Promising brain cancer vaccine developed at Roswell Park receives orphan drug status from FDA

Aug 8 2017

The U.S. Food and Drug Administration has awarded orphan drug status to a promising immunotherapy vaccine developed at Roswell Park Cancer Institute. The FDA notified MimiVax LLC, a Roswell Park spinoff company, on Aug. 3 that its application for orphan status for SurVaxM as treatment for glioblastoma, a type of brain cancer, had been approved.

Orphan status is a special designation awarded to encourage innovation and exploration of approaches to treat rare diseases that affect relatively few people. SurVaxM, also known as DRU-2017-5947, is an immunotherapy drug that targets survivin, a cell-survival protein present in most cancers.

Invented by two Roswell Park faculty members, the vaccine stimulates the immune system to kill tumor cells that contain survivin, a protein that helps cancer cells to resist conventional treatments. A phase II study of SurVaxM given in addition to standard treatment for patients with newly diagnosed glioblastoma is ongoing at Roswell Park and four other institutions: the Cleveland Clinic, Dana-Farber Cancer Institute, Massachusetts General Hospital and Beth Israel Deaconess Medical Center.

"We are excited by the results to date and appreciative of this acknowledgment that SurVaxM holds promise," says SurVaxM co-inventor Robert Fenstermaker, MD, Chair of Neurosurgery at Roswell Park and Chief Medical Officer at MimiVax. "Those of us working to help patients with glioblastoma to live longer realize that the gains from existing therapies have been quite limited. We are eager to move this work forward to a larger multicenter randomized study with the momentum provided by the orphan status designation."

The vaccine is designed to control tumor growth and recurrence.

"There are a couple of things that distinguish our approach," adds co-inventor Michael Ciesielski, PhD, Assistant Professor of Neurosurgery at Roswell Park and Chief Scientific Officer for MimiVax. "SurVaxM is an engineered molecule capable of stimulating the immune system in several different ways to recognize and kill cancer cells. And the fact that its target, survivin, is present in many different types of cancer suggests potentially broad application against cancer."

On the strength of an interim analysis of the in-progress phase II study, the investigators expect to pursue late-stage clinical trials with SurVaxM, pending FDA approval. The vaccine is also being studied in other types of cancer, with a separate clinical study underway looking at SurVaxM as part of combination therapy for multiple myeloma.

The vaccine team credits donations to the Roswell Park Alliance Foundation with moving their vaccine research forward. The Alliance Foundation raises funds in support of research and patient-care programs at Roswell Park Cancer Institute, including the current study of SurVaxM in patients with newly diagnosed glioblastoma.