NINDS Launches New Translational Funding Programs 4

Posted by Rajesh Ranganathan and Story Landis

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New treatments for the hundreds of disorders that affect the brain are too few and far between. One reason for this lack of treatments is that we do not yet understand how the brain and nervous system work at a fundamental level (see our recent blog post about the need to support basic science). In addition, when potential treatments for nervous system disorders are discovered in the laboratory, researchers in academia often lack the expertise and access to critical infrastructure required to develop a new drug, biologic agent, or device to the point where the biotech/pharma industry will pursue it further. Compounding the problem, and reflected in statistics for pharmaceutical development, is that treatments for brain disorders have particularly long clinical development and approval times (8.8 years) and a low clinical approval success rate (~8%). With such deep risks in terms of time and money, pharma has dialed back early-stage investments in drug development for neurological diseases, despite the large number of people affected who urgently need new and better treatments. Taken together, these factors mean that the clinical application of many promising potential therapies is never fully explored.

bp_neurotherapeutics_logo_smNINDS has supported researchers through the discovery and preclinical phases of therapeutic development with several research funding mechanisms, including those developed by the Institute’s Office of Translational Research (OTR). Recognizing the need to play a bigger role in bridging the gap between the discovery of a potential therapy and its development and clinical testing, NINDS staff thoroughly assessed the strengths and weaknesses of our existing programs. Based on that assessment and in consultation with the community, NINDS has just launched three carefully crafted funding programs, each tailored to specific treatment modalities—the Blueprint Neurotherapeutics Network for small molecules, CREATE (Cooperative Research to Enable and Advance Translational Enterprises) Bio, and CREATE Devices. These new milestone-driven programs offer support for preclinical development and potentially small clinical trials and allow researchers in academia and small companies the opportunity to play a more active part in translating their basic neuroscience discoveries into treatments. We hope novel therapies advanced through these programs will become attractive enough to hand off to biotech/pharma companies, which can then lead later-stage development and testing and ultimately produce treatments approved for use in humans. More…