Immune approaches for U1‑mutated Sonic Hedgehog medulloblastoma
Official title: Project 3: Exploit Immune Consequences of U1 Mutations in Sonic Hedgehog (Shh) Medulloblastoma
Funded at University of Tx Md Anderson Can Ctr · NIH-11193455 · Data updated 2026-11-12
This project looks at whether immune-based therapies can help people with Sonic Hedgehog medulloblastoma that carry a U1 spliceosome mutation.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Tx Md Anderson Can Ctr NIH-funded |
| Lab location | 1 site (Houston, United States) |
| Project start | September 1, 2008 |
| Project end | August 31, 2029 |
| Funding year | 2025 |
| Award (this year) | $487,130 |
| NIH project number | 5P50CA127001-17 |
| Project ID | NIH-11193455 on NIH RePORTER |
Who could benefit from this research
✓Could help
The ideal candidates are people (children, adolescents, or adults) with Sonic Hedgehog medulloblastoma whose tumors carry the U1 (r.3A>G) mutation, especially Shh‑δ adults and Shh‑α cases with TP53 changes.
✕Less likely to help
Patients without the U1 mutation or those with non‑Shh medulloblastoma subtypes are unlikely to benefit from approaches targeted to U1‑mutant tumors.
What this research studies
If you or your child has a Sonic Hedgehog (Shh) medulloblastoma with a U1 mutation, researchers are studying how those tumors make many new, tumor-specific protein pieces that could alert the immune system. The team examines tumor tissue and lab models to identify the novel antigens created by abnormal splicing and tests whether these tumors respond to immune checkpoint drugs or other immune approaches. Their work combines analysis of patient tumor samples, molecular lab studies, and preclinical testing to build therapies that could move into clinical trials. Successful findings would guide future treatment options tailored to people whose tumors have the U1 change.
Why it matters
Potential benefit: If successful, this work could lead to immune-based treatments that improve outcomes and reduce the need for harsh standard therapies in patients with U1‑mutant Shh medulloblastoma.
How similar studies have gone: Immune checkpoint inhibitors have helped several hypermutant cancers, but applying immune therapies specifically to U1‑mutant Shh medulloblastoma is a new and largely untested approach.
Where this research is happening
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University of Tx Md Anderson Can Ctr
Houston, United States Active
Researchers
- Principal investigator: Majd, Nazanin Koohbanani — University of Tx Md Anderson Can Ctr
- Study coordinator: Majd, Nazanin Koohbanani
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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.