Novartis, Juno Conduct New Studies on Leukemia Therapies

Cancer treatments that genetically modify patients’ blood cells to target the disease have shown amazing results in clinical trials. Now drug companies and biotechs must overcome big hurdles to get them into hospitals, including their potential cost.

In two separate clinical trials—sponsored by Novartis AG NOVN.VX -0.51% of Switzerland and Seattle-based biotech Juno Therapeutics Inc.—almost 90% of patients saw their leukemia disappear after being given experimental so-called CAR T-cell therapies. The results were published in December and February, respectively.

Both trials were in small numbers of patients: 22 children in the Novartis trial and 16 adults in the Juno trial. The patients had acute lymphoblastic leukemia—the most common childhood cancer—and had exhausted standard treatments. Both companies are now conducting larger trials.

“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, who wasn’t involved in either study.

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 in an interview with The Wall Street Journal. “Our job is to get this into patients as soon as we feasibly can.”