FDA Approves Groundbreaking CAR T Cell Therapy for Kids With Leukemia

LOS ANGELES, Aug. 31, 2017 /PRNewswire-USNewswire/ -- As the largest private funder of childhood cancer research grants, the St. Baldrick's Foundation is proud to have contributed to the research and advocacy that led to the FDA approving the first gene therapy treatment in the U.S. for children with cancer today, called CAR T cell 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."

Dr. Grupp is also a member of the SU2C-St. Baldrick's Pediatric Cancer Dream Team (PCDT), which has been instrumental in the development of this new approach by committing millions of dollars to the research of CAR T cell therapy. The Dream Team has been key to helping understand why the treatment works, and helping to push the drug approval over the finish line as quickly as possible.

"This is a remarkable achievement and a hallmark day for our community and our children most importantly," said Kathleen Ruddy, CEO of the St. Baldrick's Foundation.  "We are so grateful for the many scientists who have labored to realize this day, and for every donor and volunteer who has helped to make this possible.  We will continue to support the best childhood cancer research, wherever it takes place, to make progress like this available to patients with all types of childhood cancers."

Clinical trial results have already shown this gene therapy's stunning success. Eighty-three percent of children and young adults whose leukemia could not be cured by any other means, went into remission within three months of being treated with CAR T cell therapy. It has already saved the lives of many children, including 2016 St. Baldrick's Ambassador Phineas Sandi.

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. 

Worldwide a child is diagnosed with cancer every two minutes. By funding research, you are giving kids with cancer hope for a cure. In recognition of Childhood Cancer Awareness Month this September, help St. Baldrick's take childhood back from cancer by donating at StBaldricks.org/Give.

About St. Baldrick's Foundation
As the largest private funder of childhood cancer research grants, the St. Baldrick's Foundation believes that kids are special and deserve to be treated that way. St. Baldrick's is leading the charge to take childhood back from cancer by funding some of the most brilliant childhood cancer research experts who are working to find cures and better treatments for all childhood cancers. Kids need treatments as unique as they are – and that starts with funding research just for them. Join us at StBaldricks.org to help support the best cancer treatments for kids.

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SOURCE St. Baldrick's Foundation