ATMP Cellbased gene therapy

In the United States, the U.S. Food and Drug Administration (FDA) has approved a gene therapy for the first time at 30th of august. Henceforth, the treatment of acute lymphoblastic leukemia (ALL) in young patients up to 25 years is allowed, who couldn’t be cured with actual methods so far.

Even the FDA describes this as a historic step towards treating cancer and other life-threatening diseases with completely new approaches.

“We’re entering a new frontier in medical innovation with the ability to reprogram a patient’s own cells to attack a deadly cancer,” said FDA Commissioner Scott Gottlieb, M.D.

Kymriah is based on the chimeric antigen receptor therapy known as CAR T-cell therapy. Patient's own T-cells are genetically modified in vitro using viral vectors in such a way, that they detect specific antigens (CD19) on the surface of the tumor cells, when given back to the patient.

This method of genetically modified autologous T-cell immunotherapy does not only attack the tumor directly, but activates the patient's own immune system to take targeted action against the cancer.

The safety and efficiency of the Kymriah method has been tested in a study with 63 ALL patients (children and young adults). The remission rate was 83% within the first three months. Despite severe side effects, which can arise and a lack of long-term prognoses, treatment with Kymriah is a great opportunity for young patients with ALL who hasn’t had hope any longer.

FDA news release