FDA Approves First ‘Living Drug’ for Childhood Leukemia

Announced on Wednesday, the Food and Drug Administration approved the first treatment that genetically engineers patients’ own blood cells that will destroy childhood leukemia. This is the first type of gene therapy to hit the U.S. market called the CAR-T cell treatment that was developed by Novartis Pharmaceuticals and the University of Pennsylvania. The first child that was treated with this therapy was near death but is now cancer free for five years and counting.

The treatment is made from scratch for every patient and costs $475,000 but would be no charge if the patient didn’t show a response within a month. It would use gene therapy techniques to charge T cells where researchers can filter those cells from a patient’s blood and reprogram it to cancel cancer and grow to millions of copies. The new cells would then continue to multiply to fight disease for months or years.

The CAR-T treatment is aimed to treat patients desperately ill with common pediatric cancer. It kills more than 3,000 children and young adults in the U.S. each year and 15% relapse from those who survived despite today’s best treatments.

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