New Treatments30 September 2026

CAR-T began with children: the paediatric origins of cell therapy

The first CAR-T therapy approved anywhere was for children and young adults with leukaemia. That history still shapes how the field thinks about paediatric patients.

By NextGenHealth Editorial Team2 min read

Purple-stained cells under magnification — Photo: National Cancer Institute / Unsplash

In brief

The first approved CAR-T therapy, tisagenlecleucel, treated children and young adults with leukaemia. How paediatric cases shaped cell therapy.

When the FDA approved tisagenlecleucel in August 2017, it was the first chimeric antigen receptor T-cell (CAR-T) therapy approved anywhere in the world. Its indication was striking: patients up to 25 years old with B-cell acute lymphoblastic leukaemia (ALL) that had not responded to treatment or had relapsed at least twice.

How it works

CAR-T therapy takes a patient’s own T cells, genetically modifies them to recognise a protein on cancer cells, in this case CD19 on B cells, expands them in the laboratory and infuses them back. The modified cells then multiply in the body and attack the leukaemia.

A paediatric story from the start

Childhood ALL is the most common childhood cancer, and most children are cured with chemotherapy. But for the minority who relapse repeatedly, outcomes had been poor. That combination of a well-understood target, a group with few options and an academic centre willing to push the science made relapsed paediatric ALL a natural first indication.

One of the earliest patients was a six-year-old treated at the Children’s Hospital of Philadelphia in 2012, whose recovery after a severe reaction became one of the defining stories of the field.

The toxicity problem

That reaction was cytokine release syndrome, a surge of immune activation that can cause high fever, low blood pressure and organ dysfunction. Clinicians found that tocilizumab, an antibody that blocks the cytokine interleukin-6, could control it, and the FDA approved tocilizumab for CAR-T-related cytokine release syndrome on the same day it approved tisagenlecleucel. Neurological toxicity is the other major risk, and managing both requires specialist centres.

Where paediatric cell therapy goes next

Most of the CAR-T products approved since have been for adult blood cancers. Researchers are working on targets for paediatric solid tumours and brain tumours, which have proved much harder. Access is the other challenge: manufacturing is complex, prices are high, and only a limited number of centres can deliver the therapy.

CAR-T
Cell therapy
Leukaemia
Oncology

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