Study Identifies Targeted Molecular Therapy For Untreatable NF1 Tumors

In the news release, Study Identifies Targeted Molecular Therapy for Untreatable NF1 Tumors, issued 10-Dec-2012 by Cincinnati Children's Hospital Medical Center over PR Newswire, we are advised by the company that the fourth paragraph, third sentence, should read "In the United States alone there are more than 100,000 people with NF1." rather than "In the United States alone there are more than 10,000 people with NF1." as originally issued inadvertently. The complete, corrected release follows:

Study Identifies Targeted Molecular Therapy for Untreatable NF1 Tumors

CINCINNATI, Dec. 10, 2012 /PRNewswire-USNewswire/ -- Researchers conducting a preclinical study in mice successfully used targeted molecular therapy to block mostly untreatable nerve tumors that develop in people with the genetic disorder Neurofibromatosis 1 (NF1).

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Scientists from Cincinnati Children's Hospital Medical Center report their findings online Dec. 10 in the Journal of Clinical Investigation.

"We can for the first time shrink the large majority of neurofibromas, at least in mice, by using a molecularly targeted treatment," said Nancy Ratner, PhD, principal investigator and program leader for the Cancer Biology and Neural Tumors Program in the Cancer and Blood Disorders Institute at Cincinnati Children's. "At present there is no treatment for these tumors and our data provide strong rationale for testing this therapy in clinical trials for NF1."

Neurofibromas are benign tumors that grow along peripheral nerves in the body. They affect up to half of the people who have NF1. In the United States alone there are more than 100,000 people with NF1. Fueled initially by mutation of the tumor-suppressing gene NF1, the tumors can grow to become quite large and compress vital organs. They can also transform into deadly malignant peripheral nerve sheath tumors (MPNSTs), the leading cause of death in people with NF1.

The researchers tested an existing experimental drug from Pfizer called PD0325901, which in the current study shrank NF1 tumors in over 80 percent of the mice treated. Study authors report it was "the most dramatic result described to date for neurofibroma bearing mice."

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