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Cancer Research Takes Turn, Offers Potential Treatment for Macular Degeneration

The National Institutes of Health has awarded a $225,000 Small Business Technology Transfer (STTR) grant to ApeX Therapeutics and its co-founder Mark R. Kelley, Ph.D., Betty and Earl Herr Professor in Pediatric Oncology Research and professor of biochemistry and molecular biology and of pharmacology and toxicology at the IU School of Medicine.

The age-related macular degeneration project represents a new direction for Dr. Kelley’s research, which has focused on the mechanisms cells use to repair damaged DNA and how those mechanisms can be manipulated when developing cancer treatments. In particular, Dr. Kelley’s work has examined a protein called APE1 and its activities in tumor development.

A new drug to treat age-related macular degeneration would be welcome – it is the leading cause of severe vision loss in people over age 60 and more than 7 million older Americans have or are at high risk to develop advanced macular degeneration.

The type of age-related macular degeneration that causes most of the vision loss is caused by abnormal blood vessel growth in the eye, which has led to treatments using drugs designed to block blood vessel growth in cancer – known as anti-angiogenesis drugs. No more than a third of patients get significant benefits from those drugs, however.

The pursuit of a treatment for macular degeneration came about because “we were studying the effects of an APE1 inhibitor in cancer and we saw that it had anti-angiogenesis effects,” said Dr. Kelley, who is associate director of the Herman B Wells Center for Pediatric Research and associate director of basic science research at the Indiana University Melvin and Bren Simon Cancer Center on the campus of Indiana University, Purdue University – Indianapolis.

Dr. Kelley has been working with Xiaoxi Qiao, M.D., Ph.D., associate professor of ophthalmology, and their experiments have reduced blood vessel growth in the laboratory. They also are able to apply the compound via eye drops rather than injections, Dr. Kelley said.

“The grant and research will provide a new dimension to the work of ApeX Therapeutics, which was founded to develop oncology related products,” said chief executive officer Martin F. Haslinger, Ph.D.

“We’re not changing the direction of the company, we’re adding on,” he said.

Haslinger said the company, which has offices and laboratory space in the IU Emerging Technology Center in Indianapolis, hopes to have a compound worthy of taking to clinical testing in about six months.

Kelley and Qiao’s research received support from the Indiana Clinical and Translational Sciences Institute, the IU Medical Group and ITRAC, a cancer center program to help researchers move their laboratory discoveries to the next steps toward development of new treatments for patient care.

“This STTR grant is a culmination of how the translational research process can work – laboratory research developments progress with CTSI assistance and are developed commercially by a local company built on school of medicine research,” said Dr. Kelley.