The National Institutes of Health (NIH) has launched three pre-clinical projects to advance potential new treatments for acute radiation syndrome, brain injury following cardiac arrest and a rare blood disorder called beta thalassemia. The projects are part of the Bridging Interventional Development Gaps (BrIDGs) programme, which is funded by the NIH Common Fund and led by NIH’s National Centre for Advancing Translational Sciences (NCATS).
BrIDGs provides eligible scientists with no-cost access to contractor services, such as toxicology studies, for pre-clinical therapeutic development. To be eligible for the programme, projects must have been effective in a disease model. Researchers often apply to BrIDGs because they have hit a roadblock and need additional expertise or lack other resources. Rather than funding successful applicants directly, BrIDGs supports expert NIH contractors who perform pre-clinical services for the researchers free-of-charge.
“BrIDGs researchers and partner scientists work together to bridge the gap between a basic discovery and clinical testing, thereby ensuring potential treatments have a chance to reach patients who need them,” said Christopher P Austin, MD, NCATS director.
A primary goal of a BrIDGs project is the submission of an Investigational New Drug (IND) application to the US Food and Drug Administration to begin human clinical trials. To date, BrIDGs scientists have generated data to support 12 INDs submitted to the FDA, as well as one clinical trial application to Health Canada. They also have evaluated 12 of the 13 projects in clinical trials. Three BrIDGs-supported therapeutic agents have gone as far as phase II clinical trials, in which researchers provide an experimental therapy to a group of patients to evaluate its safety and effectiveness. Third-party investors have licensed seven compounds during or after their development through the BrIDGs programme.
BrIDGs scientists selected the following new projects from the 2013 application solicitation: Henry Hebel, MBA, vice president of drug development Terapio Corp., Austin, Texas: acute radiation syndrome; manufacturing of RLIP76-LyoPL for acute radiation syndrome; Brian MacDonald, Ph.D., president and CEO, Merganser Biotech LLC, Newtown Square, Pensylavania: beta thalassemia; the development of Minihepcidins for the treatment of beta thalassemia; Laurence Katz, MD, associate professor of emergency medicine, University of North Carolina at Chapel Hill: cardiac arrest-induced acute brain injury; HBN-1 Regulated hypothermia formulation and evaluation of toxicity.
“Although each project is selected for its scientific merit, not all projects will lead to treatments because the pre-clinical phase of drug development is fraught with failures related to issues such as adverse side effects,” said John McKew, Ph.D., acting director of NCATS’ Division of Pre-clinical Innovation and chief of the Therapeutics Development Branch. “Still, the support that BrIDGs provides gives each project a fighting chance.”
The NCATS aims to catalyze the generation of innovative methods and technologies that will enhance the development, testing and implementation of diagnostics and therapeutics across a wide range of human diseases and conditions.
NIAID conducts and supports research — at NIH, throughout the United States, and worldwide — to study the causes of infectious and immune-mediated diseases, and to develop better means of preventing, diagnosing and treating these illnesses.
The NIDDK, a component of the NIH, conducts and supports research on diabetes and other endocrine and metabolic diseases; digestive diseases, nutrition and obesity; and kidney, urologic and hematologic diseases.
The NIH Common Fund supports a series of exceptionally high-impact research programmes that are broadly relevant to health and disease. Common Fund programmes are designed to overcome major research barriers and pursue emerging opportunities for the benefit of the biomedical research community at large. The research products of Common Fund programs are expected to catalyze disease-specific research supported by the NIH Institutes and Centres.
NIH, the nation's medical research agency, and is a component of the US Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases.