The United States authorizes the marketing of gene therapy against leukemia

2017/08/31 Galarraga Aiestaran, Ana - Elhuyar Zientzia Iturria: Elhuyar aldizkaria

• Genetika
• Medikuntza
• Farmakologia

For the first time, the U.S. Department of Food and Drug Administration (FDA) has authorized the marketing of gene therapy. It will be marketed by the pharmaceutical company Novartis under the name Kymriah and is aimed at children and young people with acute B-cell lymphoblastic leukemia.

Kymriah is a personalized immunotherapy, adapted to each patient. Patient T-cell reception and genetic transformation. Specifically, a gene is introduced so that these T cells can be associated with the CD19 antigen of leukemic cells. These genetically modified T cells are injected into the patient and destroy leukemia cells.

They warn that side effects can be serious, so now they are investigating how to reduce them. In addition, it is not the first option of those who suffer it, it is only given to patients who have not benefited from the initial treatment (15-20% of patients). In any case, the FDA decision has opened the doors to those who are developing similar treatments for other diseases and has given hope to patients suffering from them.