In the summer of 2010, Novartis and the University of Pennsylvania began testing their anti-CD19 CAR-T therapy, CTL019, in patients with relapsed and refractory chronic lymphocytic leukemia (CLL). The mature results from the trial were published in Science Translational Medicine. Dr. Carl June led the study.
The trial enrolled 14 heavily pretreated CLL patients. These patients were treated with CTL019 at doses of 1.4 × 10^7 to 11 × 10^8 cells. Four patients had a complete response (CR), and additional four patients had a partial response (PR). Six patients did not respond to the therapy and progressed soon.
One of the CR patients died at 21 months due to infections after removal of the skin cancer on his leg. The three other patients are still alive with no signs of recurrence. At the time of the report, they have survived for 28, 52, and 53 months after receiving CTL019.
Two of the PR patients died of disease progression at 10 and 27 months. One of the PR patients died from a pulmonary embolism (肺动脉栓塞) at 6 months. The other patient was switched to other therapies due to the disease progressed at 13 months.
The CR rate in CLL was much lower than that in ALL (acute lymphocytic leukemia). No CLL patient with CR has relapsed, while in a previous ALL trial, 37% of CR patients have subsequently relapsed. The CAR-T cells remain in CLL patients’ blood and retain their ability to hunt for cancer cells beyond four years.
 Sci Transl Med. 2015, 7(303), 303ra139.
 ASH2014, https://ash.confex.com/ash/2014/webprogram/Paper69932.html