The drug Gleevac has nearly doubled the survival rate for patients with some forms of leukemia, and scientists believe a similar medication may do the same for brain cancer.
Gleevac (imatinib mesylate), one of the first targeted cancer therapies, was specifically designed to target an abnormal molecule -- a fusion of two normal cell proteins -- that fueled a tumor's growth. Now there’s hope that a similar drug, which works in the same way, could effectively treat glioma, a common form of brain cancer.
The Columbia University Medical School team had previously discovered that a fusion of two proteins also drives some cases of glioma, so in their most recent work they focused on two patients affected by recurrent glioblastoma with the fused proteins.
The new study was the first human trial of a drug that targets half of the fusion protein. The researchers found that the two subjects responded particularly well to the drug, with improvements that lasted 115 and 134 days, respectively.
The drug could have a meaningful impact, the researchers said.
"This suggests that if we developed a drug that hits the fused protein more precisely, while leaving normal cells alone, we may get even better results," said Antonio Iavarone, M.D., the team’s leader. Further drug development and more clinical trials are needed, he added.
The study was published in the journal Clinical Cancer Research.
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