US FDA approves second gene therapy for cancer

23 October 2017

The US Food and Drug Administration has approved a second gene therapy for cancer, which is the first to target non-Hodgkin lymphomas.

The therapy, Yescarta (axicabtagene ciloleucel), is indicated for adult patients with certain types of large B-cell lymphoma after other treatments fail.

Non-Hodgkin lymphomas, which account for about 4 per cent of all cancers in the US, are malignancies that begin in certain cells of the immune system. According to estimates, 72,000 new cases of the disease are diagnosed each year in the US.

Diffuse large B-cell lymphoma is the most common form of non-Hodgkin lymphoma, accounting for about one in three newly diagnosed cases.

Upto half of all patients with large B-cell lymphoma relapse or develop resistance to treatments, which may include chemotherapy, stem cell transplants, and immunotherapy.

According to a statemement by Dr Scott Gottlieb, commissioner of the FDA, the approval of the gene therapy has ''gone from being a promising concept to a practical solution'' for treating deadly forms of cancer in just several decades. He termed the approval a ''milestone.''

California-based Kite, which is a subsidiary of drug giant Gilead Sciences had developed the new drug. The company develops chimeric antigen receptor and T-cell receptor (CAR-T) cell therapies, which use a patient's own T-cells to seek and destroy cancer cells.

CAR-T treatment gene therapy techniques work not on disease-causing genes, rather they turbocharge T cells, the immune system soldiers. Cancer can give the slip to T cells. 

The T cells are filtered from a patient's blood, and reprogrammed to target and kill cancer cells. Hundreds of millions of copies of the cells are then grown.

On return to the patient, all the revved-up cells continue to multiply and fight the disease for months or years. That is the reason for immunotherapy treatments to be called ''living drugs.''

''Today's approval of Yescarta is a very significant advance for lymphoma patients and for the cancer community as a whole,'' Louis J DeGennaro, president of the Leukaemia & Lymphoma Society, said in a statement. ''Immunotherapy is dramatically changing the way we approach blood cancer treatment.''

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