The FDA has awarded orphan drug status to DRU-2017-5947 (SurVaxM), an immunotherapy drug, for the treatment for glioblastoma.
Orphan status is a special designation awarded to encourage innovation and exploration of approaches to treat rare diseases that affect relatively few people.
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 DRU-2017-5947 given in addition to standard treatment for patients with newly diagnosed glioblastoma is ongoing at Roswell Park Cancer Institute, Buffalo, N.Y., 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 acknowledgement that SurVaxM holds promise," said DRU-2017-5947 co-inventor Robert Fenstermaker, MD, Chair of Neurosurgery at Roswell Park. "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," added co-inventor Michael Ciesielski, PhD, Assistant Professor of Neurosurgery at Roswell Park. "S[DRU-2017-5947] 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 DRU-2017-5947, pending FDA approval. The vaccine is also being studied in other types of cancer, with a separate clinical study underway looking at the drug as part of combination therapy for multiple myeloma.