Leukemia : first gene therapy in the United States

Novartis

Published the 02.09.2017 at 08h17

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leucémieNovartisimmunotherapie

This Wednesday, the Food and Drug Administration (FDA), the u.s. equivalent of MSNA, has approved the first gene therapy against cancer. This is the Kymriah, which has been developed by the laboratories of Novartis in the fight against acute lymphoblastic leukemia in children and young adults, up to 25 years.

“There was an urgent need for therapeutic options that are innovative, that could improve the chances of survival of patients with relapse or resistance, and for which the prognosis is bad,” says Novartis in a press release. Patients are often subjected to multiple treatments, including chemotherapy, radiation therapy, targeted therapy or stem cells, but only 10 % are still alive 5 years later. “

Reprogrammed cells

What is proposed in the Kymriah, this is a single treatment, with a remission rate of 83% in the three months following treatment. It is individualized. T-lymphocytes, cells of the immune system of the patient, are collected, reprogrammed, cultured and frozen. They are modified to attack other lymphocytes failed, causing the disease, which proliferate quickly.

They identify a protein, CD19, which is expressed at the surface of these cells, and attack them. Once re-injected into the patient’s body, the T cells modified to replicate quickly, and the remission is rapid.