Translational Research Through Teamwork

Posted: January 9, 2014 at 7:54 am

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Newswise Imagine you are building a house. You would need a team of specialists, including an architect, a general contractor, carpenters, an electrician, a plumber and many others. Now picture yourself leading an effort to develop a new therapeutic drug or device. For that, youd need a very different kind of specialized team.

The National Eye Institute (NEI), part of the National Institutes of Health, has a research program designed to support this team-based approach. NEIs Translational Research Program (TRP) on Therapy for Visual Disorders provides a lead investigator with up to $1.75 million per year for up to five years in order to assemble a multidisciplinary team, and moveor translatepotential new therapies beyond the research lab and into clinical trials.

In addition to bringing together an expert research team, investigators can use funding from the TRP to recruit experts on navigating the drug and device approval process overseen by the Food and Drug Administration (FDA). They can also use TRP funds to seek help in patenting new therapies. These steps are no less essential than lab work for bringing new therapies to patients.

The program enables investigators to assemble multidisciplinary teams that can tackle scientific, technical, and regulatory issues that are beyond the capabilities of any single research group, said Neeraj Agarwal, Ph.D., who oversees programs in research training and workforce development at NEI. The TRP began in 2000, and has funded one or two projects each year since.

Eyeing new drugs for retinal diseases

Krzysztof Palczewski, Ph.D., professor and chair of the pharmacology department at Case Western Reserve University in Cleveland, received a grant (EY021126) through the TRP in 2010. His goal is to develop new drugs for diseases that damage the retina, the light-sensitive tissue at the back of the eye.

Vision begins with cells inside the retina called photoreceptors. Chemicals called retinoids, which are derived from vitamin A, play a key role inside these cells. One type of retinoid, when combined with a protein called opsin, acts as a light-sensitive switch, converting light into electrical signals that are ultimately sent from the photoreceptors to the brain.

Unfortunately, the supply of retinoids is limited and they need to be recycled. Moreover, the recycling process isnt 100 percent efficient. Dr. Palczewski has found that it can lead to the formation of a toxic byproduct called all-trans-retinal (atRAL), which may contribute to some diseases of the retina, such as age-related macular degeneration (AMD) and Stargardt disease. AMD is a leading cause of vision loss among people age 50 and older. Stargardt disease is a rare genetic disease that begins in childhood but has some similarities to AMD.

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Translational Research Through Teamwork

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