CAR-T: Obstacle Race
Clinical trials of gene therapy suspended after patient's death
Julia Korowski, XX2 century
The Food and Drug Administration (FDA) has suspended two clinical trials of the innovative gene therapy CAR-T due to the death of a patient. This was announced on September 4 by Cellectis, the company responsible for conducting research. Nevertheless, it's too early to give up on CAR-T: last week, the FDA approved the therapy of Novartis Pharmaceutical Corporation.
CAR-T therapy works differently than traditional cancer treatments: a sample of T-lymphocytes is taken from a patient, and with the help of genetic engineering they are modified so that they effectively destroy tumors. Then they are multiplied and injected back into the patient. This method has serious drawbacks: firstly, the medicine is actually made anew for each patient, so it is very expensive, and secondly, it is impossible to get the necessary cells from some patients. The French company Cellectis tried to solve this problem and create a "universal" therapy: instead of patient cells, scientists used donor cells. If this type of treatment turned out to be safe and effective, it would be much cheaper, and the manufacture of the drug would take less time.
Cellectis conducted two clinical trials: acute myelocytic leukemia and neoplasm from blast plasmocytoid dendritic cells (OBPDC) were treated with CAR-T. After the drug was administered to a 78-year-old man with OBPDK, his immune system "reared up." The reaction was suppressed, but on the eighth day it returned, and the patient began to suffer from capillary leakage syndrome, and despite the efforts of doctors, it was not possible to save him. After that, the FDA suspended both trials – now Cellectis employees, together with the regulator, are trying to find out the causes of the incident and make the necessary changes to the treatment protocol.
This is not the first case of death as a result of CAR-T therapy. Last year, the company Juno Therapeutics has stopped clinical trials due to the death of seven patients. Kite Pharma also faced the death of a patient during the tests, but the FDA representatives stated that the patient was extremely severe. At the moment, Kite is awaiting approval for registration of the drug. Novartis wins the race – last week the regulator approved the company's gene therapy for the treatment of acute lymphoblastic leukemia. A course of the drug called Kymriah will cost $475,000.
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