Wednesday, August 24, 2011

Empowering Drug Development

NIH awarded up to $50 million over five years to work on developing new drugs for disorders of the nervous system. The research aimed at treating conditions such as vision loss, neurodegenerative disease, and depression are being funded through the “NIH Blueprint for Neuroscience Research” program.

Nervous system disorders affect tens of millions of Americans, and there is a substantial unmet need for treatment but the process for developing new drug therapies is costly and carries high risks. Only about 10 to 20 percent of candidate drugs for all disease indications survive the early phases of development and reach clinical trials.

The NIH Blueprint works with 15 of the agency’s institutes and centers to leverage their resources to study major, cross-cutting challenges in neuroscience research. The “Blueprint Neurotherapeutics Network” will help make it possible for researchers to develop new drugs for nervous system disorders and conduct clinical trials using the new drugs.

“The Network will pair neuroscientists with experts in therapy development, and enable them to pursue their new ideas for new drugs without having to redirect the focus of their laboratories” said Story Landis, Ph.D., Director of NINDS within NIH.

The project teams supported by the Network will not only receive funding but will also have access to millions of dollars worth of services normally only available to pharmaceutical companies. Now it will be possible to assist investigators throughout the entire drug development process.

For example, Emory University with $969,000 in funding will be developing new drugs to use for stroke as part of the research program. The research team led by Raymond Dingledine, PhD., aims to develop drugs based on compounds called prostaglandins which have been shown to protect animals from brain damage following a stroke.

The awards went to seven research teams at six academic institutions that includes Brigham and Women’s Hospital, Ohio State University, Columbia University, Emory University, University of California San Diego, the University of Washington, and the Fred Hutchinson Cancer Research Center, plus Trevena Inc a drug discovery company. Go to for more details on each academic institution.

In another scientific project with funding from NIH’s National Institute of General Medical Sciences, Howard Hughes Medical Institute, and PhRMA Foundation, researchers at the Stanford University School of Medicine have paired up medicines and maladies with help from databases on the internet. When the scientists applied an opposites attract algorithm to databases, they found potential compatibilities between numerous existing drugs and diseases that may possibly work together to help in various diseases.

As Atul Butt MD PhD, Associate Professor of Systems Medicine in Pediatrics explained, “the degree to which a drug is effective to treat epilepsy may prove effective to use for another disorder as different as Crohn’s disease. For instance, an ulcer drug to treat adenocarcinoma of the lung might be used with topiramate an anti-seizure drug to treat lung cancer.

Today, new technologies have made it routine to simultaneously measure activity levels for every gene in a cell or tissue. Today there are 750,000 results of such analyses in publicly available databases. For a variety of reasons, in the past, very few scientists have used the data already entered to connect drug possibilities.

In another development, Butt serving on the scientific advisory board of NuMedii, a startup company that aims to commercialize some of the technologies used in the research is hoping to use the results of the research studies to help find funding sources to enable a new approach to getting both on and off patent compounds into the right clinical trials.