Stand Up To Cancer, St. Baldrick's Foundation hail green light for CAR T cell therapy

Stand Up To Cancer and the St. Baldrick's Foundation welcomed the Oncology Drugs Advisory Committee's (ODAC) strong recommendation for approval by the Food and Drug Administration (FDA) of a revolutionary new therapy for a deadly form of leukemia, an approach whose development has also been supported by SU2C and St. Baldrick's.

"When fully commercialized, this therapy will no doubt save the lives of many children and young adults who have had no other effective therapy for relapsed and refractory B-cell acute lymphoblastic leukemia (ALL)," said John Maris, MD, a pediatric oncologist at The Children's Hospital of Philadelphia who holds the Giulio D'Angio Chair in Neuroblastoma Research, and leader of the SU2C-St. Baldrick's Pediatric Cancer Dream Team. "This is truly a turning point in the management of this disease."

"SU2C and the St. Baldrick's Foundation have committed millions of dollars to the development of the chimeric antigen receptor (CAR) T cell therapy," said Sung Poblete, PhD, RN, president and CEO of SU2C. "The Dream Team has especially investigated why some patients with B-cell ALL relapse after receiving the CD19 CAR T cell therapy, and how the problem of tumor cells escaping the therapy can be overcome," she said.

"The team also played a major role in developing standardized management of cytokine release syndrome (CRS), a potentially fatal side effect of the treatment," added Lee J. Helman, MD, director of the Cancer Research Program at the Saban Institute at Children's Hospital of Los Angeles and a member of SU2C's Scientific Advisory Committee.

"We are very proud that we are playing a key role in the overall development of CAR T cell therapy, and hope that the treatment will soon be available to children and young adults around the world," said Crystal L. Mackall, MD, professor of pediatrics and internal medicine at Stanford University, associate director of Stanford Cancer Institute, and co-leader of the Dream Team.

"The St. Baldrick's Foundation has been an early supporter of bringing new immunotherapy approaches to childhood cancer, and this Dream Team is the largest of these grants," said Chief Mission Officer Becky Chapman Weaver. "Children are alive today because of this crucial research."

The Oncology Drugs Advisory Committee (ODAC) voted 10-0 Wednesday to recommend that FDA approve a biologics license application (BLA) from the pharmaceutical company Novartis for tisagenlecleucel (CTL019) for the treatment of patients aged 3 to 25 years old with relapsed/refractory B-cell acute lymphoblastic leukemia. The FDA will take up the application in the near future.

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