An experimental gene therapy nearly doubled the overall survival of patients with a recurrence of one of the deadliest and most aggressive brain cancers, glioblastoma.
Glioblastoma kills two-thirds of patients within five years; a patient’s outlook with recurrence of the disease is considered to be weeks or months.
A Phase 2 clinical research study evaluated the gene therapy, called VB-111, in continuous and intermittent doses and in comparison to the standard treatment, the chemotherapy Avastin. Patients receiving VB-111 survived 15 months on average, compared to an average of eight months for patients receiving Avastin alone.
“In addition to the benefit in overall survival, VB-111 was safe and well-tolerated in the patients, and proved to be effective both as a single therapy for recurrent glioblastoma and in combination with Avastin,” said Andrew J. Brenner, M.D., Ph.D., a medical oncologist with the Cancer Therapy & Research Center and associate professor in medicine, neurology and neurosurgery.
The CTRC and three other centers enrolled 62 patients with recurrent glioblastoma for the studies.
The gene therapy effectively starves the tumor by blocking its ability to grow new blood vessels, said Dr. Brenner, principal investigator of the studies. Tumors themselves begin the process by secreting a factor that activates the VB-111 drug.
“This drug outsmarts the cancer,” he said.
The drug is administered intravenously once every two months. The most frequent side effect in the study was fever, lasting one to two days following the infusion. This suggests an immune system response to the drug, which may play a role in its effectiveness, Dr. Brenner said.