19 February 2018

The results of a landmark international study that led to the approval of CAR T-cell therapy to treat paediatric acute lymphoblastic leukaemia (ALL) have been published for the first time.

The research published in The New England Journal of Medicine reports on 75 patients between three and 21 years of age with relapsed or treatment-refractory B-cell ALL. 61% of these patients had relapsed after allogeneic haematopoietic stem cell transplantation. 

Senior authors on the study include Stephan A. Grupp, of Children's Hospital of Philadelphia (CHOP) and Dr Michael Pulsipher, of Children's Hospital Los Angeles (CHLA).

"Stephan Grupp and his team at CHOP pioneered this therapy and were able to show it was effective when manufactured and administered at a single site," said Dr Pulsipher.

"For this study, we worked together with many colleagues to demonstrate that treatment with CAR T-cell therapy is not only feasible in a global, multisite setting but that it is also equally safe and effective."

Dr Pulsipher, who is also chair of the Pediatric Blood and Marrow Transplant Consortium, worked with Grupp and the paper's primary author, Shannon Maude from CHOP, as well as researchers from the US, Canada, Europe, Japan, and Australia to run the trial at 25 sites in 11 countries. The therapy was approved by the FDA in August 2017 to treat paediatric ALL.

"Previously, for patients who relapsed after transplant or did not respond to treatment - there wasn't much else we had to offer them and often, they went to hospice," he said.

“Now, instead of sending them to [a] hospice we treat them with CAR T-cells, make them better, then send them home.”

The updated analysis showed an overall remission rate of 81% within three months of treatment. All patients who responded to therapy showed no minimal residual disease and overall survival was 90% at six months and 76% at 12 months.

The research showed that a single transfusion of the therapy, tisagenlecleucel, stayed in the blood for up to 20 months, and provided persistent remission in the children and young adults tested.

"CAR T therapy is truly a game-changer for paediatric leukaemia," added Dr Alan S. Wayne, director of the Children's Center for Cancer and Blood Diseases at CHLA.

Source: Maude, Shannon L., et al. "Tisagenlecleucel in Children and Young Adults with B-Cell Lymphoblastic Leukemia." New England Journal of Medicine 378.5 (2018): 439-448.

Link: http://www.nejm.org/doi/full/10.1056/NEJMoa1709866


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