|At left, NINDS director Dr. Story Landis (l) and Dr. Vicky Whittemore of the Tuberous Sclerosis Alliance discuss “NIH 101.” At right, Dr. Nancy Wexler (standing) of the Hereditary Disease Foundation participates in the nonprofit forum.
Representatives from 60 nonprofit organizations across the country recently joined NINDS at its fifth nonprofit forum, “Partnering to Advance Therapeutics for Neurological Disorders.” The meeting gave patient advocacy groups an opportunity
to learn from each other and about NIH and NINDS, provided them with an environment to share interests and allowed them to interact directly with program staff.
NINDS director Dr. Story Landis opened with “NIH 101: What It Is and Isn’t and How Council Works,” an overview that covered NIH’s mission and makeup, NINDS funding basics and a brief description of the NIH Blueprint for Neuroscience
“Basic scientists think NIH is about enabling them to discover exciting new facts about how the brain works and how the heart works, and people interested
in diseases think NIH is about learning about how to treat diseases,” she explained. “But it’s the marriage of that basic science and the application of that basic science that makes NIH unique.”
‘Take Off Your Disease Hat’
Dr. Vicky Whittemore, former vice president and chief scientific officer of the Tuberous Sclerosis
Alliance, spoke on the importance of serving on the institute’s advisory council. “As a council member, you learn quickly to take off your disease
hat. You are not there to push your disease or interest,” said Whittemore, who just ended a council term. “Your voice is heard just as much as the clinician or clinician-scientist sitting at the table with you.”
Participants discussed the proposed National
Center for Advancing Translational Sciences (NCATS)—the new NIH component scheduled to open later this year. Amy Rick, chief executive officer of the Parkinson’s Action Network, provided
a nonprofit group’s perspective. “We support
NCATS because it is a smart way to address some of the significant issues that affect translational
Four parallel breakout sessions dealt with peer review and priority setting, translational and preclinical
science, resources for patient registries and recruitment and clinical research. Session spokespersons reported back to the larger group and gave brief accounts of key lessons— how to set up a patient registry, what three things NINDS looks for in a grant, the importance of establishing
partnerships and how to use a natural history study to build a clinical trial.
During lunch, small networking groups were divided among six topics: Rare Disease Network, Multisystem Diseases, the Benefits of Building Nonprofit Alliances, Nuts and Bolts of Establishing a Nonprofit
Agency/Selecting a Scientific Advisory Board, Public-Private Partnerships
and Working with FDA. Representatives could talk directly with staff who oversee NINDS research portfolios as well as with patient advocacy colleagues, and other NIH and FDA staff.
Lunchtime networking sessions give group representatives a chance to talk with NINDS program staff.
Photos: Ernie Branson
Collaboration Equals Success
The afternoon looked at case histories, highlighting three groups’ accomplishments,
experiences and knowledge and showing how collaboration with NIH, other HHS agencies, industry and Congress could lead to successful outcomes.
Dr. Anne Rutkowski, chair of Cure CMD, discussed her group’s achievements in growing from a small group of only three volunteers to developing natural history studies, outcome measures and a patient registry—all on a “shoestring”
Friedreich’s Ataxia Research Alliance (FARA) President Ronald Bartek and Executive Director Jennifer Farmer spoke on the necessity of partnering with the information technology and pharmaceutical industries. Using limited resources, FARA coordinated and collected data from a natural history and outcome measures study and developed a patient registry with volunteer help from a major IT company and five college students.
Dr. Kenneth Fischbeck, chief of NINDS’s Neurogenetics Branch, pinch hit for the Spinal Muscular Atrophy Foundation in its absence by relaying the group’s success in creating tools such as in vitro assays for screening and optimizing drug candidates, animal models that mimic different disease types and developing
a platform for in vivo screening of compounds.
“The NINDS Nonprofit Forum is a recognition and acceptance of the evolving
role of patient advocacy groups as research partners in the quest for better diagnostics and interventions for rare diseases,” concluded Dr. Stephen Groft, director of the NIH Office of Rare Diseases Research. He helped lead a networking
session. “It was an outstanding conference. The interchange that occurs between staff and patient group leaders really is remarkable,” he said.