The annual NINDS Nonprofit Forum, taking place July 24-25, 2023, brings together organizations that represent the many neurological diseases within the NINDS portfolio. It’s a valuable venue for the organizations to collaborate and share lessons learned among themselves; and for NINDS to understand how we can most effectively work with organizations to address research needs and challenges. NINDS has organized the event since 2005. This year, we are excited to return to an in-person event while also employing web-based technology to enable virtual participation: the meeting will take place on the NIH campus in Bethesda, and virtual attendees will be able to watch the meeting through NIH videocast with new interactive capabilities.
The Forum addresses the importance of relationships between public, private, and nonprofit stakeholders to patient-centered research for neurological disorders. This year, the agenda will highlight the growing opportunities for the development of gene-targeted therapies including a keynote on gene replacement therapies by Terry Pirovolakis of CureSPG50.org, an organization to treat an ultra-rare condition, called Spastic Paraplegia Type 50, caused by a mutation in a single gene, AP4M1. In launching Elpida Therapeutics, he is working to deliver gene therapies to children with other rare neurogenetic disorders. Two other panels will continue discussions on the opportunities and hurdles of the translation of gene therapy to the clinic. Nonprofit Forum Executive Planning Committee member and President of the Friedreich’s Ataxia Research Alliance (FARA) Ron Bartek will moderate a panel on “Gene Therapy-Partnerships and Collaborations” with panelists from the Food and Drug Administration, National Center for Advancing Translational Sciences, Foundation for the National Institutes of Health, and Prevail Therapeutics. NINDS Program Director Dr. Jill Morris will moderate a panel entitled “Gene Therapy: Ethics in Cell Therapies and Dilemmas Researchers Face.” Panelists will include a gene therapy clinician-researcher, an applied bioethics researcher, and gene therapy patient advocate. With the acceleration of advances in gene therapy technologies as well as the FDA approval of numerous gene therapy products, it is critical that we prioritize such conversations between regulators, researchers, industry, and patient advocates. Continued close collaboration will be required to ensure gene therapy options are as safe as possible, made available as soon as possible and are accessible to those who can benefit.
The Forum is not only for NINDS staff to connect with the nonprofit patient organization community, but also fosters opportunities for nonprofit organization representatives to connect with one another. This year’s agenda will include two sessions to foster that sharing. “Nonprofit funding for Nonprofit Research” will be moderated by Dr. Penny Dacks, Executive Planning Committee member and Senior Director of Scientific Initiatives at the Association of Frontotemporal Degeneration. The panel will include speakers from the Parkinson’s Foundation, Target ALS, and others. A second panel, and recurring conference favorite, highlights “Success Stories”—this year focused on “Newly Approved Drugs and the Stages of Approval” with stories of drugs for ALS, Rett Syndrome and Friedrich’s Ataxia. Attendees learn from the successes and solutions of other organizations through their direct experiences, which helps advance efforts within their own organization. Learning from each other about how to navigate common issues is one of the most valuable benefits of the Nonprofit Forum.
Throughout the Forum, attendees will have opportunities to engage with each other and NINDS program staff and leadership. I will offer a “State of the Institute” address to the meeting attendees, who will also have the opportunity to meet with NINDS division directors and program staff followed by a poster session with over 40 in-person and virtual posters. Attendees are also welcome to sign up for 1:1 meetings with NINDS program staff who are focused on research relating to specific disorders.
On the second day, NINDS Acting Deputy Director Dr. Amy Bany Adams will reflect on the Forum and connect discussions to NINDS efforts to strengthen the engagement of people with lived experience of neurological disorders in research, a commitment outlined in our 2021-2026 strategic plan. The priorities of patients and their families are paramount, and hearing their perspectives throughout the scientific process will improve the impact of our research. Consistent with this commitment, the final two sessions will emphasize the patient-centered approach. Patient Advocate Christine Von Raesfeld, CEO of People with Empathy, will lead a session exploring the opportunities, hurdles and ethics of collecting, sharing, using, and respecting patient data with speakers from the National Organization for Rare Disorders (NORD) and the Critical Path Institute (C-Path). Our last Forum session will explore “Community Engagement Strategies to Boost Diversity Enrollment in Clinical Trials from the Pharma Perspective.” NINDS Program Director Dr. Cheryse Sankar will discuss NINDS efforts in this area and moderate a panel of speakers from pharmaceutical companies including Lilly, Genentech, and a patient focused nonprofit organization, as they discuss their efforts to use community engagement to diversify clinical trial participation.
The NINDS Nonprofit Forum always showcases the power of partnerships and collaboration, and the hybrid format of this year’s meeting will further strengthen those partnerships and allow for the development of new ones. Whether meeting in person or virtually, attendees can continue to support research that will provide a greater understanding of the brain and nervous system and for new and better treatments for neurological disorders. Across our various communities, including academic, government, industry, or nonprofit backgrounds, we can all make a difference together.
I commend the 2023 Nonprofit Forum executive planning committee, composed of representatives from nonprofit patient centric organizations and NINDS staff, whose hard work is a reflection of this annual event that coalesces speakers and sessions covering diverse topics, opinions, and expertise. Previous years have seen over 200 attendees from more than 130 nonprofits. And this year’s hybrid format may have even more attendees, a testament to our NINDS staff in enabling a broader reach for this exciting event. I look forward to seeing you there!