Weill Cornell to Test Cancer-Selective Viral Gene Therapy for Brain Tumors in New Clinical Trial

The Weill Cornell Brain and Spine Center is now enrolling patients in a new clinical trial testing a retroviral replicating vector (Toca 511) against recurrent glioblastoma multiforme (GBM) and anaplastic astrocytoma (AA).   

In the trial, which will be conducted at Weill Cornell by Dr. Rohan Ramakrishna, patients who are undergoing resection surgery for a first or second recurrence of GBM or AA will be treated with either the investigational treatment (Toca 511, which is used in combination with an oral medication called Toca FC) or with standard of care, which is either single-agent chemotherapy (lomustine or temozolomide) or bevacizumab (Avastin).

For patients in the investigational treatment arm of the study, the neurosurgeon will administer biological Toca 511, which encodes a modified yeast cytosine deaminase gene, into the wall of the tumor cavity after the tumor has been resected. Toca 511 is designed to infect remaining actively dividing cancer cells. Approximately six weeks after surgery, patients will take oral Toca FC, an extended-release formulation of an antifungal drug, flucytosine. The cytosine deaminase protein within these infected cancer cells then converts Toca FC to the anticancer drug 5-fluorouracil, which may result in effective tumor killing. A short video explaining how Toca 511 & Toca FC is designed to work is available here.

The trial is a randomized multicenter study that will compare overall survival of patients treated with Toca 511 and Toca FC to those given the current standard of care. For more information, contact Dr. Rohan Ramakrishna at 212-746-1996.

More information about this trial can be found at clinicaltrials.gov

Weill Cornell Medicine Neurological Surgery 525 East 68 Street, Box 99 New York, NY 10065 Phone: 866-426-7787