Three Penn labs receive NIH grant to speed translation of medical research into patient care

Oct 4 2011

Three labs from the University of Pennsylvania have received $12.5 million from the National Institutes of Health (NIH) as part of its $143.8 million national grant program to challenge the scientific status quo with innovative ideas that have the potential to speed the translation of medical research into improved health for the American public.

These awards are granted under three innovative research programs supported by the NIH Common Fund: the NIH Director's Pioneer, New Innovator, and Transformative Research Projects Awards. The Common Fund, enacted into law by Congress through the 2006 NIH Reform Act, supports trans-NIH programs with a particular emphasis on innovation and risk taking.

Penn has recipients in each of the three categories.

The key investigators on the Pioneer Award are Jean Bennett, MD, PhD, Principal Investigator of the study, Luk Vandenberghe, PhD, and Albert M. Maguire, MD, all at the F.M. Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, Perelman School of Medicine. They have been awarded $4 million over the next five years to use gene therapy to treat inherited forms of blindness, which can be caused by mutations in any of hundreds of different genes. The researchers plan to develop a small number of therapeutics that could restore vision to millions of patients who are blind due to a diverse set of retinal disorders. They propose re-sensitizing the blind eye by delivering light-sensitive molecules to the remaining retinal cells. This "optogenetic therapy" approach takes advantage of circuitry between the retina and the brain that remains intact in many individuals long after they have become blind. Preclinical studies in blind animals have demonstrated that this strategy is effective. This study aims ultimately to test the safety and efficacy of this approach in blind patients in the clinic. The results from this project could lead to a significant improvement in the quality of life for millions of individuals, and could also pave the way for development of novel gene therapy approaches for the treatment of other devastating sensory diseases.

Arjun Raj, PhD, assistant professor of Bioengineering, School of Engineering and Applied Science, received the New Innovator Award, for $1.5 million over five years. His research involves the development and application of new microscopic imaging tools to reveal how the physical organization of the genetic code determines the manner in which the cell reads the code itself. The development of these methods will establish a "nuclear GPS," allowing researchers to directly visualize genetic organization in single cells. An understanding of this organization will be important for elucidating how defects in translating the genetic code contribute to such diseases as cancer.

A $7 million, five-year Transformative Research Project Award was given to a team of researchers from the Perelman School of Medicine, Emory University, and Georgia Tech, including Sunil Singhal, MD, director of the Thoracic Surgery Research Laboratory at Penn. If a tumor is more visible and easier to distinguish from surrounding tissues, surgeons will be more likely to be able to remove it completely. To that end, the team has developed fluorescent nanoparticle probes that home in on cancer cells. At present, a significant group of patients who undergo surgery leave the operating room without complete removal of the tumor. The researchers' main goals are to help surgeons distinguish tumor edges; identify diseased lymph nodes and to determine if the tumor has been completely removed. Having these capabilities can be expected to make a major impact in reducing recurrence rates of lung cancer after surgery. The grant includes plans for tests of the nanoparticles in animal models and a first-in-human clinical trial for patients with lung cancer. The proposed technologies could be broadly applicable to many types of solid tumors.

"The NIH Director's Award programs reinvigorate the biomedical work force by providing unique opportunities to conduct research that is neither incremental nor conventional," said James M. Anderson, M.D., Ph.D., director of the Division of Program Coordination, Planning and Strategic Initiatives, who guides the Common Fund's High-Risk Research program. "The awards are intended to catalyze giant leaps forward for any area of biomedical research, allowing investigators to go in entirely new directions."

Since inception, the NIH Director's Award Program has funded a total of 406 High-Risk Research awards: 111 Pioneer Awards since 2004, 216 New Innovator Awards since 2007, and 79 Transformative Research Projects Awards since 2009. This tally includes this year's 13 Pioneer Awards, 49 New Innovator Awards, and 17 Transformative Research Projects Awards.

The NIH expects to make competing awards of approximately $10.4 million to Pioneer awardees, $117.5 million to New Innovators, and $15.9 million to Transformative Research Projects awardees in Fiscal Year 2011. The total funding provided to this competing cohort over a five-year period is estimated to be $245.6 million.

Source: University of Pennsylvania School of Medicine