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FDA Recently Gave Approval to First Gene Therapy in The U.S.

By jeremyc | September 7, 2017

On Aug 31st the FDA gave approval to Kymriah for treating certain patients, pediatric as well as young adults, suffering from a type of acute lymphoblastic leukemia. This is a landmark decision because now gene therapy is available in America for the first time. Cancer patients and people with other serious conditions will now have an additional treatment option.

Kymriah is indicated for use in patients who are 25 years of age or less and are suffering from refractory or second-stage or a relapse of B-cell precursor acute lymphoblastic leukemia. Kymriah is a cell-based gene therapy.

Kymriah’s every dose is a tailor-made treatment made by taking the patient’s T-cells, also known as lymphocyte. The collected T-cells of a patient are modified genetically so that they include an additional gene which has a particular protein that instigates T-cells to liquidate leukemia cells having a particular antigen on surface. After T-cells are altered, they are injected into the patient so that they can terminate cancer cells.

In acute lymphoblastic leukemia, lymphocytes are produced in abnormal quantities. This cancer progresses rapidly and occurs more commonly than any other childhood cancer.

It is estimated that every year about 3,100 children and young adults under 20 years of age are diagnosed with acute lymphoblastic leukemia, which can be either B-cell or T-cell. B-cell is more common and Kymriah is approved for those young adult and pediatric patients whose cancer didn’t respond to or has come back following initial treatment. According to experts, this happens in about 15%-20% of patients.

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