Stopping N‑myc–driven protein production in childhood medulloblastoma
Remodeling the translatome in N-myc mediated medulloblastoma and its therapeutic implications
['FUNDING_R01'] · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · NIH-11144473
This project looks at ways to block how N‑myc and the mTOR pathway change protein production in aggressive childhood medulloblastoma.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | UNIVERSITY OF CALIFORNIA, SAN FRANCISCO (nih funded) |
| Locations | 1 site (SAN FRANCISCO, UNITED STATES) |
| Trial ID | NIH-11144473 on ClinicalTrials.gov |
What this research studies
From the patient perspective, researchers are examining how the N‑myc (MYCN) protein hijacks the cell's protein‑making machinery to drive an aggressive form of pediatric medulloblastoma. They use ribosome profiling and molecular experiments to find which messages (mRNAs) are preferentially translated under N‑myc and how mTOR contributes. The team will dissect the mechanisms behind altered translation and test whether targeting those steps creates vulnerabilities in tumor cells. Findings could guide development of targeted drugs that specifically disrupt N‑myc–dependent protein production.
Who could benefit from this research
Good fit: Children with SHH‑subgroup medulloblastoma that show MYCN (N‑myc) amplification or high N‑myc activity would be the most relevant candidates for interventions arising from this work.
Not a fit: Patients whose tumors lack N‑myc/MYCN involvement or who have other medulloblastoma subgroups (for example WNT or Group 4 without MYCN amplification) are less likely to benefit from N‑myc–targeted approaches.
Why it matters
Potential benefit: If successful, this work could lead to new targeted treatments that slow or stop growth of N‑myc–driven medulloblastoma and reduce reliance on damaging therapies like high‑dose radiation and chemotherapy.
How similar studies have performed: mTOR inhibitors and other translation‑targeting strategies have shown some preclinical activity but have had limited clinical success so far, making this focused approach on N‑myc–directed translation a relatively new direction.
Where this research is happening
SAN FRANCISCO, UNITED STATES
- UNIVERSITY OF CALIFORNIA, SAN FRANCISCO — SAN FRANCISCO, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: WEISS, WILLIAM A — UNIVERSITY OF CALIFORNIA, SAN FRANCISCO
- Study coordinator: WEISS, WILLIAM A
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.
Conditions: Cancer Genes, Cancer-Promoting Gene, Cancers