Groundbreaking Cancer Gene Therapy

On July 12, a Food and Drug Administration panel unanimously recommended approval for the first-ever gene therapy treatment for cancer. The treatment, known as CTL019, is a T-cell therapy developed by the pharmaceutical company Novartis. It is tailored for each individual patient and has already been proven effective for treating a type of childhood leukemia. The New York Times reportsthat in a study of 63 patients, 52 of them went into remission after receiving the treatment.

Researchers have long been working to perfect gene therapy for a variety of cancers, but CTL019 will be the first to reach the market. If the FDA moves to approve CTL019, the decision could open the door for more gene therapy treatments for other diseases.

Mansoor Amiji is University Distinguished Professor in the Department of Pharmaceutical Sciences at Northeastern. His research focuses on the development of targeted therapies, including gene therapy, for treatment of the most lethal cancers, such as pancreatic, lung, ovarian, and brain tumors, as well as other chronic diseases. For one project, Amiji’s lab is interested in reprograming immune cells through genetic engineering to become more effective in treating cancer and inflammatory diseases.