A US trial of a new cancer therapy has been showing remarkable results – and it could be approved by the US Food and Drug Administration for use by the end of this year.

“This new therapy could benefit up to 10,000 chemotherapy refractory cancer patients each year, patients without other hope,” says medical oncologist Frederick Locke, co-lead investigator on the Zuma 1 clinical trial, taking place in 22 centres nationwide.

On trial, a CAR-T cell product called axicabtagene ciloleucel (axi-cel; formerly KTE-C19) was given to patients with aggressive B cell lymphomas that had not responded to chemotherapy or returned after an autologous stem cell transplant.

“These are patients whose lymphomas have shown no response to the last treatment, and have a less than 10% chance of complete response with existing therapies” The possibility of using axi-cel for acute lymphoblastic leukaemia is also being researched.

“The patient’s T cells are collected from their blood and shipped to a central facility where they are reprogrammed with the CAR gene turning them into the equivalent of heat-seeking missiles targeting CD19, a protein on the surface of cancerous B cells. When they come in contact, the CAR-T cells destroy the lymphoma cells,” says Locke.

Results so far have been impressive. “On the Zuma-1 trial, of 101 patients given axi-cel, 54% saw a complete disappearance of the cancer, while another 28% saw at least a significant reduction,” says Locke. With a median of nine months follow up, 44% were still responding positively, with some patients in continued complete remission over one year after receiving a single infusion of axi-cel.

“If patients remain in remission for years following a single treatment, this could save thousands of lives and provide great value,” says Locke.