Press briefing moderator George Daley, MD, PhD, of Boston Children’s Hospital, was enthusiastic about these results, despite the small number of patients treated in studies of CAR-T cells thus far. Most of the results have been in a positive direction, he noted. “It is remarkably exciting to see clinical proof of principle for CAR‑T therapies and other gene therapies,” Dr. Daley said.

At a premeeting teleconference, outgoing ASH President David Williams, MD, of Boston Children’s Hospital, singled out this study as important. “Two immunologic approaches are gaining attention in hematologic malignancies. One is to take the brakes off of the immune system [with checkpoint inhibitors]. Another is to use genetically engineered immune T cells to ‘put the pedal to the metal,’” Dr. Williams told listeners.

“The study of modified T cells takes the donor’s T cells and revs them up. Although this could theoretically increase graft-vs-host disease, which is not an issue with the patient’s own cells, no graft-vs-host disease occurred in this study,” he pointed out. ■

Disclosure: Dr. Daley disclosed financial relationships with True North Therapeutics, Raze Therapeutics, Ocata Therapeutics, MPM Capital, Solasia, Epizyme, and Verastem.