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New Gene Therapy For Childhood Leukemia Gets Approval

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Health officials in the United States have approved a revolutionary treatment that uses genetic engineering to combat childhood leukemia.

This breakthrough allows scientists to manipulate a patient’s blood cells, turning them into an army that can seek and kill cancer cells. The Food and Drug Administration called the approval historic, this being the first gene therapy on the American market, ABC News reports. It’s one of many “living drugs” that scientists are developing in order to fight certain diseases.

Novartis Pharmaceuticals, the company behind the treatment, is setting the price for this one-time treatment using “CAR-T cells” at $475,000. The hefty tag comes with a guarantee: patients who don’t show any response within a month don’t have to pay. Novartis came up with the gene therapy with the University of Pennsylvania.

Dr. Stephan Grupp of Children’s Hospital of Philadelphia, lead researcher on the therapy, treated the first child with the new therapy. The patient was nearing death, but has now been cancer-free for five years. Grupp said,

This is a brand new way of treating cancer. That’s enormously exciting.

CAR-T treatment is different from the popular immunotherapy drugs that treat cancers by helping the body to spot tumors easily. This gene treatment boosts T cells – the immune system’s soldiers – by reprogramming them to carry a “chimeric antigen receptor” or CAR. This allows the cells to replicate quickly and zero in on cancer cells, fighting the disease for years.

FDA Commissioner Scott Gottlieb said, “We’re entering a new frontier in medical innovation with the ability to reprogram a patient’s own cells to attack a deadly cancer.”

This first version of CAR-T is approved for patients who are suffering from acute lymphoblastic leukemia, or ALL. This particular form of cancer affects over 3,000 children and young adults in the USA yearly. Although it has a high survival rate, around 15% of patients relapse.

To make sure patient safety is the priority, the FDA is requiring Novartis to offer the treatment only through medical centers that have been specially trained and certified. The drug company expects to open some 32 centers by the end of this year. Executives likewise said that Novartis is working with Medicaid and other insurance providers to hash out coverage for patients.

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