Largest CAR-T trial for pediatric solid tumors opens in the US and UK
The largest CAR-T trial for pediatric solid tumors has opened in the US and UK, enrolling up to 60 young patients across four sites. The NexTGen study targets relapsed or refractory cancers and focuses first on safety and feasibility.
The world’s largest clinical trial of CAR-T cell therapy for pediatric solid tumors has opened in the United States and the United Kingdom, enrolling up to 60 children and young adults across four sites. The study will test a new generation of CAR-T cell immunotherapies for children and young people with relapsed or refractory solid tumors, and the first two patients, including one in their early 20s, have already begun treatment.
The international NexTGen team, funded by Cancer Grand Challenges, has launched the trial across Children’s National Hospital in Washington, Dana-Farber Cancer Institute and Boston Children’s Hospital, and the UCL Cancer Institute at Great Ormond Street in the UK. The clinical trial is structured as a multi-trial with three parallel arms.
Children diagnosed with solid tumors such as rhabdomyosarcoma, Ewing sarcoma, and desmoplastic small round cell tumor have seen survival rates that have barely budged in the past 30 years, while treatments originally designed for adults can leave survivors with long-term side effects such as learning difficulties and infertility. The trial is open to children and young people whose cancer has returned or failed to respond to standard treatments.
CAR-T cell therapy reprograms a patient’s own T cells to hunt down and destroy cancer cells and has already been used in pediatric leukemia and lymphoma. Solid tumors have been more resistant because of physical and biological barriers that limit immune cell infiltration and allow cancer cells to escape detection.
The NexTGen approach targets two different cancer markers at once and uses two separate T-cell therapy platforms. Safety and feasibility are the primary focus at this early stage, with the research aimed at developing treatments that are less toxic than current standards of care.