move toward remote and decentralized trials may actually make studies more accessible to diverse populations who faced challenges with participating in more traditional research requiring travel, time off work, and access to an institution active in research.”

MDA remotely monitors the diversity of participants at clinical sites participating in its neuroMuscular ObserVational Research clinical database. Hesterlee said she and her colleagues work to benchmark current levels of diversity and hypothesize about why participant diversity still lags in places where they’d expect it to be higher. Then they respond accordingly. For example, if MDA reps discover that people aren’t able to take time off work during standard clinic hours, “we could pilot some alternative hours at different sites, obviously in partnership with those sites,” she said.

Hesterlee pointed to a handful of other priorities, the first of which applies to all neurological conditions included in our upcoming white paper — the need for more reliable and affordable diagnostics. “We still have a large number of people in particular disease areas like limb-girdle muscular dystrophy, congenital muscular dystrophy, congenital myopathy, and Charcot-Marie-Tooth (CMT) disease who remain undiagnosed,” Hesterlee said, before noting that MDA has been funding research efforts to address this problem.

All of which brings us back to one of the ripple effects from the “good problem” that is the proliferation of new treatments across the past 10 years. Hesterlee said that MDA and several organizations in the neuromuscular space with large clinical databases are discussing joining forces to create a publicly available data source that would let researchers access and share data across a standardized infrastructure.

This effort reminded me of a question posed by Kavli Foundation Life Sciences Director Amy Bernard in a chat back in October. “Who fills that gap of making sure a researcher in France can compare their data with, say, somebody in Australia?” Bernard asked. (The answer, just in case there’s any confusion, is philanthropy.)

Funders are also grappling with how to keep up with an expanding range of activity across multiple organizations to avoid duplicating their efforts. To address this problem, MDA is partnering with the CMT Association and the CMT Research Foundation to co-fund CMT research projects. This work dovetails with Hesterlee’s sense that the field is becoming more open to collaboration. “It’s less common for organizations to have closed meetings,” she said. “We invite one another to our meetings now to encourage these types of collaborations, and I predict we will see larger-scale efforts going forward.”

For additional insights gleaned from our research for the upcoming white paper on funding for neurological research organizations, check out previous chats with leaders at CURE Epilepsy, the Christopher and Dana Reeve Foundation and the ALS Association.

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