Reprogramming Cells to Fight Leukemia

The FDA may soon approve a new cancer therapy that genetically alters a patient’s own existing T-cells to fight leukemia. This new, investigational treatment is known as CTL019 and is a type of chimeric antigen receptor T-cell (CAR-T) therapy.

CTL019 utilizes a process in which T-cells are carefully harvested from each individual leukemia patient. These patient-specific T-cells are then genetically reprogrammed to express a chimeric CD19 antigen receptor and subsequently transfused back into the specific patient from whom they were originally collected. Once back inside the patient, these reprogrammed T-cells multiply, hunt down, and attack CD19-positive leukemia cells.

Click here to read about this FDA update in the NY Times.

A Food and Drug Administration panel opened a new era in medicine on Wednesday, unanimously recommending that the agency approve the first-ever treatment that genetically alters a patient’s own cells to fight cancer, transforming them into what scientists call “a living drug” that powerfully bolsters the immune system to shut down the disease.

If the F.D.A. accepts the recommendation, which is likely, the treatment will be the first gene therapy ever to reach the market in the United States. Others are expected: Researchers and drug companies have been engaged in intense competition for decades to reach this milestone. Novartis is now poised to be the first. Its treatment is for a type of leukemia, and it is working on similar types of treatments in hundreds of patients for another form of the disease, as well as multiple myeloma and an aggressive brain tumor.

To use the technique, a separate treatment must be created for each patient — their cells removed at an approved medical center, frozen, shipped to a Novartis plant for thawing and processing, frozen again and shipped back to the treatment center.

For more reading on the history of leukemia, read about Gertrude Belle Elion’s, and her influence on medicine. A biochemist and pharmacologist, her work paved the way for breakthroughs in cancer and leukemia medication that would save thousands of lives.

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