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In this photo taken March 29, 2017, Dr. David Maloney of the Fred Hutchinson Cancer Research Center is greeted by patient Ken Shefveland, whose lymphoma was successfully treated with CAR-T cell therapy
- The FDA approved a second gene therapy to treat cancer, called Yescarta.
- The therapy works by taking a person's own cells, reprogramming them, and inserting them back into the body to fight the cancer - in this case, certain forms of blood cancer.
- This second approval could have a bigger impact than the first, because it could be used treat more than 10,000 adults a year, whereas the original approval could only impact a few hundred a year.
A wave of new cancer treatments that reprogram a person's own cells to fight cancer is gaining momentum.
On Wednesday, the FDA approved Yescarta, which treats a type of blood cancer called aggressive B-cell non-Hodgkin lymphoma.
The highly personalized cancer treatment is a type of CAR T-cell therapy (CAR is short for chimeric antigen receptor). It was the second of such treatments to get approved following the August approval of Kymriah, which was approved to treat pediatric acute lymphoblastic leukemia in people up to age 25.
But Wednesday's approval could up being a much bigger deal, in large part because the drug is approved in adults.
Dr. Fred Locke, an oncologist and researcher at Moffitt Cancer Institute in Tampa, Florida, estimates that there are more than 10,000 adults who might benefit from these treatments, whereas the pediatric approval was initial geared for only about 600 patients a year. Moffitt has been working with CAR-T since 2015, helping run clinical trials for Kite Pharma, the company that developed Yescarta that has since been acquired by Gilead Sciences.
Novartis, the company that makes Kymriah, is also working to get the drug approved in an aggressive form of lymphoma in adults, which could introduce some competition into the adult market.
That competition could help drive down prices, as the one-time treatments don't come cheap. Yescarta has a list price of $373,000, which is lower than Kymriah's $475,000 price tag. Because not everybody with these advanced forms of cancer respond to the treatment, if patients don't respond to Kymriah within a month, the patient doesn't have to pay for it. Yescarta did not make the same arrangement.
How CAR-T cell therapy works
Yescarta and Kymriah aren't your run-of-the-mill pill - or even a biologic drug, like insulin - that can be mass produced. Since the therapy is made from a person's own immune system, the process can take about three weeks.
- To start, a doctor removes some white blood cells, the part of our body's immune system responsible for combatting infections and foreign substances, from a patient. In a healthy body, the immune system can recognize abnormal, cancerous cells, but for people with cancer, it doesn't recognize that the cells are spreading.
- Then the cells are taken to a manufacturing facility at which point the cells are reengineered to recognize cancer cells and wipe them out.
- Those reprogrammed cells are sent back and administered to the patient.
Ana Pelisson/Business Insider; Novartis
While that's a one-time process, it's not the end of the road. Many experience cytokine-release syndrome, a response to the reprogrammed cells running loose in the body. The treatments can also cause neurotoxicity, which can lead to brain damage. The side effects can lead to high fevers and flu-like symptoms, and it can be life-threatening.