Gene-Edited super cells take on childhood leukemia

NCT ID NCT05942599

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This early-stage trial tests a new treatment for children aged 6 months to 16 years whose acute myeloid leukemia has returned. The therapy uses donor immune cells that have been gene-edited with a technique called base editing to better target and kill leukemia cells. The main goal is to check safety and see if the cells can clear the cancer before a bone marrow transplant.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance

Base-edited CAR T cells (BE CAR-33) made from donor white blood cells

What this could lead to

If it works, this could offer a way to clear leukemia before a bone marrow transplant, potentially preventing the cancer from coming back.

What could go wrong

This is a very early, small phase 1 trial with only 10 children. The therapy may cause severe side effects like cytokine release syndrome or graft-versus-host disease, and it may not work for everyone.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Conditions

The condition(s) this trial relates to.

acute myeloid leukemia

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • Great Ormond Street Hospital for Children

    London, WC1N3JH, United Kingdom