Targeting the ATIC enzyme in diffuse intrinsic pontine glioma (DIPG)

ATIC is a novel molecular target in diffuse intrinsic pontine glioma

['FUNDING_R01'] · EMORY UNIVERSITY · NIH-11233309

Researchers are testing ways to block a metabolic enzyme called ATIC to try to slow or stop aggressive DIPG tumors in children.

Quick facts

Phase['FUNDING_R01']
Study typeNih_funding
SexAll
SponsorEMORY UNIVERSITY (nih funded)
Locations1 site (ATLANTA, UNITED STATES)
Trial IDNIH-11233309 on ClinicalTrials.gov

What this research studies

This work looks at a metabolic enzyme, ATIC, that appears increased in DIPG tumors driven by the H3K27M mutation. Scientists used metabolomics and gene data from patient-derived tumor lines to identify ATIC as a possible vulnerability. In mice, removing ATIC with genetic tools improved survival, but a single ATIC-blocking drug was not enough, so the team will test drug combinations that target purine and one‑carbon metabolism. The experiments use molecular tools and two independent DIPG mouse models built from human tumor lines to see whether these approaches reduce tumor growth and extend survival.

Who could benefit from this research

Good fit: Children diagnosed with diffuse intrinsic pontine glioma, particularly those whose tumors carry the H3K27M mutation, would be the likely future candidates for treatments developed from this work.

Not a fit: People without DIPG, those with other brain tumor types, or patients seeking immediate clinical treatment are unlikely to receive direct benefit from this preclinical research.

Why it matters

Potential benefit: If successful, this research could lead to new combination therapies that slow DIPG growth and extend survival for affected children.

How similar studies have performed: This approach is relatively novel for DIPG—preclinical CRISPR removal of ATIC improved survival in mice, but small-molecule ATIC inhibition alone has not yet been effective and requires combination strategies.

Where this research is happening

ATLANTA, UNITED STATES

Researchers

About this research

  1. This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
  2. Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
  3. For full project details, budget, and progress reports, visit the official NIH RePORTER page below.

View on NIH RePORTER →

Last reviewed 2026-05-15 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.