Novel approach to treat lymphoblastic leukemia “Kymriah” and its scope in India

Abstract

Acute lymphoblastic leukemia is a type of cancer which occurs when a bone marrow cell develops errors in its DNA and affects the white blood cells. Kymriah is first FDA approved gene therapy which treats B-cell acute lymphoblastic leukemia (ALL) in patients aged 25 years and younger. It is used in patients whose cancer has relapsed or is refractory. It is formulated in-vivo using a patient’s T cells and a gene, code for a special receptor called chimeric antigen receptor (CAR). It is reprogrammed to destroy cancerous B-cell and is administered by IV infusion. Tisagenlecleucel is a CD19-directed genetically modified autologous T cell immunotherapy marketed as Kymriah by Novartis. The whole process takes 22 days as the T cells from a person with cancer are removed, genetically engineered to make a specific T-cell that reacts to cancer, and transferred back to the person. It was invented and initially developed at the University of Pennsylvania; Novartis completed development, obtained FDA approval, and markets the treatment. In August 2017, it became the first FDA-approved treatment that included a gene therapy step in the United States. It is administered in a single treatment, which will have high cost, if not successful money is refunded. Present study highlight first gene therapy based “LIVE” treatment for lymphoblastic leukemia and conducts a survey on knowing the attitude of Indian population towards gene therapy to understand the scope of Kymriah in India.