Intramural research can play an important role in fulfilling the NIH mission. Unencumbered by grant writing and teaching responsibilities, investigators in the NINDS intramural research program (IRP) are free to engage in high-risk, high-reward science. IRP investigators are further supported in pursuing novel research directions by remarkable infrastructure including cutting-edge technology and access to the world's largest research hospital, the NIH Clinical Center. To ensure that each IRP lives up to its extraordinary potential, NIH mandates independent Blue Ribbon Panel review of each program that summarize its strengths, the challenges it faces, and opportunities for improvement.
NINDS recently convened the first Blue Ribbon Panel review of its IRP. The Panel was chaired by Dr. Robert Darnell, the Heilbrunn Professor and Senior Physician at Rockefeller University, and consisted of nine other extramural experts with a broad perspective on basic neuroscience and clinical neurology and considerable knowledge about NINDS. The goal of the review, which examined both scientific achievement and overall program organization, was to provide the IRP with feedback for enhancing the program's contribution within the larger neuroscience community.
Last Thursday, Dr. Darnell presented the Panel's findings and recommendations and submitted the Panel's full report to the National Advisory Neurological Disorders and Stroke Council. The Panel summarized the IRP's strengths as outstanding leadership, integrative science within NINDS and among IRPs, outstanding support for basic science in the context of clinical neuroscience, and vigilance on opportunities to strengthen and expand the clinical mission. Among the Panel's recommendations, which will help inform the IRP's future endeavors, were to recruit up to five top-tier physician-scientists, capture phenotypic, genetic, and biomarker data on all research subjects, develop robust bioinformatics infrastructure, and increase collaboration across the board. An executive summary of the Panel's report with the full list of recommendations can be read at the bottom of this post.
The NINDS IRP has a strong legacy of supporting outstanding scientists and major breakthroughs in neuroscience. Among the first investigators in the IRP were Julius Axelrod (Nobel Prize in Medicine in 1970 for the study of neurotransmitters), Carleton Gajdusek (Nobel Prize in Medicine in 1976 for showing that kuru was a transmissible prion disease), Roscoe Brady (Lasker award in 1982 for uncovering the pathogenesis underlying Gaucher and Fabry diseases), and Kenneth Cole (National Medal of Science in 1967 for the invention of the voltage clamp technique).
Among our current crop of researchers, senior investigators Kenneth Fischbeck and Thomas Reese are members of the National Academy of Sciences and the Institute of Medicine. Junior investigators Antonina Roll-Mecak and Lucy Forrest are recent recipients of the Biophysical Society's Margaret Oakley Dayhoff Award, which is given to exceptional woman researchers. Dr. Roll-Mecak was also the recipient of a 2010 Searle Award. Kevin Briggman, another junior investigator, was named a Pew Scholar in 2014.
The Panel recognized the IRP's many strengths in both clinical and basic research. They noted that the clinical neuroscience program has been an excellent training ground for clinical trials, and has continuously produced top-notch physician-scientists. The program thrives on its ability to go from bedside to bench and back to the bedside. This approach ushered in the development of enzyme replacement therapy for lysosomal storage diseases and facilitated the initial discovery that spongiform encephalopathies (prion diseases) were caused by a transmissible element. More recently, the program has struck up a unique collaboration with two local hospitals to perform innovative stroke research and in the process providing outstanding acute care for stroke patients in the DC area. Also, owing to its connection with the NIH Clinical Center, which can fly in patients from all over the world, the NINDS IRP has been a leader in studies of rare diseases, such as spinal muscular atrophy and hereditary spastic paraplegias.
The Panel remarked that the NINDS IRP has made important contributions to the fundamental understanding of membrane proteins, electrophysiology, and synaptic function, areas that form the foundation of our basic neuroscience program. The program funds strong research in the structure and function of ion channels, transporters, and cytoskeleton, as well as cell and developmental processes that underlie neurological disorders. They were particularly impressed by the many important advances in brain imaging made in the IRP, including the development of next-generation MRI technologies.
As far as the structure and organization of the IRP, many of the Panel's comments endorsed changes I oversaw during my tenure, dating back 19 years to when I first came to NINDS as Scientific Director. One of my early priorities was eliminating the hierarchical structure of the IRP whereby the program's Branch chiefs dictated how much funding each of their investigators received, and to some extent the direction of their research. Now, the program has a flat structure, with funding tied to rigorous external review of individual investigators' recent past performance. Labs that were not optimally using their resources were allowed to wind down and those funds used to both recruit outstanding young investigators and bolster tenured researchers doing the most innovative science. Over my tenure at NINDS, the program has shrunk from 64 primary investigators (PIs) to 46. This contraction has allowed the IRP to remain vibrant in the face of recent budget cuts.
Also reflecting the IRP's flat structure is a bottom-up approach towards setting both recruitment and research project priorities. PIs from across the program, as well as PIs from other Institutes, weigh in on potential hires, while a Special Initiative Program offers supplemental funds for new and collaborative research projects that are proposed by investigators.
Another priority I have worked towards from the beginning was better integration of the activities of the various Institutes at NIH engaged in neuroscience research. In an effort to build collaboration across these Institutes, the NINDS IRP has initiated the creation of several trans-NIH core facilities such as the NIH Nuclear Magnetic Resonance (NMR) Research Facility in the NIH In Vivo NMR Center and the Human Stem Cell Facility. We have also instituted a shared seminar series, created a website to tie together neuroscience across NIH, and joined with other Institutes to recruit world-class researchers.
Recently, the IRPs' collaborative vision has been crystalized with the completion of the Porter Neuroscience Research Center. This state-of-the-art research building on the NIH main campus brings together more than 800 scientists in 85 laboratories from nine Institutes. Previously, these labs were spread out among at least eight different buildings. The proximity of these labs in their new spaces in Porter will hopefully create numerous opportunities for learning and joint projects.
I appreciate the Blue Ribbon Panel's careful evaluation of the IRP, which was, overall, highly supportive of our efforts and plans for the future. Their recommendations for specific actions will surely help us continue to leverage our research strengths and strive for balance in conducting important basic, clinical and translational neuroscience.
Executive summary of 2014 NINDS Blue Ribbon Panel Report
(As submitted to the NANDSC on September 11, 2014)
Highlights of pre-meeting materials provided to Blue Ribbon Panel
Charge to the Blue Ribbon Panel