A drug to fight leukemia in young people approved in the USA


An approval has been confirmed, as well as a finding, considered “historic” by the US Food and Drug Administration. This new drug, named Kymriah, will be produced by Novartis and will fight leukemia in patients up to 25 years.

The treatment is the first gene therapy to be allowed in the US and involves cells from the patients themselves to fight the disease. This will be used in children and young people whose disease does not respond to traditional treatments, is also being thought for other diseases and gives new signs in the treatment of cancer by mobilizing the body’s immune system, the Washington Post explained.

The treatment does not replace the genes that cause the disease with healthy genes, but instead a complicated process developed by the University of Pennsylvania picks up cells called T cells that are extracted from the patient and sent to a center in a way to be modified. Thus, new genes that target T cells to attack leukemia are included. These cells are then put back into the patient’s body, the same newspaper said.

“We are pushing the boundaries of medical innovation with the ability to reprogram a patient’s cells to attack a deadly cancer,” said FDA commissioner Scott Gottlieb.

The treatment is approved for patients up to 25 whose disease does not respond to treatment or who returns after care, as in 20% of cases, according to the FDA. And in a study of about 60 patients, this new drug resulted in remission in 80% of cases.

Therefore, it is still being studied how long the effect of treatment lasts on other people. The treatment is expected to be expensive and have side effects such as fever and neurological problems, which can be counteracted by another medication called tocilizumab.

The FDA, because of the risks and novelty of the treatment in question, requires hospital staff to be specially trained. Novartis has not yet revealed prices, but it is already being pressured on information about the cost of the drug.