Advances and Future Directions for Tuberous Sclerosis Complex Research: Recommendations From the 2015 Strategic Planning Conference.
On March 10 to March 12, 2015, the National Institute of Neurological Disorders and Stroke and the Tuberous Sclerosis Alliance sponsored a workshop in Bethesda, Maryland, to assess progress and new opportunities for research in tuberous sclerosis complex with the goal of updating the 2003 Research Plan for Tuberous Sclerosis (http://www.ninds.nih.gov/about_ninds/plans/tscler_research_plan.htm). In addition to the National Institute of Neurological Disorders and Stroke and Tuberous Sclerosis Alliance, participants in the strategic planning effort and workshop included representatives from six other Institutes of the National Institutes of Health, the Department of Defense Tuberous Sclerosis Complex Research Program, and a broad cross-section of basic scientists and clinicians with expertise in tuberous sclerosis complex along with representatives from the pharmaceutical industry. Here we summarize the outcomes from the extensive premeeting deliberations and final workshop recommendations, including (1) progress in the field since publication of the initial 2003 research plan for tuberous sclerosis complex, (2) the key gaps, needs, and challenges that hinder progress in tuberous sclerosis complex research, and (3) a new set of research priorities along with specific recommendations for addressing the major challenges in each priority area. The new research plan is organized around both short-term and long-term goals with the expectation that progress toward specific objectives can be achieved within a five to ten year time frame.
Department of Neurology, Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts. Electronic address: mustafa.sahin@childrens.harvard.edu.
Mustafa
M
Sahin
Division of Pulmonary and Critical Care Medicine, Brigham and Women's Hospital, Harvard Medical School, Boston, Massachusetts.
Elizabeth P
EP
Henske
Department of Genetics and Complex Diseases, Harvard T.H. Chan School of Public Health, Boston, Massachusetts.
Brendan D
BD
Manning
Vanderbilt Kennedy Center for Research on Human Development, Department of Pediatrics, Vanderbilt University, Nashville, Tennessee.
Kevin C
KC
Ess
University of Tennessee Health Science Center, Le Bonheur Children's Hospital and St. Jude Children's Research Hospital, Memphis, Tennessee.
John J
JJ
Bissler
Center for Neural Science, New York University, New York, New York.
Eric
E
Klann
Division of Pulmonary and Critical Care Medicine, Brigham and Women's Hospital, Harvard Medical School, Boston, Massachusetts.
David J
DJ
Kwiatkowski
Tuberous Sclerosis Alliance, Silver Spring, Maryland.
Steven L
SL
Roberds
Departments of Neurobiology, Psychiatry and Psychology, Integrative Center for Learning and Memory, Brain Research Institute, University of California at Los Angeles, Los Angeles, California.
Alcino J
AJ
Silva
National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, Maryland.
Coryse St
CS
Hillaire-Clarke
Division of Pulmonary Medicine, Department of Pediatrics, Vanderbilt University School of Medicine, Nashville, Tennessee; Division of Allergy, Pulmonary, and Critical Care, Department of Medicine, Vanderbilt University School of Medicine, Nashville, Tennessee.
Lisa R
LR
Young
Department of Neuroscience, Amgen Inc, Cambridge, Massachusetts.
Mark
M
Zervas
National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, Maryland. Electronic address: mamounas@ninds.nih.gov.
Laura A
LA
Mamounas
D000818:Animals; D035843:Biomedical Research; D004195:Disease Models, Animal; D006040:Goals; D006801:Humans; D000070318:Strategic Planning; D014402:Tuberous Sclerosis / Q000235:genetics / Q000503:physiopathology* / Q000628:therapy*; D014481:United States