The U.S. Food and Drug Administration (FDA) announced its first ever approval of a cell-based gene therapy on Wednesday.
The drug, Kymriah, takes about two to three weeks to be made and is taken from a patients’ white blood cells. The genetically modified T-cells are made to attack cancer in children and young adults up to age 25 diagnosed with a form of acute lymphoblastic leukemia.
Most patients who have this form of leukemia, referred to by the acronym ALL, either do not respond to standard treatments or have suffered relapses.
Novartis Pharmaceuticals Corp, the company that has developed the treatment, says it is working to “change the course of cancer care.”
Cancer continues to be the bear of health burdens in the U.S., as 1.6 million new cases were diagnosed in 2016 alone. Kymriah is a form of gene therapy working to take a more innovative approach to anti-cancer therapy tactics.
Scientists are describing Kymriah as a “living drug” by because it is genetically modified and designed to attack cancer cells.
“We’re entering a new frontier in medical innovation with the ability to reprogram a patients own cells to attack a deadly cancer,” said FDA Commissioner Scott Gottlieb.
The FDA says Kymriah was shown to be safe and effective in multiple clinical trials; 63 pediatric and young adult patients included.
In addition to approving Kymriah to treat those with this type of bone marrow and blood cancer, the FDA has also approved a Risk Evaluation and Mitigation Strategy for the drug. The program will be made to educate healthcare professionals about the treatment and the possible risks associated.
“Kymriah is a first-of-its-kind treatment approach that fills an important unmet need for children and young adults with this serious disease,” said FDA Center for Biologics Evaluation and Research Director Peter Marks.
Although Novartis’ treatment could potentially improve how effective cancer fighting therapy is, as with any drug, there are possible side effects to attempting this type of therapy.
A list of possible side effects, and a more in depth look at how Kymriah is made and administered can be found here.
[NPR] [RT America] [Photo courtesy Stem Cell Next]