CHICAGO Rush University Medical Center has just enrolled the first U.S. patient in an international clinical trial testing a novel drug to treat certain kinds of melanoma, a deadly skin cancer that in its advanced stages currently has few effective treatments.
Rather than blocking or killing all rapidly dividing cells, whether malignant or not, the drug, called nilotnib, is one of a new class of agents that have been designed to sabotage aberrant molecules characteristic of individual cancers in this case, the c-kit protein.
Dr. Howard Kaufman, director of the Rush University Cancer Center and lead investigator of the study at Rush, said that this kind of "targeted" therapy holds out hope of transforming cancer from a lethal disease into a chronic, but manageable disease.
"For advanced melanoma, there are currently few satisfactory treatments," Kaufman said. "But new targeted therapies, including vaccines, antibodies and small molecules like nilotnib are in clinical trials now, adding to an arsenal of treatments that appear to be promising. This trial is especially significant since the c-kit mutation is found more commonly in melanoma arising from the mucosa and foot, which are historically very difficult types of melanoma to treat."
Nilotnib, marketed by Novartis under the brand name Tasigna, is an oral drug approved by the Food and Drug Administration for the treatment of chronic myelogenous leukemia but is now being tested for the first time for the treatment of melanomas that express the c-kit gene. As a small molecule, nilotnib is able to slip across the cell's membrane and into the machinery inside. There it targets, and turns off, the abnormal c-kit protein, created by a mutated c-kit gene, shutting it down and thus disrupting the relay team of molecular signals the protein participates in that ultimately spur cell growth and cause melanoma lesions to proliferate.
Patients with melanomas expressing a mu
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Rush University Medical Center