A new form of gene therapy for acute lymphoblastic leukemia has received approval from the U.S. Food and Drug Administration (FDA). Using the technique, doctors can reprogram a patient’s immune system to attack cancer, according to FDA commissioner Scott Gottlieb. This is the first gene therapy to be approved in the United States for the treatment of cancer.
Acute lymphoblastic leukemia is a cancer of the blood and bone marrow that causes the body to produce abnormal lymphocytes. The disease affects children and young adults and is the commonest form of cancer to affect children. More than 3,100 new cases are diagnosed each year. This new form of gene therapy for acute lymphoblastic leukemia could be a real game changer for patients suffering from the disease and could usher in a new era of cancer treatment.
While physicians can treat acute lymphoblastic leukemia, approximately 15%-20% of patients do not respond to treatment. The FDA has approved the use of this new gene therapy for acute lymphoblastic leukemia for patients that have not responded to initial treatment and for patients who have been treated only for the cancer to return.
The drug – called Kymriah (tisagenlecleucel) – is produced by Novartis, with the one-time treatment costing approximately $475,000. Initially, the expected price of treatment was expected to be in the region of $600,000 to $700,000 per patient. As with other treatment methods, this gene therapy for acute lymphoblastic leukemia may not work. Novartis has said that there will be no charge for the treatment if patients do not respond within a month.
Kymriah is a genetically-modified autologous T-cell immunotherapy that uses a patient’s own T-Cells to attack the cancer. T-cells are harvested from a patient and are genetically modified and reprogrammed with a new gene responsible for synthesizing a protein called a chimeric antigen receptor, or CAR. That protein helps T-Cells target leukemia cells as the receptor corresponds to a specific antigen on the surface of leukemia cells. The treatment takes approximately 22-days from the cells being harvested to them being infused back in patients.
There are two forms of the cancer, one which has a T-cell origin and one which has a B-cell origin, the latter being the most common. The new treatment has only been approved for B-cell origin acute lymphoblastic leukemia.
In a recent clinical trial conducted on 63 children and young adults with acute lymphoblastic leukemia, 83% of patients went into remission within 3 months of receiving the one-time treatment, and 89% of patients were still alive 6 months after treatment. There was a 79% survival rate 12 months after treatment.
The treatment will be made available at 20 hospitals throughout the United States, with Novartis planning to expand the centers offering the treatment to 32 hospitals.