FDA approves gene therapy for leukemia patients


On Aug. 30, the U.S. Food and Drug Administration approved the first gene therapy for treating cancer, reported the New York Times. The drug, called Kymriah, uses a patient's own cells to fight against an advanced form of leukemia.

What is gene therapy?

Gene therapy is a new type of treatment, still very much in the experimental phase. Few physicians today have experience with gene therapy, as it is mostly limited to animal or clinical trials. As the FDA approves new forms of treatment and efficacy levels rise, gene therapy may become a standard replacement for medications with harsh side effects.

In general, gene therapy often involves inserting a new gene into a patient's cells, swapping one gene for another or repairing a damaged gene, according to the National Library of Medicine.

In the case of Kymriah, the treatment involves harvesting the patient's own white blood cells, freezing and shipping them to a laboratory where they are engineered to fight leukemia, then shipping them back. Finally, the modified blood cells are reintroduced to the patient's body where they serve as a "living drug" to fight against the deadly cancer.

B-cell acute lymphoblastic leukemia typically affects children and young adults.B-cell acute lymphoblastic leukemia typically affects children and young adults.

Who is the new treatment for?

Kymriah is designed specifically for children and young adults with b-cell acute lymphoblastic leukemia (ALL). According to the American Cancer Society, ALL spreads quickly and is typically fatal within a few months, if left untreated. Kymriah is for patients who have not responded to other forms of cancer treatment or those who have relapsed after a successful treatment.

"Not only does Kymriah provide these patients with a new treatment option where very limited options existed, but a treatment option that has shown promising remission and survival rates in clinical trials," said Dr. Peter Marks, director of the FDA's Center for Biologics Evaluation and Research.

Patients can only receive Kymriah once, and a single treatment costs $475,000. The New York Times reported that the drug's maker, Novartis, has promised to provide financial assistance to patients who cannot afford the treatment.

What are the potential side effects?

Altering a patient's genes can have serious side effects, and Kymriah is no exception. Potential side effects include cytokine release syndrome (CRS) - which involves high fever, nausea and neurological events - as well as severely low blood pressure, hypoxia and kidney damage. Therefore, the FDA requires that all clinics providing the drug must have specially trained staff on hand to deal with these life-threatening effects. Clinics must also have a supply of tocilizumab available to treat CRS.

Though Kymriah is the first approved gene therapy for cancer patients, many similar drugs could come to market in the near future. This type of treatment requires a network of certified hospitals and laboratories, so the cost could come down as more professionals receive training and additional labs come online.

For now, Kymriah provides greater treatment options for young leukemia patients, their families and their care providers.

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