Blocking key gene activity in childhood brainstem glioma (DIPG)
Targeting Transcriptional Elongation in Pediatric Glioma
Researchers are looking for drug combinations that stop or slow DIPG tumor growth in children by targeting gene activity that helps tumors survive.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Alabama at Birmingham NIH-funded |
| Lab location | 1 site (Birmingham, United States) |
| Project ID | NIH-11308192 on NIH RePORTER |
What this research studies
Researchers are using genome-wide CRISPR gene-editing screens on cells derived from children’s DIPG tumors to find which genes let the tumors survive BRD4-blocking drugs. Promising targets are being tested in patient-derived tumor models grown in animals to see which drug combinations prevent or delay resistance. The team prioritizes targets that can be hit with existing drugs so successful combinations can move toward clinical trials for kids. Families who can donate tumor tissue or participate in future trials could directly help guide which treatments move forward.
Who could benefit from this research
Good fit: Children diagnosed with diffuse intrinsic pontine glioma (DIPG), especially families willing to provide tumor tissue or consider future clinical trials, would be most relevant.
Not a fit: People without DIPG, including adults with other brain tumors or children who cannot undergo biopsy or trial procedures, are unlikely to benefit directly from this work.
Why it matters
Potential benefit: Could identify drug combinations that prevent resistance to BRD4-targeting drugs and lead to clinical trials that extend survival for children with DIPG.
How similar studies have performed: Preclinical studies showed BRD4 inhibitors like JQ1 can delay DIPG growth in animal models, but tumors often become resistant, so this genome-wide CRISPR approach to find combination partners is relatively new.
Where this research is happening
Birmingham, United States
- University of Alabama at Birmingham — Birmingham, United States (Active)
Researchers
- Principal investigator: Hashizume, Rintaro — University of Alabama at Birmingham
- Study coordinator: Hashizume, Rintaro
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page below.