The first gene therapy was approved in the USA


It is an innovative treatment for leukemia, which modifies the DNA of the patient’s own immune system cells to kill the cancer cells, which can now be marketed in the United States. The Food and Drug Administration (FDA) has approved the treatment, named Kymriah, this week.

The therapy consists of using the cells of the immune system, more precisely the T lymphocytes, present in the body of the patient himself. These cells are removed from the patient and sent to a laboratory to be genetically modified with the help of a virus. The cells gain genetic information that allows them to detect and kill the cancer cells. Then, the cells are reintroduced into the patient’s body, having the ability to recognize and kill the source of the disease.

This therapy will be used specifically for acute lymphoid leukemia (a type of leukemia that occurs in the bone marrow and in which the cancer cells are the precursors of the cells of the immune system). The disease occurs mainly in children. Most patients are cured with chemotherapy or radiation therapy, but when there are relapses there is usually no cure for it.

Thus, the new therapy is only used if the first treatments are not successful. Clinical trials of the new therapy show that 79% of people treated survive another year, and most do not have any relapses.

“The FDA issued a history action providing the first gene therapy in the United States, ushering in a new approach in the treatment of cancer and other serious and possibly deadly diseases”, the agency said in a statement. “The FDA has approved Kymriah for certain pediatric patients and young adults with a form of acute lymphoblastic leukemia”.

“We are entering a new front of medical innovation with the ability to reschedule the patient’s own cells to attack a deadly cancer”, said Scotti Gottlieb, the FDA’s commissioner”.

The FDA also warns that hospitals and clinics that want to implement the procedure must have a certificate for this purpose. Since the treatment can cause a reaction of the immune system that causes excessive decrease of blood pressure and can kill the patient. Institutions wishing to use Kymriah will need to be prepared to respond quickly to this side effect.

Novartis, a pharmacist responsible for Kymriah, also revealed that the base price for this treatment is $ 475,000, but patients who do not improve within the first month after treatment will be free of any cost. The company will have to continue to conduct studies to ensure the safety of Kymriah in the long run.