Data from a large study conducted by the European Organization for Research and Treatment of Cancer (EORTC) in collaboration with the National Cancer Institute of Canada (NCIC) Clinical Trials Group demonstrate that addition of a novel chemotherapy agent, Temozolomide, to radiation therapy increases survival in patients with glioblastoma, according to an article in the March 10th issue of the New England Journal of Medicine.

Further, molecular analyses of tumor tissue allowed researchers to identify the patients most likely to benefit from combined treatment. The findings lead to a new standard of care for patients with this famously aggressive and devastating cancer.

Primary brain tumors account for fewer than 5 percent of all cancer cases. However, brain cancer frequently affects previously healthy younger men and women in the middle of their most active life. Glioblastoma is the most common type of primary malignant brain tumor in adults with a yearly incidence of 5-7 persons per 100,000.

Prior to the discovery of the new therapy, the average life expectancy of patients with glioblastoma was about one year. The results of this study demonstrate a clear improvement of survival. At two years, only 10 percent of patients treated with radiotherapy alone were alive compared with 26 percent of patients receiving the combination of radiotherapy and temozolomide chemotherapy.

If patients were selected according to molecular profile -- the investigators analyzed the functionality of a gene responsible for DNA repair, the so called MGMT gene -- the improvement is even more dramatic, with almost half of the patients whose tumors carried an inactivated MGMT gene copy alive at two years. Importantly, the study also showed that the new combined therapy did not have a negative impact on quality of life.

Lead author Roger Stupp, MD, said "This is the first trial to demonstrate that we can truly impact this devastating disease with chemotherapy. This is only a first step toward cure of brain cancer patients and should now fuel interest, continued international collaboration and research to further improve the outcome of these patients."

"Without the close collaboration between the hospital based research laboratory and the leading clinicians, this interdisciplinary success would never have been possible. I hope this example will stimulate a closer collaboration between basic and clinical research in the future," said Monika Hegi, PhD, signing author for the translation research work.

"Until now, there have been few treatment options for glioblastoma patients," said Dr. Gregory Cairncross, one of the study's primary investigators. "The results of this trial will dramatically improve treatment and outcome for many of these patients and will open the door to further trials to expand this treatment combination. The key to the new therapy's effectiveness is that temozolomide causes very few side effects and is well tolerated by patients. This means patients can take the drug every day during their radiation treatment instead of once every eight weeks -- the common dosage for other chemotherapy drugs."

"This landmark study represents the most important advance in the management of glioblastoma since radiotherapy was shown to be of benefit over 35 years ago," commented Dr. Warren Mason, a lead investigator in Canada. "This study also identified MGMT the first clinically relevant molecular marker for glioblastoma which not only serves as a prognostic factor for survival, but also as a predictor for response to chemotherapy. This observation paves the way for using the unique tumor genetic signature as a guide for individualizing therapy and optimizing outcome."