A Food and Drug Administration panel has approved the first-ever gene therapy to treat Leukemia. The immunotherapy works by genetically alters a patient’s own cells to fight cancer and bolsters the immune system to shut down the disease.
A panel of cancer experts unanimously endorsed the leukemia treatment on Wednesday in a 10-0 vote, according to AP. The FDA is not required to follow the panel’s recommendation, but often does.
“This is a major advance,” said panel member Dr. Malcolm A. Smith of the National Cancer Institute. He added that the treatment is “ushering in a new era.”
Called CART-T, and devised by the University of Pennsylvania and Novartis Corp, it works by creating a separate treatment for each patient.
Millions T-cells (a type of immune cell) are from removed the patient’s blood. The technique employs a disabled form of HIV, the virus that causes AIDS, to carry new genetic material into the T-cells to reprogram them. The process turbocharges the T-cells to attack B-cells, a normal part of the immune system that turn malignant in leukemia. The T-cells hone in on a protein called CD-19 that is found on the surface of most B-cells.
Dr. Carl H. June, a leader of the University of Pennsylvania team that developed the treatment, called the turbocharged cells “serial killers.”
A single one can destroy up to 100,000 cancer cells, the New York Times reported.
The treatment destroys not only leukemic B-cells but also healthy ones, which help fight germs, patients then need treatment to protect them from infection. Every few months they receive infusions of immune globulins.
The one-time leukemia treatment, which will cost in the hundreds of thousands of dollars, would be for children and young adults with the most common form of childhood cancer, B-cell acute lymphoblastic leukemia, and who are facing death because other treatments have failed.