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FDA Votes In Favor Of Gene Therapy To Treat Childhood Leukemia

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A new treatment for leukemia could soon become the first gene therapy for cancer in the USA, after a Food and Drug Administration panel voted in its favor.

Developed by the University of Pennsylvania and Novartis Corp., the therapy could be the first of its kind – an innovative treatment custom-made to target specific forms of cancer, USA Today reports. Called CAR-T, this treatment procedure involves taking out immune cells from a cancer patient’s blood then “reprogramming” them to produce cells that can hone in and destroy cancer cells. These are then injected back into the patient.

The FDA voted 10-0 in favor of allowing the therapy to proceed. It came after a long discussion that included please from the fathers of two leukemia patients whose lives were saved because of CAR-T.

Dr. Malcolm A. Smith of the National Cancer Institute, one of the panel members, said,

This is a major advance.

He said the treatment is “ushering in a new era.”

This therapy is a one-time treatment for children and young adults affected by the most common form of childhood cancer, acute lymphoblastic leukemia or ALL. Novartis is seeking approval to use the therapy on patients ages three to 25, particularly in patients who have failed to respond to standard treatments – something that occurs in over 600 people in the US yearly. ALL makes up around 25% of all cancers in children under 15 years old.

In a test conducted, gene therapy results exceeded chemotherapy, and even newer types of cancer medicines. Of the 52 patients examined, 83% had complete remission, although most of them suffered serious side effects. Eleven patients died – four from side effects and seven from the cancer. The whole testing process took 16 weeks on average, which FDA advisers noted could be too long for severely ill patients.

Novartis said that it has believed that CAR-T could “change the cancer treatment paradigm.” Dr. Carl June, leader of the Penn research team, said, “It is encouraging to see the FDA panel’s recommendation and continued momentum behind this innovative therapy.”

The FDA is expected to make a decision on approving Novartis’ treatment within the next few months.

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