Older Drug Effective in Treating Pediatric Nerve Cancer

Jan. 21 -- TUESDAY, Jan. 20 (HealthDay News) -- An anti-cancer drug shelved because it was thought to be too toxic may be effective in small doses against a devastating pediatric cancer, a new study found.

In tests on animal models, the drug a-difluoromethylornithine, or DFMO, helped prevent the spread of neuroblastoma, a cancer of the nervous system responsible for 15 percent of all pediatric deaths related to cancer.

"The drug, which was developed as a cancer therapy and later shelved because of toxicity concerns, has been around since the 1970s," John Cleveland, chair of the Scripps Florida department of cancer biology, whose laboratory conducted the study, said in a news release issued by the institution. "But over the past five years, it has undergone a rebirth as a chemoprevention agent, first showing efficacy in animal models of human cancer and, more recently, in human prostate and colon cancer. Our study shows that it likely works in a large cast of tumors, even those having poor prognosis, like high-risk neuroblastoma."

In the study, published in the Jan. 15 issue of Cancer Research, researchers successfully used DFMO to target ornithine decarboxylase (Odc), a protein that aids in cancer cell growth and has been found in abundance in neuroblastoma with a poor prognosis, to prevent neuroblastoma in genetically altered mice.

"What's even more compelling, we used low doses of the drug, and DFMO only had to be given for a moderate amount of time to prevent cancer," Cleveland said of the study.

Neuroblastoma usually occurs in infants and young children. Those with stage IV, high-risk neuroblastoma have a less than a 40 percent chance of long-term survival.

More information

The U.S. National Cancer Institute has more about neuroblastoma.

SOURCE: The Scripps Research Institute, news release, Jan. 15, 2009