The Novartis CAR-T cancer therapy is recommended for patients with B-cell ALL that has not responded to other treatments or has responded but relapsed.
Treatment with CTL019 involves removing some blood from the patient and modifying the T-cells in the blood before the cells are returned into the body. The advisory panel determined, unanimously, 10-0, that the benefit of the therapy outweighed the risks.
“It is encouraging to see the FDA panel’s recommendation and continued momentum behind this innovative therapy”, said the Penn team’s leader, Dr. Carl June. The FDA doesn’t have to follow the advice of the advisory committee, but it often does. Around 60 percent of them are young adults and children. There are about 3,100 new cases of ALL each year, but roughly 70 percent can be pushed into remission by standard therapy.
Scientists are calling the treatment “a living drug” as it harnesses the immune system to beat cancer, according to The New York Times. Since that time she has been cancer free. The more significant question for FDA advisers involved safety.
In 2012, she was dying from one of the most common forms of leukemia called ALL.
But the therapy poses serious risks. The syndrome – characterized by high fevers, organ failure and other symptoms – can be life-threatening.
But there have been no deaths from CRS in CTL019 trials so far, and the panel was satisfied that expert clinicians administering the therapy would be able to deal with CRS effectively. One possibility is that the agency is anticipating that CAR-T products will be developed for patients who aren’t at the end of the line.
“Although this therapy is technologically somewhat complicated and is associated with certain serious side effects, it, indeed, has been proven to be amazingly effective”, said Kanti Rai, chief of the chronic lymphocytic leukemia research and treatment program at Northwell Health Cancer Institute in NY.
The FDA is expected to decide whether to approve the Novartis treatment in the next few months. To use the technique, the removed cells must be frozen and shipped to a Novartis plant for thawing and processing. There, staff use a virus to insert genes into the T cells that encode a cellular receptor that will recognize leukaemia cells. “These challenges can include variability in the starting materials (e.g., patient’s own leukapheresis cells) and human- or animal-derived reagents (e.g., serum, antibodies), and control of critical components that may be manufactured under contract (e.g., transfer vectors that encode auto, final container)”.
The treatment is complex and must be personalized for each patient.
Several committee members expressed concern about the uncertainties that still swirl around the therapy. He says CAR-T is more precise than Chemotherapy, which attacks all rapidly dividing cells instead of just the cancerous ones. The M.D. Anderson approach, by contrast, involves taking donated cord blood, separating out NK cells, and inserting a CD19-targeting auto into them.
Aiman Shalabi, chief medical officer of the Cancer Research Institute, viewed the positive tone of the meeting and unanimous recommendation as a similarly momentum-generating event for CAR-T and immunotherapy more broadly.
The drug turns the patient’s own immune system against the cancer. “Without them, we wouldn’t have had a clinical trial and I don’t think we’d be where we are today”.