Over the last four decades, the U.S. FDA has granted full approval for three drugs for the treatment of brain cancer.
CCNU (lomustine) was approved as a single agent for intracranial tumors in 1976 and marketed under the brand name CeeNu.
In 1996 BCNU (carmustine wafer) was approved and marketed under the brand name Gliadel. Gliadel is indicated for newly diagnosed high grade malignant glioma as an adjunct to surgery and radiation and is indicated for recurrent GBM as an adjunct to surgery. In a randomized, double blind, placebo-controlled study in patients with newly diagnosed high grade malignant glioma, Gliadel demonstrated a median survival advantage of 2.3 months (13.9 months for Gliadel versus 11.6 months for placebo). In a randomized, double blind, placebo controlled study in patients with recurrent malignant glioma, Gliadel demonstrated a median survival advantage of 2.0 months (28 weeks for Gliadel versus 20 weeks for placebo).
Temodar (Temozolomide, TMZ) was approved in 1999 for patients with Grade 3 anaplastic astrocytoma in a single arm response rate clinical trial. In a randomized trial comparing radiation with TMZ versus radiation alone, radiation with TMZ produced a median survival advantage of 2.5 months (14.6 months for TMZ+RT versus 12.1 months for radiation alone). Subsequently, TMZ was approved for the treatment of patients with newly diagnosed GBM concomitantly with radiation followed by maintenance treatment.
Avastin (Bevacizumab) was granted accelerated approval in 2009 for the treatment of patients with progressive GBM following prior therapy. However, subsequent studies demonstrated no survival advantage.
In addition to drug approvals for malignant brain tumors, in 2010 Afinitor (everolimus) received accelerated approval for the treatment of a benign brain tumor called subependymal giant cell astrocytoma (SEGA). An open-label, single-arm response rate clinical trial was conducted in patients with SEGA associated with tuberous sclerosis. At six months, nine out of 28 patients had a ≥ 50 percent reduction in the tumor volume of their largest SEGA lesion site.
Brain Tumor Quick Facts
- Three drugs have been approved for treating brain cancer in four decades
- Alkylating agents are the main class of drugs for treating brain cancer
- Response rate is a common approval end point for drugs targeting brain tumors