Trials of oral drug show promise in fighting pediatric tumors
NF1 affects 1 in 3,000 people and approximately 50 percent of those diagnosed develop inoperable plexiform neurofibromas that are diagnosed in early childhood and cause significant pain, disability and disfigurement. Most of the 24 children (11 girls and 13 boys) in the study tolerated the medicine well and many saw some tumor shrinkage. Seventy percent of the patients saw 20 percent or more reduction in tumor volume.
The study was led by Dr. Brigitte Widemann, acting chief of the National Cancer Institute’s Pediatric Oncology Branch and sponsored by the institute's Cancer Therapy Evauation Program.
“Some may say that a 20 percent volume reduction is too small to be meaningful, but to me, just stopping the growth of these devastating tumors is an important achievement,” Widemann said. “The difference we see in these patients is truly unprecedented.”
Two independent teams first identified the disease-causing gene for NF1 in 1990; one team was led by National Institute of Health Director Dr. Francis Collins, who was working as chief of Medical Genetics at the University of Michigan at the time. Ray White of the University of Utah led the other team that identified the gene.
Selumetinib is a selective inhibitor of the MEK protein and has demonstrated activity in some advanced cancers, but is not yet approved by the U.S. Food and Drug Administration.
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