Researchers at the Sidney Kimmel Comprehensive Cancer Center involved in a preliminary study of the Hedgehog signaling pathway report positive responses to an experimental anticancer drug in a majority of people with advanced or metastatic basal cell skin cancers. One patient with the most common type of pediatric brain cancer, medulloblastoma, also showed tumor shrinkage.
Initial results of the drug trial, conducted at Johns Hopkins in Baltimore, the Karmanos Cancer Center in Detroit and the Translational Genomics Research Institute in Scottsdale, Ariz. are published online in the Sept. 3 edition of the
New England Journal of Medicine. The publication also details side effects of the drug, including muscle cramping, hair loss, fatigue and low blood sodium.
Known as GDC-0449, the compound is designed to inhibit the Hedgehog signaling pathway, which researchers believe fuels the growth of some cancers. The pathway was originally named for the oblong hedgehog-like shape of fly embryos when a key gene in the pathway is disrupted.
Related research by Johns Hopkins and Genentech investigators reported online in the Sept. 3 issue of Science Express reveals more findings on the medulloblastoma case.
"We know that both of these cancer types have mutations in Hedgehog pathway genes, and our results with Hedgehog inhibitors could be the starting point for developing a new type of therapy for these intractable cancers," says Charles Rudin, M.D., Ph.D., associate director for Clinical Research at the Johns Hopkins Kimmel Cancer Center.
In the Phase I clinical study, 33 patients with advanced basal cell skin cancer were treated with GDC-0449, an oral drug made by Genentech. Patients were enrolled at Johns Hopkins, Wayne State University's Karmanos Cancer Center, and the Translational Genomics Research Institute.
Writer: Walaika Haskins
Source: Dr. Charles Rudin, John Hopkns Kimmel Cancer Center