New CAR-T Cell Trial for Children With Solid Tumors Opens in US and UK

A new clinical trial is enrolling up to 60 children and young people with solid tumors across the United States and United Kingdom to test a personalized CAR-T cell immunotherapy designed to help the immune system recognize and attack cancer cells.

Led by the international NexTGen team and funded by Cancer Grand Challenges — a partnership between Cancer Research UK and the National Cancer Institute — this study, according to a news release, represents the world’s largest effort to date testing this specific type of therapy for pediatric solid tumors. The trial seeks to find more effective treatments that reduce the long-term side effects often caused by traditional care.

Researchers involved in the NexTGen project report that while CAR-T cell therapies have changed the landscape for pediatric leukemia and lymphoma, the same level of success has not yet been achieved for patients with solid tumors. Solid tumors grow in organs or tissues and form masses, creating complex defenses that make it difficult for the immune system to penetrate.

Current data shows that survival rates for children with these types of tumors have seen very little improvement over the last 30 years. Many existing treatment options were originally designed for adults decades ago and are associated with severe long-term health effects, including learning difficulties and infertility.

The NexTGen team has identified new cancer targets and developed novel therapies intended to break down the barriers surrounding solid tumors. By using a "first in human and child" approach, the study targets two different cancer markers simultaneously. This method combines two separate T-cell therapy platforms to help the modified cells infiltrate the tumor and destroy cancer cells more effectively than previous iterations of immunotherapy.

Trial details

The clinical trial is structured as a "multi-trial" consisting of three parallel arms operating across the UK and the US. The first two patients have already been enrolled, including one individual in their early 20s. The study is open to children and young people, including those in early adulthood, who have specific types of difficult-to-treat solid tumors. These include rhabdomyosarcoma, Ewing sarcoma and desmoplastic small round cell tumor.

In the United States, the trial is being conducted at two primary locations. One arm is headed by Dr. Holly Meany and Dr. Catherine Bollard at Children’s National Hospital in Washington. The other US arm is led by a team at Dana-Farber Cancer Institute and Boston Children’s Hospital. In the UK, the trial is led from the UCL Cancer Institute at Great Ormond Street, in partnership with University College London Hospitals.

This international collaboration is the first clinical trial funded by Cancer Grand Challenges, an initiative co-founded in 2020 by the National Cancer Institute (NCI) in the US and Cancer Research UK. The project is also co-funded by The Mark Foundation for Cancer Research. The team includes experts from the UK, US and France who are working to move these investigational therapies from the lab to the clinic at an accelerated pace. Patients and families interested in the study are encouraged to speak with their doctors to determine eligibility at one of the four participating sites.

Safety

The primary focus of this early-phase clinical trial is to evaluate the safety and feasibility of the new CAR-T cell platforms in children and young people. Because these patients often have relapsed or refractory solid tumors — meaning the cancer has returned or stayed the same after standard treatments — the study is designed for those who have exhausted other available options.

A major goal of the research is to develop treatments that are less toxic than the current standards of care. By reprogramming a patient’s own T cells in a laboratory to specifically recognize cancer cells, researchers hope to create a more precise treatment that spares healthy tissue. This approach aims to mitigate the harsh, long-term side effects frequently seen with older treatments.

Editor's note: This article is for informational purposes only and is not a substitute for professional medical advice, as your own experience will be unique. Use this article to guide discussions with your oncologist. Content was generated with AI, reviewed by a human editor, but not independently verified by a medical professional.

Reference:

“First Patients Enrolled in First-of-Its-Kind International Trial Harnessing the Immune System Against Childhood Cancers,” news release.

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