FDA APPROVES FIRST GENE THERAPY TO GO TO MARKET, TREAT CHILDHOOD LEUKEMIA
This week, a new treatment that involves the genetic modification of a patient’s own blood cells was approved by the Food and Drug Administration. Children battling childhood leukemia will now have a new treatment, programming the patients’ own cells to attack the leukemia itself at the cellular level. This approval opens a new frontier for R&D and begins a new era of FDA-sanctioned human gene modification.
CAR-T REPROGRAMS KIDS’ WHITE BLOOD CELLS, TURNS THEM INTO CANCER ASSASSINS, INJECT THEM BACK IN TO GET THE JOB DONE
This new treatment has been developed and brought to market by the drug company Novartis. It’s called CAR-T and involves the harvesting of a patient’s white blood cells which are then reprogrammed into literal tumor assassins in a lab. These tumor assassins are then put back into the patients to do just that, where they track down and destroy cancerous cells. The related drug from Novartis, called Kymriah, is approved to treat both children and young adults who have suffered relapses after initial treatment for acute lymphoblastic leukemia, a very rare and often deadly blood cancer.
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SOME DISAGREE CAR-T SHOULD BE CONSIDERED FIRST APPROVED GENE THERAPY
It’s worth noting that while the FDA has called this the first approved “gene therapy,” there has been some disagreement among experts as to whether that term applies.
DRUG COMPANY NOVARTIS WINS RACE FOR FIRST APPROVED GENE THERAPY PRODUCT, CAR-T
The drug was one of several CAR-T therapies rushing to be the first to market. In a key clinical trial of 63 people, 83 percent of participants were cancer-free after three months. (Juno, another company developing a CAR-T therapy, was forced to halt its trial earlier this year after patient deaths.)
Some of those patients, though, did eventually relapse after several months. And the drug comes with serious side-effects, including a reaction known as cytokine release syndrome, that can prove fatal if not controlled with immunosuppressant drugs.
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Novartis did not disclose how much it intends to charge for the game-changing drug, but analysts have projected it will likely cost hundreds of thousands of dollars for a course of treatment.
On Twitter, FDA commissioner Scott Gottlieb called the approval historic.
“Today is an important milestone in a long journey to transform clinical medicine using modern advances in genomics,” he said.