The research team behind an emerging immunotherapy for cancer will present interim findings from an ongoing clinical study in patients with brain cancer at a major research meeting next month. Collaborators from organizations, including Roswell Park Comprehensive Cancer Center, the Cleveland Clinic, and MimiVax LLC, will share data from their phase 2 study of SurVaxM as part of a combination treatment for patients with glioblastoma at the 2018 American Society of Clinical Oncology (ASCO) Annual Meeting in Chicago. These results show that the therapy appears to be safe, effective, and worthy of further evaluation.

Glioblastoma is the most common and aggressive form of primary brain cancer in adults. With standard therapy—surgery followed by chemoradiation and then adjuvant chemotherapy—the median overall survival is 15 months, with only 3%-5% of patients living 5 years or more.

The researchers are exploring whether adding SurVaxM to standard therapy improves outcomes for patients with newly diagnosed glioblastoma. Analyzing results to date for 63 patients treated with this combination, the team found that 91% of patients receiving SurVaxM as part of this combination therapy were still living 12 months after initiating treatment, compared to 61% in a historical analysis of patients treated with standard therapy alone. Also, 96% achieved 6-month progression-free survival, compared to 54% among the historical comparison group. Moreover, 13 of 63 patients continue to be without progression a year or longer into their participation in the study.

“These interim phase 2 trial results in newly diagnosed glioblastoma patients are very promising and offer the potential for longer-term survival in this group where there is great, unmet medical need,” says the study’s senior author, SurVaxM co-inventor Robert Fenstermaker, MD, who is Chair of Neurosurgery at Roswell Park and Chief Medical Officer at MimiVax. “We believe this drug has the potential to change the glioblastoma treatment paradigm.”

SurVaxM, which was invented at Roswell Park, targets a cell-survival protein called survivin that is present in 95% of patients with glioblastomas, and also in patients with many other cancers. Awarded orphan drug designation by the US Food and Drug Administration (FDA) in 2017, the vaccine has dual mechanisms of action to stimulate a patient’s T-cell immunity and also employs antibody-directed inhibition of the survivin pathway to control tumor growth and prevent or delay tumor recurrence.

“Glioblastoma is one of the most common and aggressive forms of brain cancer, with few effective treatment options. That dire need underscores the importance of these promising interim results, as we seek to develop new and better therapies,” notes co-inventor Michael Ciesielski, PhD, assistant professor of neurosurgery at Roswell Park and CEO for MimiVax. “And because survivin is present in a huge percentage of cancers in general, we expect that SurVaxM could have broad applicability in many cancers beyond glioblastoma.”