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November 17, 2017
NINDS Nonprofit Forum Explores Progress Through Partnership

NINDS recently held its 11th “Progress Through Partnership” nonprofit forum bringing together more than 130 participants—including representatives from 63 patient advocacy groups from around the country—to learn about NINDS research and funding, network with each other and interact with program staff.

“The purpose of the meeting is two-fold,” said NINDS director Dr. Walter Koroshetz. “First, to listen to the nonprofit organizations and get their input and feedback, and second, to inform them about what we are doing here at NINDS and where their tax dollars go.”

New this year was a pre-forum orientation session for first-time attendees that featured a primer on NINDS research priority-setting, decision-making and NIH basics given by Dr. David Owens of the Division of Extramural Activities and Dr. Ernie Lyons of the Scientific Review Branch. Also on the bill was “A Day in the Life of a Program Director,” by Dr. Jill Morris of the Neurogenetics Cluster.

Shriver stresses the importance of working together, especially in times of need. Shriver stresses the importance of working together, especially in times of need.
At NINDS’s recent forum (from l) Lauren Dunlap of the Advocacy & Awareness for Immune Disorders
Association discusses her poster with NINDS director Dr. Walter Koroshetz; Janet Hieshetter of the Dystonia
Medical Research Foundation leads the “Maximizing Impact in a Tough Budget Era” discussion.

The forum featured discussions on patientdriven research, access to new drugs for rare neurological disorders, continuity of care and issues and challenges in functional endpoints and neurological scales, which focused on Food and Drug Administration guidance for neurological clinical trials.

Dr. Margie Frazier of the Batten Disease Support and Research Association shares a success story.
Dr. Margie Frazier of the Batten Disease Support and Research Association shares a success story.


“There is no other area I can think of besides neurology that is so rich with opportunities but so devoid of beneficial therapies,” said panelist Dr. Billy Dunn of FDA’s Center for Drug Evaluation and Research, describing how difficult neurological disorders are to treat. “To be an effective drug, we [at FDA] evaluate how it makes the patient feel, function and survive.”

Throughout the 2-day meeting, nonprofit representatives shared lessons as well as success stories, which ranged from discovering new treatments to achieving therapy readiness.

Dr. Anne Pariser of NCATS and Ron Bartek of the Freidreich’s Ataxia Research Alliance led a presentation on the new, web-based NCATS Toolkit for Patient-Focused Therapy Development. The toolkit, which debuted in September, was created as a one-stop resource to guide patient groups through the therapy development process.

“Patient involvement is critical and necessary at every stage of rare diseases research and therapy development,” Pariser said.

Eric Dishman, director of the All of Us Research Program, provided an in-depth look at NIH’s newest research initiative. All of Us is an effort to gather data from 1 million or more people living in the United States to accelerate research and improve health.

“I became a patient advocate because patient advocates helped me understand what was happening to me,” said Dishman, a 23-year kidney cancer survivor. “At the heart of the All of Us Research Program is a participant-centeredness that undergirds all that we do.”

During the meeting, nonprofit representatives also informally met with NINDS program staff and attended a session featuring 25 posters on key nonprofit initiatives involving the institute.

Forum Session Emphasizes Importance of Continuity of Care

Continuity of Care—one of the many patient-centered topics presented during NINDS’s nonprofit forum—explored transitional care, which is the process beginning in early adolescence to prepare children with chronic illness and their families for adult care.

“From the perspective of the young adult, it is a challenge to shift from a long-standing family-based medical relationship to what can be an alien adult medical system that is individual-based, often narrowly focused and with fewer supports like social workers, therapists or special education resources,” said Dr. Lawrence Brown of Children’s Hospital of Philadelphia (CHOP).

Panelist Dr. Lawrence Brown speaks on transitional care.
Panelist Dr. Lawrence Brown speaks on transitional care.

Brown focused on the neurologist’s role in transition. He discussed barriers to as well as core elements of good health care transition.

“Little attention was ever directed on what we as pediatric specialists and primary care providers could do to ensure that all youth—especially those with special health care needs—would reach their full potential in terms of knowledge of their medical condition, ability to manage their condition and enjoy the best quality of life,” he said. “Only in recent years has transition been understood as a process that takes many years of preparation.”

Ten years ago, Brown started a program to help families whose children with Lennox Gastaut syndrome, a severe form of epilepsy, were aging out of CHOP. The program grew to include all adolescents and youth with neurological disorders and became a national project later adopted by the Child Neurology Foundation (CNF).

After organizing a consensus panel that published transition guidelines for youth with neurological disorders, CNF looked for ways to integrate the process into daily practice and to help adolescents understand the importance of preparing for adulthood. This led them to develop resources for neurologists on the CNF web site, projects to incorporate transition into the electronic medical record and a comic book titled Understanding Transitional Care in Epilepsy.

Programs like CHOP’s are being developed throughout the United States and Canada, but creating a standard plan remains a challenge.

“Clearly, there is no one size fits all,” Brown concluded. “The needs within neurology are as variable as those across other specialties. For example, transition preparation for a youth with Duchenne dystrophy is very different than for one with tuberous sclerosis, and what is needed for sickle cell disease is different than for cystic fibrosis.”

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