There is evidence that the MTOR pathway plays an important role in renal cell cancer. It is also effective in neuroendocrine cancer. AFINITOR®(everolimus) is indicated for the treatment of renal cell carcinoma and progressive neuroendocrine tumors of pancreatic origin (PNET) in patients with unresectable, locally advanced or metastatic disease. The safety and effectiveness of AFINITOR® in the treatment of patients with glioblastoma has not been established.
Clinicaltrials.gov cites more than 30 studies of Afinitor in glioblastoma. Preliminary results have not been encouraging. Twenty-two patients with recurrent glioblastoma (GBM) were prospectively treated with everolimus and gefitinib, designed to test the combined inhibition of two drugs mammalian targets by both drugs as part of a larger clinical trial. The primary endpoint was radiographic response rate. Secondary endpoints included progression-free survival and correlation of molecular profiles with treatment response. 36% of patients had stable disease and 14% a partial response; however, responses were not durable and only one patient was progression-free at six months. Radiographic changes were not well characterized by conventional response criteria, and implied differential effects of therapy within the tumor and/or anti-angiogenic effects. EGFR and PTEN status did not clearly predict response to treatment. Clearly more study is in order.
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