Experimental cell therapy targets deadly childhood brain cancer

NCT ID NCT03396575

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

Summary

This early-phase trial tested a new immunotherapy approach for children with DIPG, a rare and aggressive brain stem tumor. After standard radiation and chemotherapy, patients received special vaccines and immune cells designed to attack the tumor. The study was small (11 participants) and was terminated early, so it is unclear how well it works, but researchers were checking for safety and immune responses.

What this could mean

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

Active substance

TTRNA-DC vaccines with GM-CSF and TTRNA-xALT T cells

What this could lead to

If it works, this could point toward a new treatment approach for DIPG, a rare and deadly childhood brain cancer.

What could go wrong

This was a very early, small Phase I trial that was terminated early, so results are limited. The treatment involves strong chemotherapy and cell therapy, which can cause serious side effects.

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.

brain cancer brain stem glioma cerebrotendinous xanthomatosis diffuse intrinsic pontine glioma influenza

As listed by the trial registrant

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

Contacts and locations

Locations

  • UF Health Shands Children's Hospital

    Gainesville, Florida, 32610, United States