NIH program bridges gap to develop new therapeutics

New projects advance treatments for acute radiation syndrome, brain injury from cardiac arrest and a rare blood disorder

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The National Institutes of Health today 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) program, which is funded by the NIH Common Fund and led by NIH’s National Center 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 program, 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, M.D., NCATS director.

A primary goal of a BrIDGs project is the submission of an Investigational New Drug (IND) application to the U.S. 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 program.

BrIDGs scientists selected the following new projects from the 2013 application solicitation:

Acute Radiation Syndrome
Manufacturing of RLIP76-LyoPL for Acute Radiation Syndrome
Henry Hebel, M.B.A., vice president of drug development
Terapio Corp., Austin, Texas

Exposure to radiation, whether from a compromised nuclear reactor or a radiation weapon, can lead to acute radiation syndrome, a life-threatening multi-organ illness. Currently, there is no FDA-approved treatment for the syndrome. This project is designed to develop a treatment that can be administered beginning 24 hours or longer after radiation exposure. NIH’s National Institute of Allergy and Infectious Diseases is co-funding the pre-clinical studies for this project through its Radiation Nuclear Countermeasures Program.

Beta Thalassemia
The Development of Minihepcidins for the Treatment of Beta Thalassemia
Brian MacDonald, Ph.D., president and CEO
Merganser Biotech LLC, Newtown Square, Pa.

Patients with beta thalassemia, a rare inherited blood disorder, suffer from severe anemia and iron overload that can damage the heart. The disorder reduces production of hepcidin, the iron regulatory hormone. The goal of this project is to develop a treatment that increases levels of hepcidin and lowers the damaging effects of too much iron. NIH’s National Institute of Diabetes and Digestive and Kidney Diseases is co-funding this project.

Cardiac Arrest-Induced Acute Brain Injury
HBN-1 Regulated Hypothermia Formulation and Evaluation of Toxicity
Laurence Katz, M.D., associate professor of emergency medicine
University of North Carolina at Chapel Hill

Patients resuscitated from cardiac arrest can suffer from acute brain injury. Lowering a patient’s body temperature into therapeutic-induced hypothermia can improve survival with good neurological outcomes in more than half of patients who remain in a coma after cardiac arrest. This project is designed to develop HBN-1 as an intravenous treatment that paramedics can give to cardiac arrest patients to induce hypothermia sooner. The NIH Common Fund is funding the pre-clinical studies for this project.

“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.”

Information about BrIDGs, including application instructions, is available at www.ncats.nih.gov/bridgs.html.

The National Center for Advancing Translational Sciences (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. For more information about NCATS, visithttp://www.ncats.nih.gov.

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. News releases, fact sheets and other NIAID-related materials are available on the NIAID website athttp://www.niaid.nih.gov.

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. Spanning the full spectrum of medicine and afflicting people of all ages and ethnic groups, these diseases encompass some of the most common, severe and disabling conditions affecting Americans. For more information about the NIDDK and its programs, see http://www.niddk.nih.gov.

The NIH Common Fund supports a series of exceptionally high-impact research programs that are broadly relevant to health and disease. Common Fund programs 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 Centers. Additional information about the NIH Common Fund can be found at http://commonfund.nih.gov.

About the National Institutes of Health (NIH): NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. 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. For more information about NIH and its programs, visitwww.nih.gov.

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