Three things health execs need to know about CAR T-cell therapy

Frederick Locke, MD, a medical oncologist at Moffitt Cancer Center, is excited that Gilead’s Yescarta (axicabtagene ciloleucel), developed by Kite Pharma, was approved by the FDA in October for the treatment of adult patients with relapsed or refractory large B-cell lymphoma. He’s also enthusiastic about the federal agency’s approval in August of Kymriah (tisagenlecleucel) from Novartis for the treatment of certain pediatric and young adult patients with a form of acute lymphoblastic leukemia.

In addition to the FDA approval of these two treatments, here are three things oncologists need to know about CAR T-cell therapy:

1. There are very positive results from clinical trials.

“All you have to do is look at the results of the clinical trials” to see why oncologists-and patients-should be excited about these therapies, says Locke. He was principal investigator for Kite’s ZUMA-1 clinical trial for the treatment of patients with chemorefractory aggressive B-cell non-Hodgkin lymphoma.

The study included 110 lymphoma patients, whose tumors were growing even though they were receiving chemotherapy, who were treated with Yescarta. Locke notes that these were “very refractory patients” and historical data holds that they should have no more than six months to live and a less than one in 10 chance of having a complete disappearance of their lymphoma.

With one infusion of CAR T cells, 82% of these patients had an objective response, and over 50% had a complete disappearance of their lymphoma. Most importantly, with a median follow up of almost nine months, 44% of patients remained in remission, he says.

“It’s exciting because these are patients without any hope,” says Locke of patients who have received the treatment. “Chemo is not working. They are out of options, so we’re able to get a large portion of them into remission; those are durable remissions.” 

2. There is a reduced chance of side effects, but when they do occur, they can be very serious.

In explaining the treatment to patients, oncologists should note that it uses the patient’s own cells, which are redirected against their cancer, says Locke.

In centers with experience, toxicities are manageable, and a relatively fit patient should be able to handle the therapy, he adds. However, if a patient has many comorbidities or is in their 80s, for example, this might not be the right treatment.

Nabil Saba, MD, director of the head and neck medical oncology program at the Winship Cancer Institute of Emory University, adds that most patients who undergo CAR T-cell therapy experience a better quality of life than patients experiencing chemotherapy; and chemotherapy patients tend to deteriorate more rapidly during treatment.

There’s a reduced chance of side effects with CAR T-cell therapy when compared with chemotherapy, says Saba. Still, side effects can occur with CAR T treatment-and some can be as serious as liver failure, activation of an autoimmune disease, colitis, or death.

Next: Costly, but only option 

3. Cost is an issue for payers, but these are patients without any other options.

Locke has had discussions with at least one major payer and has witnessed their receptivity to CAR T-cell therapy. “There’s really no other pathway for [these] patients,” says Locke. “The only alternative is rounds of therapy that we know didn’t work … or they go to hospice.”

What could work well with payers is the centers of excellence approach, which is used with stem cell transplants, says Locke. That’s because CAR T-cell therapies are complex and require expertise to manage the unique toxicities; it also involves handling a patient’s cells, which means the healthcare facility has to collect the cells, ship, and receive the cells, and then infuse the cells in the patients.

Still, the treatments are expensive: $475,000 for Kymriah and $373,000 for Yescarta. In addition, the healthcare facility administering the treatment needs to be reimbursed for the care provided to patients, adds Locke.