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New Drug for Brain Cancer Too Dangerous for Pediatric Patients
Date:3/10/2008

eration. However, that same pathway regulates bone development.

Concerned that the drug might throw the baby out with the bathwater among younger patients, the researchers focused on mice between 10 and 14 days old, all genetically engineered to have cancer.

Several doses of HhAntag were tested over two-week treatment periods. The highest dose -- 100 mg/kg offered twice daily -- was found to be an ideal recipe for killing cancer.

However, X-rays revealed that after just two days, the therapy provoked widespread skeletal, cartilage and joint abnormalities, as well as impaired growth and weight loss. Even after cessation of treatment, the damage was irreversible.

Curran believes that the harsh STI consequences observed in young mice would be similarly manifested in children -- though to what degree remains unclear.

"So, for children we could potentially try to figure out how to deliver these drugs to the brain tumor and not the bones," he said. "But that will require a lot more research, which is unlikely to be performed by the pharmaceutical companies who wouldn't benefit financially, because the potential patient pool amounts to just a few hundred children per year for this kind of cancer."

"Without this research, we think the drug will only be useful for a subset of young patients," Curran noted. "Children with recurrent brain tumors, for example. Because, unfortunately, children who have relapsed don't live very long, and the side effects become irrelevant."

Dr. John S. Yu, co-director of the Comprehensive Brain Tumor Program at Cedars-Sinai Medical Center's Maxine Dunitz Neurosurgical Institute in Los Angeles -- shared Curran's concern.

"This study brings up a cautionary note for the long-term well-being of the young cancer patient," he said. "It certainly has significant repercussions for children with medullablastoma and other tumors that may be treatable with an expectation for a l
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