Have you been diagnosed with a brain tumour? Order your free information pack.

Gene therapy to treat recurrent high-grade gliomas gets accelerated to Phase 3 clinical trials

A gene therapy to treat recurrent high-grade gliomas is accelerated to Phase 3 clinical trials, after the success of Phase 1 clinical trials by the US Food and Drug Administration (FDA)

Gliomas are the most common primary brain tumours and are classified as either low grade or high grade. High-grade gliomas are malignant, fast-growing tumours that have a high chance of recurrence.

Patients with recurrent high-grade gliomas have a median survival of approximately seven to nine months. With such poor prognosis, there is a huge unmet need for treatments to improve the survival and quality of life of patients with high-grade gliomas.

A clinical-stage, cancer-selective gene therapy company, called Tocagen, has shown promise to meet the need for new treatments for high-grade gliomas. Their gene therapy treatment consists of two drugs, Toca 511 and Toca FC.

The two components of this treatment have the ability to cross the blood brain barrier and target the tumour cells. The drugs Toca 511 and Toca FC work together, with Toca 511 containing the protein which has the ability to convert Toca FC to the anti-cancer drug 5-fluoracil.

The gene therapy has successfully completed Phase 1 clinical trials, with 23 patients out of 56 demonstrating increased survival rates.

Based on the success of Phase 1 trials, the FDA has accelerated the approval of this treatment by combining Phase 2 and 3 clinical trials.

“Patients and physicians urgently await new treatments for high-grade glioma, one of the deadliest cancers,” says Asha Das, chief medical officer of Tocagen.

“We are encouraged by the maturing durable response results of our earlier trial, and remain committed to advancing Toca 511 & Toca FC through the ongoing Phase 3 Toca 5 trial to bring a potentially transformative product to patients as quickly as possible.”

This treatment could have a tremendous clinical impact, as it could significantly increase the survival of individuals diagnosed with recurrent high-grade gliomas.