The U.S. Food and Drugs Administration (FDA) has approved CAR T cell therapy for treatment of children with acute lymphoblastic leukemia (ALL).

"This is a landmark moment for cancer research and for paediatric oncology, as a whole new kind of cancer treatment, CAR T therapy, is approved," said Dr Stephan Grupp said at Children’s Hospital of Philadelphia. "It is amazing to me as a paediatric oncologist to see the first approval of a new treatment like this in paediatric leukaemia."

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 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 laboured to realise 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 success. Eighty-three percent of children and young adults whose leukaemia could not be cured by any other means, went into remission within three months of being treated with CAR T cell therapy.

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.