FDA approves CAR T cell therapy for kids with leukemia

FDA has announced the approval of the first gene therapy treatment in the U.S. for children with cancer, called CAR T cell therapy. the St. Baldrick's Foundation (private funder of childhood cancer research grants) has contributed to the research and advocacy of this ground-breaking therapy.

Cancer takes more children's lives than any other disease in the U.S., and this revolutionary therapy will save the lives of many children who have had no other effective treatment.

CAR T cell therapy has been approved for treatment of children with acute lymphoblastic leukemia (ALL), and genetically modifies a patient's own cells in the lab to then infuse them back into the body to make his or her immune system attack leukemia cells.

St. Baldrick's has supported immunotherapy research since 2012, including the work of scientists in Dr. Stephan Grupp's lab at Children's Hospital of Philadelphia. Dr. Grupp made history when he treated the first child with CAR T cell therapy, Emily Whitehead, who is now more than five years cancer-free, after once being given a one-in-one-thousand chance of surviving.

"This is a landmark moment for cancer research and for pediatric oncology, as a whole new kind of cancer treatment, CAR T therapy, is approved," Dr. Grupp said. "It is amazing to me as a pediatric oncologist to see the first approval of a new treatment like this in pediatric leukemia."

While most new cancer treatments that come to market are first approved by the FDA for adults, the CAR T cell therapy is unique, as it was first approved for the specific treatment of children – making a huge stride in the childhood cancer research community.