Engineered immune cells take on childhood cancers that Won't quit
NCT ID NCT04539366
First seen Jun 25, 2026 · Last updated Aug 06, 2026 · Updated 7 times
Summary
This phase 1 trial is testing a new treatment called GD2CART for children and young adults with osteosarcoma or neuroblastoma that has returned or does not respond to standard treatments. GD2CART is made by taking a patient's own T cells (a type of immune cell), adding a new gene so they can recognize and attack cancer cells that have the GD2 protein on their surface. The study aims to find the safest dose and see if the treatment can shrink tumors.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- GD2CART (a patient's own T cells genetically modified to target the GD2 protein on tumor cells)
- What this could lead to
- If this works, it could point toward a new treatment option for children and young adults with hard-to-treat osteosarcoma or neuroblastoma that has come back or not responded to standard therapy.
- What could go wrong
- This is an early phase 1 trial, so it is primarily testing safety and dosing. The therapy may not shrink tumors or provide lasting benefit, and there are risks of side effects from the cell infusion and chemotherapy.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Children's Hospital Colorado
Aurora, Colorado, 80045, United States
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Children's Hospital Los Angeles
Los Angeles, California, 90027, United States
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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Lucile Packard Children's Hospital Stanford University
Palo Alto, California, 94304, United States
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National Institutes of Health Clinical Center
Bethesda, Maryland, 20892, United States
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University of Wisconsin Carbone Cancer Center - University Hospital
Madison, Wisconsin, 53792, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Slowing down immunotherapy infusion may reduce pain in children with Tough-to-Treat neuroblastoma
- Supercharged immune cells take aim at childhood cancers
- Desperate hope: single patient gets custom RNA therapy for returning bone cancer
- Targeted drug shows promise for kids with tough cancers
- New hope for kids with Hard-to-Treat cancers: targeted drug shows promise
- New hope for kids with tough cancers: experimental drug PEEL-224 enters trials