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Briefing
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- First FDA Approved Gene Therapy – U.S. Food and Drug Administration approves first gene therapy in U.S., called Kymriah, designed by Novartis to cure acute lymphoblastic leukemia (ALL) among patients up to 25 years of age
- How It Works – Sample of patient’s T-cells are taken and modified to include new gene that contains protein called chimeric antigen receptor or CAR, making T-cells induced to target and kill leukemia cells with antigen CD19 on surface
- Test Results – In clinical trials, 83% of patients that received gene therapy had no presence of cancer, with 89% still living after six months, and 79% surviving within 12 months
- Cost – Costs $475,000, but charge is waived if patient does not respond to treatment within one month
- Side Effects – Include cytokine release syndrome (CRS), which causes high fever and flu-like symptoms, serious infections, low blood pressure (hypotension), acute kidney injury, fever, and decreased oxygen (hypoxia)
- Acute Lymphoblastic Leukemia Risk – 3,100 patients aged 20 and younger are diagnosed with ALL every year, making it most common childhood cancer
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