From Lab to Clinic: NINDS Support of Translational Research

By Walter J. Koroshetz, M.D., Director, NINDS, Amir Tamiz, Ph.D., Director of the Division of Translational Research at NINDS, and Emily Caporello, Ph.D., Program Director, NINDS Small Business Program

NINDS-funded science is the foundation that supports the development of new diagnostics and treatments for neurological disorders. However, advancing a therapeutic molecule or device from the laboratory to adoption by industry and then dissemination into healthcare is challenging and expensive. Along the way, researchers must overcome unique challenges to make sure novel products are safe and benefit patients. At NINDS, the Division of Translational Research (DTR)’s mission is to accelerate basic research findings towards patient use for neurological disorders and stroke by providing researchers in academia and biotechnology with funding, expertise, and resources using a diversity of programs. NINDS’s strategy is to fund investigators to obtain the data that would “de-risk” projects so that they can attract substantial private funding and move toward commercialization. We especially focus on developing treatments for conditions for which there is limited investment by the private sector, so that anyone with a neurologic disorder may benefit from NINDS-funded research.  Here, we highlight some of these programs, including the NINDS Small Business Program and translational research initiatives supported by the collaborative NIH Blueprint for Neuroscience.

NINDS Small Business Program

The NINDS Small Business Program, part of the congressionally mandated National Institutes of Health (NIH) Small Business Program, encourages research and development that can lead to commercialization applications arising from neuroscience research. The program supports small businesses and start-ups whose innovative ideas run the gamut from applied bench research to early-stage human clinical trials. With over $83 million in funding available each year, the NINDS Small Business Program can support approximately 150 companies at any one time, many of which are early-stage spinouts formed from basic research discoveries at institutions, and who are working on turning discoveries into treatments.

Small companies often need funding and resources to further develop initial discoveries into products that are ready for industry partnership and commercialization to ultimately reach patients who need them. Providing an early pillar of support for commercialization, the NINDS Small Business Program has two main components: the Small Business Innovation Research (SBIR) program and the Small Business Technology Transfer (STTR) program. Both use funding mechanisms to support projects and ideas across the spectrum of research and development from early-stage partnerships between universities and small businesses to business cooperative grants that have the option to fund limited-size clinical trials. Additionally, most of our programs offer non-dilutive funding and resources, which does not require business owners to give up equity as they further develop their ideas.

For years, our small business program has helped bring several promising therapies closer to commercial application. Recently, we helped fund a clinical trial testing a variation of CAR-T cell therapy that aims to treat myasthenia gravis. We supported research for a variety of neurological conditions, injuries, and research tools that have led to success stories such as:

In addition, NINDS leads oversight of the SBIR/STTR pain management programs through the Helping to End Addiction Long-term® Initiative (or NIH HEAL Initiative®). Here, the goal is to encourage the development of innovative and commercializable technologies and products that help diagnose and treat pain as well as improve pain management, by mitigating the limited amount of private investment in this space. Fourteen NIH Institutes, Centers and Offices currently participate in the HEAL SBIR/STTR pain management programs, which have provided approximately $60 million in funding to U.S. small businesses to date. Some recently funded projects include development of a novel therapy for migraine, a novel anesthesia guidance system, and a pain management technology for surgical patients.

Translational Research Support through the NIH Blueprint for Neuroscience Research

The NINDS DTR also supports translational research programs for the NIH Blueprint for Neuroscience Research. This NIH-wide initiative with 12 collaborating Institutes, Centers, and Offices supports cross-cutting neuroscience activities aimed at accelerating transformative discoveries in brain function as it relates to health, aging, and disease, including a track for US small businesses. Two recently established programs of the NIH Blueprint are designed to further support important translational research.

First, the NIH Blueprint Neurotherapeutics Network for Biologics (BPN-Biologics), built on the early success of BPN for Small Molecules, was developed with the recognition that there are considerable challenges involved in moving biologics discoveries (e.g., gene therapies, cell therapies, and antibodies) from a lab or small startup into a clinical setting. This process requires navigating complex issues regarding characterization, optimization, manufacturing (including good manufacturing principles), delivery, regulatory, and route of administration. The program has started six projects over the past year covering Muscular Dystrophies, Spinal Cord Injury, Glaucoma, and Epilepsy. BPN-Biologics supports successful biotherapeutic drug discovery and development as well as access to resources, including ones that facilitate connecting investigators with consultants with deep expertise in drug discovery and development to help funded investigators navigate through the preclinical development process. If you are interested in learning more about BPN-Biologics, you can view a recent webinar that provided a program overview and answered attendee questions.

A second new translational research program of the NIH Blueprint is Blueprint MedTech, an NIH funded incubator program that aims to address challenges facing the development of groundbreaking medical device technologies. Blueprint MedTech was built to help catalyze the translation of discovery from early-stage development to clinical trial readiness, this time with a focus on medical devices. Blueprint MedTech, is structured into two main components:

  1. The Blueprint MedTech Incubator comprises two hubs—the Center for Innovative NeuroTech Advancement (CINTA) and NeuroTech Harbor (NTH)—that are funded by NIH to in turn provide funding and in-kind resources to innovator teams. The third cycle of funding through this program closed in August 2023, and the next solicitation is expected to be announced in February 2024.
  2. The Blueprint MedTech Translator program includes two funding opportunities aimed at supporting investigators (through the UG3/UH3 mechanism) and small business concerns (SBCs; U44 mechanism) in their pursuit of translational activities and clinical feasibility studies that contribute to the development of therapeutic and diagnostic devices for disorders of the nervous system.  

Blueprint MedTech supports many earlier-stage neurotechnology development efforts, in collaboration with CINTA and NTH to bring promising neurotechnologies to readiness for first-in-human clinical studies. NINDS recently funded three “Translator” projects through the Blueprint MedTech program:

  • at-home hydrocephalus shunt monitoring, to reduce unnecessary emergency department visits, frequent invasive testing, and reduce inpatient stay duration;
  • a low-field magnetic resonance imaging (MRI) scanner for adaptive neurosurgical intraoperative imaging not possible with currently available technologies;
  • magnetic resonance (MR) image-guided focused ultrasound (MRgFUS) stimulation system – a noninvasive device-based neuromodulation system for non-addictive pain treatment.

Together, these and other efforts at NINDS and across NIH bolster support for new technologies and treatments at a critical phase in the process of bringing them into clinical use. We encourage you to learn more about our translational research programs, including our small business program, as we work to accelerate fundamental basic research findings towards improving the health of those with neurological disorders and stroke.

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