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November 20, 2015
Nonprofit Forum Urges Collaboration

“Progress through Partnership,” NINDS’s ninth nonprofit forum, drew its largest crowd as 145 participants—including representatives from 69 patient advocacy groups—gathered to network with colleagues, learn the inner workings of NIH and NINDS and participate in discussions with staff.

“This is an interactive meeting with patients and their representatives that we are serving,” said NINDS director Dr. Walter Koroshetz in opening remarks. “It is our relationships with the patient organizations that make NINDS hum. These groups also give the people who work at NINDS a real sense of the importance of NIH’s research. We are all motivated by the patients you represent.”

The forum officially kicked off with a plenary talk on patient registries by Dr. Joe Selby of the Patient-Centered Outcomes Research Institute (PCORI). The panel that followed featured six nonprofit representatives sharing case studies in working with patient registries. Panelists covered a wide range of expertise—from those involved in small, low-tech registries to those working with larger, research-level registries.

NINDS director Dr. Walter Koroshetz (l) shares a light moment with Ronald Bartek, president of the Friedreich’s Ataxia Research Alliance panelist Dr. Erika Augustine of the University of Rochester offers lessons learned about registries co-moderator Susan Dickinson, executive director of the Association for Frontotemporal Degeneration, speaks on working with government and industry partnerships on translational research.
At the nonprofit forum, NINDS director Dr. Walter Koroshetz (l) shares a light moment with Ronald Bartek, president of the Friedreich’s Ataxia Research Alliance; panelist Dr. Erika Augustine (c) of the University of Rochester offers lessons learned about registries; and co-moderator Susan Dickinson, executive director of the Association for Frontotemporal Degeneration, speaks on working with government and industry partnerships on translational research.

Co-moderator Dean Suhr of the MLD Foundation—a small 2-person organization he runs with his wife that serves families affected by metachromatic leukodystrophy—talked about the registry he designed to capture data about MLD families.

“Families are our mission but they are also our currency,” he said. “We have to know our families because we are here to serve them but that’s also the source of value we bring to every contact that we make with industry or regulatory agencies.” Suhr’s foundation now has teamed up with a PCORI-funded Patient-Powered Research Network, which allows them to collaborate and share data and resources with other groups in the network.

NINDS acting deputy director Dr. Alan Willard provided “NIH 101”—how funding decisions are made and priorities set, how the budget affects the payline, what role the National Advisory Neurological Disorders and Stroke Council plays in funding and other fundamental grant information. He concluded with suggestions on how individual groups can contribute to NINDS’s process by providing comments to requests for information, helping principal investigators recruit for clinical studies and recruiting young investigators to the field.

Day two focused on ways patient advocacy organizations can work with partners in industry, academia and government to nurture research on particular diseases. Dr. Paul Scott, director of the NINDS Office of Science Policy and Planning, reviewed the NIH Strategic Plan. Three panels representing major research areas—basic, translational and clinical—followed his talk.

Co-moderators Ronald Bartek of the Freidreich’s Ataxia Research Alliance and NORD, and Illene Miller of Hope for Hypothalamic Hamartomas, led “Strategies for Stimulating a Basic Research Portfolio.” Panelist Dr. Laura Mamounas, NINDS program director in the neurogenetics cluster of the Division of Extramural Research, gave five defining principles for advancing research:

  • Partnerships are critical
  • Fund only the best science
  • Feed the pipeline
  • Diseases are not silos
  • Invest in the future workforce
co-moderators Paul Gross (l), chief executive officer of the Hydrocephalus Association, and Dean Suhr, president of the MLD Foundation, share patient registry case studies. NINDS acting deputy director Dr. Alan Willard gives “NIH 101” presentation at the 1½-day nonprofit forum.

At left, co-moderators Paul Gross (l), chief executive officer of the Hydrocephalus Association, and Dean Suhr, president of the MLD Foundation, share patient registry case studies. At right, NINDS acting deputy director Dr. Alan Willard gives “NIH 101” presentation at the 1½-day nonprofit forum.

PHOTOS: DANIEL SOÑÉ

“We need to ensure that rigor and reproducibility are second nature to us,” she said. “NIH can’t do it alone. We have to have journal editors, we have to have patient advocates and we have to have the investigators really understand the importance of conducting only the most rigorous reproducible work. Whether a target or a drug is successful or not, that research has to provide a solid foundation so that the next investigator and the next project can build upon it.”

In the second panel, co-moderators Dr. Amir Tamiz, program director in NINDS’s Office of Translational Research, and Susan Dickinson of the Association for Frontotemporal Degeneration, explored the value of working with government and industry partners on translational research. Dickinson emphasized to her fellow advocates the importance of knowing the science of the disorder they represent.

“You have to understand your science,” she said. “There is this huge pressure to jump toward the clinic and create the animal models…and to develop compounds and get them tested, but unless you understand the basic mechanisms you’re not going to do that successfully.”

Dr. Elizabeth McNeil, acting director of the NINDS Office of Clinical Research, and Cynthia Rothblum-Oviatt of the A-T Children’s Project, co-moderated the panel on partnering effectively with industry in clinical trials.

“At NIH we think a lot about how we can involve the patients as well as industry and academia,” said McNeil. She used the NeuroNEXT (NIH Network for Excellence in Neuroscience Clinical Trials) program—NINDS’s initiative to conduct exploratory trials in neurological conditions—as an example. “The patients help us write the protocols, they serve on the protocol steering committee for grants that are funded and they sit on the data safety and monitoring boards,” she concluded. “So they are involved throughout the entire process.”

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