New treatment approaches for children's tumors without obvious mutation targets
Developing new therapeutic strategies for pediatric tumors that lack clinically actionable mutations
Finding drugs that target essential non-mutated genes to help children whose tumors lack the usual mutation targets.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | St. Jude Children's Research Hospital NIH-funded |
| Lab location | 1 site (Memphis, United States) |
| Project ID | NIH-11160519 on NIH RePORTER |
What this research studies
Researchers will use machine learning to combine large laboratory datasets (like DepMap, CCLE, and PRISM) with genetic and molecular data from children's tumors to find genes the cancers depend on even when those genes are not mutated. They will test these candidate vulnerabilities in cancer cells grown in the lab and in animal models to see if drugs can safely block them. The goal is to nominate druggable targets for specific pediatric tumor subtypes and carry out the preclinical work needed to move promising treatments toward clinical testing. The project focuses on pediatric cancers that currently have few mutation-based treatment options.
Who could benefit from this research
Good fit: Children and adolescents with tumors that have few or no actionable mutations, especially those with relapsed or treatment-resistant pediatric cancers.
Not a fit: Patients whose cancers already have effective mutation-targeted treatments or whose tumors do not depend on the identified non-oncogene vulnerabilities may not benefit from this work.
Why it matters
Potential benefit: Could produce new, less-toxic targeted therapies for children whose tumors lack actionable mutations and reduce reliance on harsh chemotherapy and radiation.
How similar studies have performed: Large-scale preclinical screens have identified non-mutated cancer dependencies and some candidate targets, but applying this approach broadly to pediatric cancers and translating it to treatments is still relatively new.
Where this research is happening
Memphis, United States
- St. Jude Children's Research Hospital — Memphis, United States (Active)
Researchers
- Principal investigator: Geeleher, Paul — St. Jude Children's Research Hospital
- Study coordinator: Geeleher, Paul
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.