Novartis, Juno Conduct New Studies on Leukemia Therapies

“CAR T cells are probably one of the most exciting concepts and fields to come out in cancer in a very, very long time,” says Dr. Daniel DeAngelo, a Boston-based hematologist and associate professor of medicine at Harvard Medical School.

Usman Azam, head of cell and gene therapies at Novartis, calls the therapies “critically important” for Novartis. “I think that a cure for cancers such as leukemia and lymphoma through a CAR technology is plausible,” said Dr. Azam . “Our job is to get this into patients as soon as we feasibly can.”

Dr. Azam heads a new unit Novartis created partly to speed the therapies’ time to market. Its leading CAR therapy was granted ‘breakthrough’ designation by the U.S. Food and Drug Administration in July, and Novartis wants to file it with regulators in 2016.

CAR therapies are a mixture of genetic tweaking and “immunotherapy,” or using the patient’s own immune system to fight disease. They involve extracting disease-fighting white blood cells called T-cells from a patient’s blood. The T-cells are then genetically modified, grown in a laboratory for around 10 days and reinjected into the patient.

Typically, the T-cells are combined with a disabled virus, which enables them to produce chimeric antigen receptors, or CARs, that recognize and target malignant proteins on a cancer cell’s surface.

Novartis and Juno are developing their treatments in partnership with top academic teams: Novartis with the University of Pennsylvania and Juno with teams at Memorial Sloan-Kettering Cancer Center in New York, Seattle Children’s Hospital and the Fred Hutchinson Cancer Research Center, also in Seattle.