Engineered immune cells take on childhood brain cancer in first human trial

NCT ID NCT04903080

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This early-stage trial tests a new treatment for children whose ependymoma brain tumor has come back or not responded to standard therapy. The treatment uses the patient's own immune cells, modified in a lab to recognize and attack tumor cells that carry a protein called HER2. The main goals are to check if the treatment is safe and if it can be delivered across multiple hospitals.

What this could mean

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

Active substance

HER2 CAR T cells (a type of immune cell therapy made from the patient's own blood, engineered to target HER2 on tumor cells)

What this could lead to

If this treatment proves safe and effective, it could offer a new option for children with ependymoma that has not responded to standard treatments.

What could go wrong

This is an early Phase 1 trial with only 50 participants, focused mainly on safety. The treatment may not shrink tumors or may cause serious side effects. It is too early to know if it will work.

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.

ependymoma

As listed by the trial registrant

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

Contacts and locations

Locations

  • Ann & Robert H. Lurie Children's Hospital of Chicago

    Chicago, Illinois, 60611, United States

  • Children's Healthcare of Atlanta

    Atlanta, Georgia, 30329, United States

  • Children's Hospital Colorado

    Aurora, Colorado, 80045, United States

  • Children's Hospital Los Angeles

    Los Angeles, California, 90026, United States

  • Children's National Medical Center

    Washington D.C., District of Columbia, 20010, United States

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229, United States

  • Dana Farber Cancer Institute

    Boston, Massachusetts, 02215, United States

  • Memorial Sloan Kettering Cancer Center

    New York, New York, 10065, United States

  • Nationwide Children's Hospital

    Columbus, Ohio, 43205, United States

  • Pittsburgh Children's Hospital of UPMC

    Pittsburgh, Pennsylvania, 15224, United States

  • Texas Children's Cancer Center

    Houston, Texas, 77030, United States

  • The Hospital for Sick Children

    Toronto, Ontario, M5G1X8, Canada

  • University of Florida

    Gainesville, Florida, 32608, United States