CAR T-cell therapy uses T cells (illustrated above) from a patient’s own immune system to attack cancer.

What if the key to a cure for cancer is already inside our own bodies?

Scientists at BWH and Dana-Farber Cancer Institute have asked that question over the past several years as they studied an immunotherapy – that is, a treatment that uses a person’s own immune system – for adult cancer patients.

Now, following a successful clinical trial and recent approval from the U.S. Food and Drug Administration (FDA), Dana-Farber/Brigham and Women’s Cancer Center (DF/BWCC) is one of a few locations nationwide certified to offer the first chimeric antigen receptor (CAR) T-cell therapy for a form of non-Hodgkin lymphoma.

CAR T-cell therapy, like all forms of cancer immunotherapy, seeks to sharpen and strengthen the immune system’s inherent cancer-fighting powers. It involves giving patients modified versions of their own immune system’s T cells – white blood cells that help protect the body from disease.

“Treating patients with CAR T cells has been one of my most exciting professional experiences, and the FDA approval of this therapy offers hope and optimism to a subset of patients whose other treatments have failed them,” said Caron Jacobson, MD, medical director of the Immune Effector Cell Therapy program at DF/BWCC. “It is extremely rewarding to be able to offer a new therapy to patients who had virtually no other options just 12 to 24 months ago.”

Clinical Trial Demonstrates Safety, Effectiveness

The drug, known as Yescarta (axicabtagene ciloleucel), was developed by Kite Pharma and can be used to treat adults with refractory aggressive B-cell non-Hodgkin lymphoma.

Over the past couple of years, Jacobson and her team have been testing Yescarta in a clinical trial at DF/BWCC, the only facility in the northeast that was part of the trial.

The FDA ruling is based on the results of this nationwide trial, which showed the therapy to be safe and effective. Of the 101 patients who received Yescarta, 82 percent responded to the treatment, with 54 percent having a complete response to therapy. Thirty-six percent of patients remain in complete remission six months after treatment.

“This therapy requires just a one-time infusion for patients, and the results are evident within one month,” Jacobson said. “It is our goal as clinicians to help patients and improve their quality of life. Seeing these patients return to work, their families and their livelihoods so quickly is an important reminder of how far we have come. It is also inspiration for the work we still need to do.”

The initial clinical trials of CAR T-cell therapy have involved pediatric and adult patients with blood-based cancers such as leukemia, lymphoma and multiple myeloma. Based on the therapy’s success so far, CAR T-cell therapy trials are now opening for certain types of solid tumors as well.

“The successful development of CAR T-cells as a therapy for cancer is a testament to the progress we have made in understanding how our immune system is regulated and how cancer evades the immune system,” Jacobson said. “It is a perfect example of how basic science research can fuel clinical progress. Now we need to take what we can from the clinic back to the laboratory to make this therapy even better.”