The Food and Drug Administration has approved the very first treatment that genetically engineers blood cells to destroy leukemia in child patients. The treatment opens up a new era in cancer care, which means a great deal to Alabama. According to Children’s of Alabama, more than 190 children in Alabama are diagnosed with various forms of cancer each year, including ALL.
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According to The Associated Press, The CAR-T cell treatment was developed by Novartis Pharmaceuticals and the University of Pennsylvania. It is the first gene therapy of its kind to hit the U.S. market. The treatment reprograms a patient’s T cells to carry a “chimeric antigen receptor” that targets cancer cells. Once hundreds of copies are grown, the cells are returned to the patient and can fight the disease for months or even years.
The treatment is a dramatic shift from traditional immunotherapy drugs. Instead of helping the body’s T cells better spot cancer, the CAR-T treatment enables them to fight those cancer cells better. This particular treatment is being aimed to treat patients with severe pediatric cancer, or acute lymphoblastic leukemia (ALL). ALL strikes 3,000 children in the United States each year.
According to the study cited by the FDA, 83 percent of the 63 patients went into remission. However, there were some severe side effects observed in study. In some cases, the immune system can trigger an overreaction that causes plummeting blood pressure, high fevers, and organ damage. The FDA also released a treatment for these side effects on Wednesday.
Researchers acknowledge that there are downsides to the treatment, however they are optimistic about what these advancements will mean for the future. “I wouldn’t say we know for sure how many will be cured yet by this therapy. There certainly is a hope that some will be,” said Dr. Ted Laetsch of the University of Texas Southwestern Medical Center. Researchers hope that this will lead to further CAR-T treatments that can fight more complicated cancers such as pancreatic, breast, and brain cancer.
Novartis said that it will sell the treatment under the name Kymriah at the price of $475,000 per treatment. However, the company says that there would be no charge if the patient did not show a response within a month.
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