Health Care

FDA approves gene-therapy leukemia treatment

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The Food and Drug Administration (FDA) on Wednesday approved a gene-therapy treatment that has the potential to aid young patients suffering from an aggressive form of leukemia.

The treatment, called Kymriah, works to strengthen the immune system by genetically altering an individual’s cells, which helps the immune system fight acute lymphoblastic leukemia. 


The disease is the most common type of childhood cancer in the U.S., making up 25 percent of cancer diagnoses for children under the age of 15, according the National Cancer Institute.

An FDA panel in July voted unanimously to recommend the therapy. 


Kymriah, which is also known as CTL019, is a type of CART-T therapy that involves immune cells being extracted and genetically modified to destroy cancer before it returns to the body. 

“We’re entering a new frontier in medical innovation with the ability to reprogram a patient’s own cells to attack a deadly cancer,” FDA Commissioner Scott Gottlieb said in a statement on Wednesday. 

“New technologies such as gene and cell therapies hold out the potential to transform medicine and create an inflection point in our ability to treat and even cure many intractable illnesses. At the FDA, we’re committed to helping expedite the development and review of groundbreaking treatments that have the potential to be life-saving,” he said. 

Swiss drug company Novartis AG developed the treatment, which is the first CART-T therapy to be approved by the FDA. 

The company announced it would be collaborating with the Centers for Medicare and Medicaid Services (CMS) on CART-T therapy.

CMS administrator Seema Verma congratulated scientists and researchers involved in developing the leukemia treatment. 

“Innovations like this reinforce our belief that current healthcare payment systems need to be modernized in order to ensure access to new high-cost therapies, including therapies that have the potential to cure the sickest patients. Improving payment arrangements is a critical step towards fulfilling President Trump’s promise to lower the cost of drugs,” she said in a statement.
Updated: 3:21 p.m.

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