The FDA or the Food and Drug Administration announced earlier this week that it gave its official approval to a new gene therapy used for treating leukemia in the United States.
An FDA board gave its approval to Kymriah, which is being referred to as a “living drug” by scientists. An advisory panel of the FDA recommended it earlier this month, at the beginning of July.
First Gene Therapy for Leukemia Will Come With Strong Warnings
This new drug therapy is being called a ‘living drug’ because of its structure. It makes use of genetically modified immune cells taken from the patients themselves. These are then specially modified to attack the cancer and help the body cure itself.
Kymriah was approved for treating children and young adults up till the age of 25 suffering from ALL or acute lymphoblastic leukemia. It will be used in cases which registered relapses or in which the standard treatment did not work.
ALL is one of the most common forms of childhood cancers in the United States, a cancer of the bone marrow and blood. Some 3,100 patients 20 years old or younger get diagnosed with it each year in the U.S.
“Kymriah is a first-of-its-kind treatment approach that fills an important unmet need for children and young adults with this serious disease,” states Peter Marks, the FDA Center for Biologics Evaluation and Research’s director.
Although hailed as a much-needed alternative, this first gene therapy for leukemia will also come with some strong warnings. These are based on the drug’s potential to cause a dangerous overreaction in the patient.
In such cases, their immune system can lead to a reaction known as the cytokine-release syndrome. So the FDA, besides giving its approval, also required that strong warnings be placed on the new treatment.
Also, this will initially be available in only 32 hospitals and clinics nationwide. However, their staff was specially trained in administering Kymriah.