By, Rodin Batcheller
Addendum Editor
On Aug. 30, the U.S. Food and Drug Administration (FDA) approved a new type of gene therapy that will fight an aggressive form of leukemia in young patients, giving them hope of pushing through the disease. The new drug is neither a pill nor an injection, but rather a “living drug” that will be marketed under the trade name Kymriah. FDA Commissioner Scott Gottlieb believed that the chimeric antigen receptor T-cell (CAR-T) therapy will revolutionize modern medicine and leukemia treatments. The two-stage process starts by harvesting a cancer patient’s T-cells.
“Therapies hold out the potential to transform medicine and create an inflection point in our ability to treat and even cure many intractable illnesses,” stated Gottlieb in a Los Angeles Times interview.
The cells are delivered to a specialized lab where scientists alter their DNA, essentially reprogramming them to target cancer cells. The new and improved cells are copied millions of times before
they are sent back to the patient. Once infused into the bloodstream, the strengthened T-cells are much better equipped to hunt down and kill cancer cells. In the past, CAR-T therapy was restricted to testing in small clinical trials for patients with advanced blood cancers. Due to the promising signs of the therapy, these treatments have since captured the attention of researchers and the public. Novartis, the company that developed the treatment, intends to. have 32 certified treatment centers up and running by the end of 2018. Patients up to the age of 25 would go to one of these centers to have their T-cells harvested and later reintroduced into their system in their modified form.
In a press release, Novartis did not disclose how much it would charge for the new therapy. It did, however, state that it was constructing a plan to make sure patients could still receive the treatment even if they could not afford it. According to Consumer Reports, experts predicted that the therapy will cost approximately $475,000, which was met with concerns from both analysts and potential patients alike because of its high cost. Acute lymphoblastic leukemia is the most common form of pediatric cancer, affecting about 3,000 children and young adults annually in the United States. Though it is considered highlycurable in most patients, about 600 each year either do not respond to chemotherapy or see their leukemia return after an initial round of successful treatment.
