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Investing in Translational Research – Bridging a Critical Gap

One of our core goals at NINDS is to turn science advances into new therapies to benefit people with neurological disorders. In particular, through translational research NINDS strives to bridge the “valley of death”—the critical gap between early discovery research and the later stages of development needed to bring interventions into clinical trials.

The NINDS has a long and distinguished history in this area. In the early ‘70s, the first proof of principle trials of enzyme replacement therapy for Gaucher’s disease, which is now standard of care, were performed by Dr. Roscoe Brady and his colleagues in the NIH Clinical Center. For more than 40 years, the Anticonvulsant Screening Program has helped advance dozens of drug candidates to clinical trials, resulting in the approval of 9 drugs by the Food and Drug Administration (FDA) for epilepsy and other conditions.

During the past ten years we have built on these early successes and created additional programs to foster translational research. The Cooperative Program in Translational Research provides support to academic and small business investigators for preclinical therapy development projects for any neurological disorder. The goal of the program is a successful application to the FDA for an Investigational New Drug (IND) or an Investigational Device Exemption (IDE). In developing the program, NINDS set out to bring together multiple types of expertise and to facilitate cooperation across organizations. These projects can include investments by foundations, academia, industry, and NINDS at various stages. The program’s progress has been encouraging: 6 candidate therapies have received FDA authorization to move into clinical trials. Not all projects succeed, however, so NINDS incorporated a milestone-based funding approach. If a project is not achieving its milestones, NINDS will stop funding and invest the resources in more promising opportunities.

In addition to the broad-based Cooperative Program, NINDS has piloted a new approach to drug development using spinal muscular atrophy (SMA) as the test case. Until fairly recently SMA was one of many poorly understood inherited disorders that affect the nervous system, and the outlook for developing treatments was bleak. The scenario changed, however, in 1995 when scientists identified the gene defect that causes SMA. This discovery opened up the possibility of therapy development. Through the SMA Project, experts from academia and industry developed a plan to implement a “virtual pharma” structure made up of organizations that serve industry drug development. The SMA Project has applied for patents on two promising novel drug candidates. The project is continuing with advanced preclinical safety testing of one candidate with the goal of completing certification for a clinical trial by the end of 2011. It also served as the model for a Neurotherapeutics Initiative sponsored by the NIH Neuroscience Blueprint (see

Anticipating that the NINDS translational efforts will provide candidate therapies for neurological diseases that can be ready for clinical testing within the next few years, NINDS has developed the Network of Excellence in Neuroscience Clinical Trials. Dubbed NeuroNEXT, the objective is to improve the speed and effectiveness of early phase clinical testing of novel therapies, which present many logistical challenges. Through NeuroNEXT, multiple clinical centers, a clinical coordinating center, and a data coordinating center will support testing of the most promising therapies, whether they arise from academia, foundations, or industry. The network is expected to be especially important for rare disorders, including pediatric diseases, which often lack infrastructure for conducting clinical trials.

The translational efforts underway at NINDS will benefit greatly from the creation of the new National Center for Advancing Translational Sciences (NCATS) at NIH. We look forward to collaborating closely with NCATS to speed the development of new treatments for neurological diseases that result from the application of basic science discoveries.

Last Modified June 24, 2011