University of Pennsylvania

FDA approves country's first gene therapy

FDA approves country's first gene therapy

The Food and Drug Administration on Wednesday approved a ground-breaking gene therapy treatment in which a patient’s blood cells are genetically engineered to attack and kill cancer cells.

Johns Hopkins Hospital will be one of 32 hospitals whose doctors will be specially trained to administer the procedure, an official with the hospital said.

The treatment was approved for children and young adults up to age 25 who suffer from acute lymphoblastic leukemia and for whom the standard treatment is ineffective, which the FDA said is about 15 percent to 20 percent of patients.

The disease, the most common childhood cancer in the United States, is a cancer of the bone marrow...

68°