Progress via Partnership
NINDS, Nonprofits Gather Virtually
NINDS recently held its 14th nonprofit forum, “Progress through Partnership,” drawing its largest crowd, albeit virtually, to date. More than 200 participants gathered in cyberspace via Zoom on June 29-30 for the event that provided an opportunity for patient advocacy groups to connect with and learn more about NINDS, NIH and each other.
“This is probably the most important meeting of the year for us,” said NINDS director Dr. Walter Koroshetz. “It’s a meeting where we exchange ideas with nonprofits—the organizations that represent the patients we serve. We go to scientific meetings where we hear about the great science, but NINDS is not an institute that’s founded to improve the lot of scientists. It’s an institute that’s trying to improve the lot of people who suffer from neurological disorders. So, the scientists, the NINDS folks and the people on this meeting are really partners in this struggle to bring new treatments to patients with neurological disorders and also to prevent them.”
Koroshetz updated attendees on the current status of NINDS amid the Covid-19 pandemic. “NINDS is open for business,” he said. “We are on schedule in terms of reviewing grants and sending out funds to scientists all over the country to perform research. The problem is that research itself is on hold because of the pandemic. Some labs here at NIH are starting to reopen, but in terms of review and moving the research forward with funding, we are on our usual schedule.”
The meeting kicked off with a forum favorite—a nonprofit’s success story in therapeutic development. In her keynote presentation, “How CureSMA has made progress by building community and collaborations,” Dr. Jill Jarecki, chief scientific officer of CureSMA, shared the remarkable scientific progress that has been made in SMA drug development.
Spinal muscular atrophy, or SMA, refers to a group of genetic diseases that destroy motor neurons. Currently there are two FDA-approved drugs to treat SMA—Spinraza and Zolgensma. Jarecki saId a third drug, Risdiplam, should be approved in August.
“One of the most important roles I think an advocacy group can have is encouraging collaboration and bringing different groups together,” she said. She provided examples of ways CureSMA has pursued collaboration: having annual SMA conferences, creating working groups from academia and medicine and working with industry partners.
Two members of the forum’s executive planning committee—Geraldine Bliss, president and co-founder of CureSHANK, and Paul Gross, chairman and founder of the Cerebral Palsy Research Network—led a session on “Covid Challenges in Nonprofit and Research Communities,” which focused on results from a survey the committee launched among the nonprofit community in the weeks leading up to the forum. The survey looked at the impact of Covid-19 on research, patient support and nonprofit revenue.
“Many of our organizations are realizing reductions in revenue at this time and it comes at such a complex time when researchers need our support,” Bliss said. “Their labs have been closed or scaled down, clinical trials and clinical research studies have been paused and our patients need us more than ever.”
The meeting also featured two panel discussions on innovative therapeutic developments for rare disorders and ways to develop clinical trials for rare diseases. The sessions, “Challenges in Therapeutic Development” and “Clinical Trials: Precision Approaches to Rare and Not So Rare Disorders,” stressed the importance of building registries, developing natural history studies, sharing data and engaging industry and pharmaceutical companies.
The next morning, NINDS deputy director Dr. Nina Schor provided an overview of NINDS’s current strategic planning process.
“It takes a village to do this,” she said. “The thing about strategic planning is that the process is almost more important than the outcome. The way people get together and hear each other’s views and get each other’s input and troubleshoot together to work out the problems and the issues and the challenges really changes an organization going forward.” She welcomed input on the plan from the nonprofit community.
Nonprofit representatives got an opportunity to meet informally with NINDS program staff in breakout sessions on both days.
“We travel this road together,” said Koroshetz. “We are all science advocates. We are all pushing to improve the understanding of the brain and nervous system so we can get new therapies and we do that together.”
Recordings of both days of the forum are available on NIH Videocast—https://videocast.nih.gov/watch=38056 and https://videocast.nih.gov/watch=38058.
Added Workshop Touts Power of Storytelling
This year’s nonprofit forum featured an additional workshop titled “The Power of Storytelling,” held on July 16; it emphasized the importance of personalizing narratives in research, medicine and patient care.
The virtual session included a presentation by Liz Neely, executive director of The Story Collider, a nonprofit organization devoted to telling true, personal stories about science. She described stories as “powerful framing devices that help humans make sense of the world,” and defined them as “believable characters experiencing meaningful events who must cope with the consequences.”
Neely encouraged nonprofit representatives to add stories to their strategic communication toolkits. “Stories are what help humans connect,” she explained. “We all have stories. I hope you tell them and I hope you tell them well.”
A special highlight during the workshop was a story shared by Dr. Sonia Vallabh, an associate scientist at the Broad Institute, of how a genetic prion disease inspired her and husband Dr. Eric Minikel to switch their career paths to become biomedical research scientists focused on developing preventive drugs for prion disease.