New gene therapy to cure brain cancer

new gene therapy to cure brain cancer

A study was conducted by the American Association for Cancer Research (AACR) in which a gene therapy was designed to treat brain cancer. This clinical research comprises Phase I clinical trial of over 56 patients, who were suffering from high-grade Glioma Brain Cancer.

The clinical trial includes three years gene therapy to the patients. According to the report, more than a quarter of the patients are still alive. Median survival for the patients was 14.4 months, while it is typically 8 months in patients with the same disease.

Dr. Clark Chen, HOD of Neurosurgery at University of Minnesota Medical School presented his comments on the report of American Association of Cancer Research after studying it. He stated, “Given the deadly nature of this disease, three-year survival is rarely reported in the recurrent setting. It is notable that the survival benefit was seen across a range of patients, and not just limited to patients with specific genetic mutations.”

The results of the research will be published at the International Conference on Molecular Targets and Cancer Therapeutics in Philadelphia. The sponsor of the research was therapy maker, Tacogen. The study was found to be effective for treating the patients.


Chen explained, “The treatment we tested in this trial delivers local chemotherapy specifically to the brain tumor. Toca 511 and Toca FC work together to turn the brain tumor into a factory that produces an anti-cancer drug while also activating the immune system through a combination of mechanisms, which together work to attack the cancer.”

The therapy involves the transfer of a replicating virus called Toca 511 inside a patient body, which targets the actively dividing cancer cells. This virus when enters the cancer cell deliver a gene, which produces an enzyme called cytosine deaminase. After this, the patients were given an oral medicine Toca FC. This medicine when comes in contact with cytosine deaminase, gets converted into an anti-cancer drug and naturally kills the cancer cells.

Chen considered the therapy highly effective and concluded, “The Toca 511 therapy approach spares the body from exposure to systemic chemotherapy, while creating high concentrations of chemotherapy in the tumor cells and their microenvironment.”

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