Venetoclax-based combination treatments for high-risk neuroblastoma
ABT-199 based therapies to treat neuroblastoma
Researchers are developing combination therapies that pair venetoclax with new drugs to better treat children with high-risk or relapsed neuroblastoma.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Virginia Commonwealth University NIH-funded |
| Lab location | 1 site (Richmond, United States) |
| Project ID | NIH-11082436 on NIH RePORTER |
What this research studies
This project combines venetoclax (a BCL-2 blocker) with two new drug types—SHP2 inhibitors and a BCL-xL–targeting antibody–drug conjugate—to find therapies that more reliably kill neuroblastoma. The team used high-throughput lab screening and tumor models to see which tumors are most sensitive, with special focus on relapsed and MYCN-amplified tumors. They study how MAPK/MEK pathway changes drive resistance and how the new combinations overcome that resistance in cell and animal models. The aim is to select the safest, most promising combinations to advance toward early human trials for children with aggressive disease.
Who could benefit from this research
Good fit: Children and adolescents with high-risk or relapsed neuroblastoma, especially those with MYCN-amplified tumors or tumors showing BCL-2 dependence, would be the most likely candidates.
Not a fit: Patients without neuroblastoma or with tumors that do not depend on BCL-2/BCL-xL pathways are unlikely to benefit from these specific combinations.
Why it matters
Potential benefit: If successful, this work could lead to more effective combination treatments for children with high-risk or relapsed neuroblastoma and open new options for clinical trials.
How similar studies have performed: Venetoclax has dramatically improved some adult blood cancers and earlier lab studies showed promise in neuroblastoma, but the specific SHP2 and BCL-xL ADC combinations here are largely preclinical and not yet proven in patients.
Where this research is happening
Richmond, United States
- Virginia Commonwealth University — Richmond, United States (Active)
Researchers
- Principal investigator: Faber, Anthony Charles — Virginia Commonwealth University
- Study coordinator: Faber, Anthony Charles
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.