Childhood Leukemia Will Finally Have An FDA Approved Treatment

Childhood Leukemia Will Finally Have An FDA Approved Treatment
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The U.S health officials have approved a treatment which genetically turns the patient’s blood cells into assassins seeking to destroy leukemia. The Food and Drug Administration (FDA) has qualified the treatment as historic since it is the first gene therapy in the U.S market.

The treatment will be available for purchasing

The treatment is personalized for every patient and Novartis Pharmaceuticals is planning to charge 475,000$ for it. They call the one-time infusion CAR-T cells and they will not charge the patient if he/she does not show signs of improvement within a month.

Dr. Stephan Grupp from Children’s Hospital in Philadelphia has already treated the first child with the CAR-T cell therapy. The patient was a little girl almost dying and is now five years cancer free.

How the treatment works

The CAR-T therapy uses gene therapy by turbo charging T cells (soldiers of the immune system) and resetting them to fight the disease. The T cells grow hundreds of millions of copies and fight back leukemia.

The FDA Commissioner, Scott Gottlieb has declared that the discovery signifies a new frontier in medical innovation. It is extremely pleasing that the patient’s own cells can be reprogrammed to destroy cancer.

According to a study on CAR-T cells on 63 patients, 83% went in remission after receiving the therapy. Since the discovery is recent, it is still unclear if the patient’s recovery lasts forever or for a determined period of time. More than 3.000 children are diagnosed with leukemia each year in the U.S and out of them, 155 relapse despite being given multiple treatments.

The therapy does have side effect for which the FDA has created a treatment. One of the side effects includes the over action of the immune system (cytokine release syndrome).


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