Treatment 3 min read

FDA Says Yes to Yescarta

The second gene therapy approved for cancer is aimed at treating adults with certain types of non-Hodgkin lymphoma.

By Knvul Sheikh featured image Image courtesy Kite Pharma

On October 18, the Food and Drug Administration approved a second groundbreaking drug that uses patients’ own genetically modified immune cells to attack cancer.

The treatment, called Yescarta (axicabtagene ciloleucel), was developed by California-based Kite Pharma and approved for adults with relapsed diffuse large B-cell lymphoma (DLBCL), the most common aggressive form of non-Hodgkin lymphoma (NHL). While NHL is diagnosed in roughly 72,000 Americans each year, about one-third of those new cases are diffuse large B-cell lymphoma (DLBCL), in which relapse is common. Ten percent of patients have refractory disease where overall survival is counted in months.

“These are patients who are contemplating their own death at this point,” says Caron Jacobson, who helped conduct clinical trials for the treatment at the Dana-Farber Cancer Institute and Brigham and Women’s Cancer Center in Boston. “[Yescarta] is a novel way to treat them that shows great efficacy and great promise.”

Yescarta has also been approved for three other less common types of B-cell lymphoma: primary mediastinal large B-cell lymphoma, high-grade B-cell lymphoma, and DLBCL arising from follicular lymphoma.

As part of the treatment, doctors extract some of a patient’s T cells, which are a type of white blood cells critical to the immune system, Jacobson says. The cells are then frozen and shipped to Kite, where they are genetically re-engineered to recognize and kill cancerous cells. When the modified T cells are injected back into the patient, two to four weeks later, they can attack the cancer without harming healthy cells in the rest of the body.

It’s a one-time treatment that is customized for each patient. And it comes right on the heels of the FDA’s approval of the first chimeric antigen receptor (CAR) T-cell therapy under the name Kymriah (tisagenlecleucel). Kymriah, which was developed by Novartis, got the green light to treat acute lymphoblastic leukemia in children and young adults on August 30.

“These approvals of CAR T-cell therapies — both Yescarta and Kymriah — have ushered in a new era in treatment for blood cancer, and eventually, we hope, for other types of cancers as well,” says Gwen Nichols, chief medical officer of the Leukemia and Lymphoma Society.

Yescarta was approved after a six-month multicenter trial established the treatment’s safety and efficacy. In the trial, more than 100 patients received Yescarta, and 51 percent had complete remissions, meaning that their tumors disappeared entirely. “That is a remarkable result, especially for a patient population that until now had very few options and an estimated response rate in the single digits to available therapies,” Nichols says.

But the treatment is not without risk of side effects. Like Kymriah, Yescarta can cause cytokine release syndrome (CRS) in some patients, which results in high fevers, crashing blood pressure, and neurological problems. In some cases, patients required additional treatment in an intensive care unit, Jacobson says.

Yescarta is also pretty expensive. Kite has listed its price at $373,000 per patient. “Blood cancers are expensive to treat, and the cost of many of the exciting new therapies is a burden for patients and one of the many aspects of the financial burden of cancer care,” Nichols says.

Regardless of its price, the treatment will have a huge impact on standard of patient care, Jacobson says. It’s already providing options for patients who previously may not have had any. “I’m hopeful and optimistic that this will be a technology that can expand to other cancer types,” she says.