Glioblastoma Multiforme is a disease in which significant progress has been achieved over the past two decades and much investigation of novel drugs in ongoing. One such drug that has been extensively studied is thalidomide. An early study(Marx et al) assessed 38 patients for response. Two patients (5%) achieved a partial response and 16 (42%) had stable disease. The median survival was 31 weeks and the 1-year survival was 35%. Patients who had a partial response or stable disease had either a stabilization or improvement in quality of life scores or performance status. It appears that thalidomide has some activity as a single agent and that it can be increased by combining it with other drugs. One combination that has demonstrated activity is thalidomide and temozolamide and combining thalidomide and irinotecan is also promising. A variety of novel drugs is in trials with thalidomide.
Marx GM, Pavlakis N, McCowatt S, Boyle FM, Levi JA, Bell DR, Cook R, Biggs M, Little N, Wheeler HR. Phase II study of thalidomide in the treatment of recurrent glioblastoma multiforme. J Neurooncol. 2001 Aug;54(1):31-8.
Olson JJ, Fadul CE, Brat DJ, Mukundan S, Ryken TC. Management of newly diagnosed glioblastoma: guidelines development, value and application. J Neurooncol. 2009 May;93(1):1-23.
David A. Reardon and Patrick Y. Wen, Therapeutic Advances in the Treatment of Glioblastoma: Rationale and Potential Role of Targeted Agents, The Oncologist February 2006 vol. 11 no. 2 152-164
M. RIVA et al, Temozolomide and Thalidomide in the Treatment of Glioblastoma Multiforme Anticancer Research March-April 2007 vol. 27 no. 2 1067-1071
nccn.org, glioblastoma, 2012
For Lay version see here