Last fall, the U.S. Food and Drug Administration approved Yescarta, a new type of cancer treatment that trains a patient’s immune system to seek and destroy lymphoma tumors.

In patients with lymphoma, the body’s disease-fighting T-cells don’t latch onto substances in the lymphoma cells — “antigens” — that typically stimulate the body’s defenses.

Yescarta is a gene therapy: The patient’s T-cells are removed from the blood, sent to a lab in California and then re-engineered with a “chimeric antigen receptor” (CAR). After being infused back into the patient, the T-cells — now CAR-Ts — are able to attach themselves to an antigen in the lymphoma and destroy the cancer.

The approval followed a national, twoyear clinical trial, co-led by Moffitt Cancer Center in Tampa, in which doctors administered the CAR-T cell therapy to about 100 adult non-Hodgkin’s lymphoma patients who already had undergone two failed regimens of chemotherapy.

“They were without other options, without hope,” says Dr. Frederick Locke, a Moffitt researcher who helped lead the study. About half of the patients were tumor-free six months later, and 41% remained in remission at the 15-month mark. “The results are pretty remarkable,” Locke says.

Yescarta is made by Kite Pharma, a subsidiary of Gilead Sciences. Given as a onetime treatment, Yescarta costs $373,000 and carries serious potential side effects, including life-threatening fevers, high blood pressure and neurologic problems, such as memory loss, seizures and confusion.

Locke says ongoing research will focus on reducing the side effects and understanding why all patients don’t benefit from treatment. Researchers also want to determine whether CAR-T cell therapy should be given to patients in earlier stages of the disease as an alternative to second-line chemotherapy.

“Is it the answer to other cancers?” Locke adds. “There’s more work to be done.”

Meanwhile, Moffitt has begun commercial use of Yescarta to treat non-Hodgkin’s lymphoma. Insurers generally are covering the expensive treatments, Locke says. “We’ve had conversations with insurers, and they understand the benefits.”

The FDA also has approved another CAR-T cell therapy, Kymriah, marketed by Novartis Pharmaceuticals, to treat acute lymphoblastic leukemia in children and young adults. Kymriah and Yescarta are the first CAR-T cell therapies to receive FDA approval.

“I think the FDA has done a good job of looking at the safety of these therapies,” Locke says, noting that the Yescarta trial ended with FDA approval after two years. “That’s amazing for any therapy, let alone a therapy like this.”

Patient Profile: Dimas Padilla

‘I’m completely in remission.’

“I was losing my battle against cancer,” says Dimas Padilla, 44, of Kissimmee. After being diagnosed with non-Hodgkin’s lymphoma in 2011, Padilla underwent chemotherapy and enjoyed a year of remission before the cancer returned. He then had a bone marrow transplant, but that remission lasted only two years, and he did not respond to another round of chemotherapy. “I had about six months to live.

In mid-2016, Padilla enrolled in the Yescarta trial at Moffitt. By then, lymph nodes in his neck had prevented him from talking. Over the course of two weeks, T-cells were removed from his blood and sent to a lab in California, where they were reprogrammed to attack his cancer, then infused intravenously into his body.

Padilla says his neck tumors disappeared almost immediately, but he also experienced side effects that landed him in the ICU. “I couldn’t remember how to spell my name or the names of my daughters. I had fevers of almost 105 degrees, and my heartbeats were crazy.”

About a month later, however, Padilla was on the upswing. In January, he celebrated 16 months of being cancer-free. “I’m completely in remission,” he says. “I’m one of the lucky ones.”

 

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