With the FDA approval of the first gene therapy, or a nucleic acid drug, we have entered a new era for fighting diseases. Kymriah was approved on August 30th to treat B-cell acute lymphoblastic leukemia (ALL) in patients 25 years old and younger which have relapsed or not responded to initial treatment. ALL is the most common pediatric cancer in the US, and close to 20% of patients do not respond to initial treatment or relapse. Kymriah is a type of chimeric antigen receptor T-cell (CAR-T) gene therapy personalized to treat each patient’s cancer cells. After collection, the patient’s T-cells are modified to include a new chimeric protein coding for a specific antigen receptor targeting CD19. The CAR-T cells are then infused back into the patient to target and kill the cancerous cells with CD19 on the surface. Normal B cells with CD19 on the surface are also killed in the process, potentially increasing the risk for infections. Other side effects include cytokine release syndrome (CRS), which causes flu-like symptoms and can be life-threatening. Since side-effects can be severe, hospitals and other medical institutions that use Kymriah must be FDA-certified.
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