On Wednesday, the FDA approved Novartis’s Kymriah, also known as tisagenlecleucel, a treatment used for pediatric acute lymphoblastic leukemia.
“I think this is the most exciting thing I’ve seen in my lifetime,” stated Dr. Tim Cripe, an oncologist who was part of the FDA advisory committee panel that voted in favor of approving the drug last month.
The highly tailored treatment is called CAR T-cell therapy. It’s a type of cancer immunotherapy — or a therapy that binds the body’s immune system to battles cancer cells.
“We’re entering a new frontier in medical innovation with the ability to reprogram a patient’s own cells to attack a deadly cancer,” the FDA commissioner, Scott Gottlieb, reported. “New technologies such as gene and cell therapies hold out the potential to transform medicine and create an inflection point in our ability to treat and even cure many intractable illnesses. At the FDA, we’re committed to helping expedite the development and review of groundbreaking treatments that have the potential to be life-saving.”
Short for chimeric antigen receptor T-cell therapy, CAR-T treatment takes a person’s cells, eliminates them from the body, reengineers them, and then places the cells back in the body where they can cancer cells. Novartis’ therapy is one of two cutting-edge treatments for blood cancers that are positioned to get approved by the end of 2017.
Also, the FDA is anticipating making a decision on another CAR-T treatment from Kite Pharma, the company just got bought out by big pharma Gilead Sciences. That treatment is used for patients with aggressive B-cell non-Hodgkin lymphoma. Data released by Kite back in February, shows that out of 101 patients, roughly 36% had a complete response to the treatment just after six months.
It’s a type of cancer that Novartis wants to get approval for in the future. In June, Novartis released data from its Phase 2 trails CTL019 in patients with diffuse large B-cell lymphoma (DLBCL for short), an aggressive form of lymphoma that’s one of the two types Kite’s data monitors. The trial discovered that of the 51 patients with DLBCL, 23 had either a complete response (meaning cancer had gone away completely) or a partial response (meaning their tumor showed signs that it was decreasing).