Innovative vaccine therapy shows promise against glioblastoma brain tumors

An innovative vaccine therapy developed at Roswell Park Cancer Institute (RPCI) is being tested in a multisite clinical trial involving 50 patients with newly diagnosed glioblastoma brain tumors. The phase II study, now accruing patients at both Roswell Park and the Cleveland Clinic, will assess the effectiveness of the SurVaxM vaccine in combination with standard chemotherapy as treatment for this often-fatal cancer.

Though relatively rare, glioblastoma is the most common and aggressive type of primary brain tumor. The SurVaxM vaccine, developed by Roswell Park faculty members Robert Fenstermaker, MD, and Michael Ciesielski, PhD, targets survivin, a cell-survival protein that's present in the vast majority of cancers, including glioblastoma. A peptide mimic, the vaccine is engineered to treat survivin-expressing cancer cells as foreigners, inciting a specific immune response. Preclinical studies suggest that the approach may be worthy of additional study in melanoma, ovarian and prostate tumors and perhaps in other survivin-expressing cancers.

"Our first clinical study established that this vaccine appears to have low toxicity, that it generated the tumor-specific immune response we were looking for and that some patients seemed to benefit from it," says Dr. Fenstermaker, who is Chair of the Department of Neurosurgery, Director of the Neuro-Oncology Program and a Professor of Neurosurgery and Oncology at Roswell Park, and principal investigator of the clinical studies. "While it was a small study, seven of the eight patients who received all doses of the vaccine — all of whom had failed standard therapy — survived longer than a year, some much longer. In a disease where few survive beyond eight months, that's an important signal."

All 50 patients in the single-arm phase II study will receive the vaccine in combination with temozolomide (Temodar), the standard chemotherapy for glioblastoma. The vaccine will be given in four doses over the first eight weeks of treatment, and once every 12 weeks from that point on.

"We'll be looking to see if patients who receive the vaccine in addition to standard therapy do better than patients who receive standard therapy alone," says Dr. Ciesielski, an Assistant Professor in the Department of Neurosurgery at Roswell Park. "In glioblastoma, a patient's immune system is healthiest early in the course of the disease, right after they've had surgery. So we're particularly excited about the opportunity to be giving the vaccine up front, in patients who are most likely to benefit."