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Cincinnati Children’s Receives $6.4 Million NIH Grant to Study Genetic Nerve Disease

Monday, November 24, 2008

Cincinnati Children’s Hospital Medical Center has received a $6.4 million grant from the National Institutes of Health to fund the Center for Neurofibromatosis Research and find possible new treatments for a rare genetic disease that causes nerve tumors, which can become malignant.

The National Institute of Neurological Disorders and Stroke awarded the grant to Nancy Ratner, PhD, a researcher in the Division of Experimental Hematology / Cancer Biology at Cincinnati Children's and director of the Neurofibromatosis center.

"Our goal is to translate research findings into patient therapies," Dr. Ratner said. "Cincinnati Children’s has an outstanding pediatric Neurofibromatosis type1 clinic with more 800 patients in its database. The medical center has participated in ongoing clinical trials for the disease, it’s a leading center for pediatric anticancer drug development, and we bring together a world class group of investigators who can carry out translational research to identify candidate drugs for treatment."

Several multi-disciplinary research projects are being funded by the grant. Those projects - from basic scientific research to the testing of potential new therapies - include colleagues at Cincinnati Children’s (John Perentesis, MD, FAAP and Timothy Cripe, MD, PhD, the University of Cincinnati (George Thomas, PhD and Sara Koozma, PhD) and the University of Minnesota (David Largaespada, PhD).

One out of every 3,000 people has Neurofibromatosis type 1. The disease can be disfiguring, can cause learning disorders, and more rarely leads to tumors or leukemia that can be fatal. In spite of its frequency and seriousness, the disease is just now beginning to be studied in earnest, in part because of research conducted by Dr. Ratner and other colleagues at Cincinnati Children’s.

A study from Dr. Ratner’s lab, published in the February issue of the science journal Cancer Cell, discovered that the timing of the NF1 gene mutation determines whether neurofibroma tumors will form. Dr. Ratner and her colleagues reported that if the NF1 gene mutated half way through the 12th day of a mouse’s embryonic development, it led to neurofibroma tumors. If the gene mutated at other times during development, tumors did not form. The discovery also improved a previous Neurofibromatosis 1 mouse model that Dr. Ratner’s team genetically altered to mimic the disease that occurs in humans.

Cincinnati Children's Hospital Medical Center is one of America’s top three children’s hospitals for general pediatrics and is highly ranked for its expertise in digestive diseases, respiratory diseases, cancer, neonatal care, heart care and neurosurgery, according to the annual ranking of best children's hospitals by U.S. News & World Report. One of the three largest children’s hospitals in the U.S., Cincinnati Children’s is affiliated with the University of Cincinnati College of Medicine and is one of the top two recipients of pediatric research grants from the National Institutes of Health. For its achievements in transforming healthcare, Cincinnati Children's is one of six U.S. hospitals since 2002 to be awarded the American Hospital Association-McKesson Quest for Quality Prize ® for leadership and innovation in quality, safety and commitment to patient care. The hospital is a national and international referral center for complex cases, so that children with the most difficult-to-treat diseases and conditions receive the most advanced care leading to better outcomes.