Dual-target therapy for B‑cell acute lymphoblastic leukemia
Combinatorial targeting for the treatment of B-cell acute lymphoblastic leukemia
A two-part treatment that delivers gene‑silencing RNA and a small drug together to fight high‑risk B‑cell acute lymphoblastic leukemia, including Philadelphia chromosome cases.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | West Virginia University NIH-funded |
| Lab location | 1 site (Morgantown, United States) |
| Project ID | NIH-11140427 on NIH RePORTER |
What this research studies
This project aims to attack B‑cell ALL cells in two ways: using RNA to switch off the STAT5 gene and a small molecule (NL‑1) that disrupts a mitochondrial protein called MitoNEET. Because the RNA and NL‑1 are unstable or insoluble on their own, researchers are developing ionizable lipid nanoparticles to carry both agents into leukemia cells. Early lab tests show these nanoparticles can encapsulate and deliver the combination to B‑cell ALL cells in vitro, and the team plans follow‑up studies to compare the combo against each agent alone. If those results are promising, the work could advance to animal testing and eventually to human studies or clinical trials.
Who could benefit from this research
Good fit: People with B‑cell acute lymphoblastic leukemia, especially those with Philadelphia chromosome (BCR‑ABL) positive or other high‑risk disease, are the intended candidates.
Not a fit: Patients without B‑cell ALL, those whose leukemia is driven by unrelated mechanisms, or people unable to receive investigational therapies would not be expected to benefit directly from this project.
Why it matters
Potential benefit: If successful, this approach could produce a more effective treatment for high‑risk B‑cell ALL and help reduce relapse rates.
How similar studies have performed: STAT5 knockdown and NL‑1 have shown anti‑leukemia activity in lab studies and lipid nanoparticles have successfully delivered RNA in other conditions, but combining STAT5 siRNA and NL‑1 for B‑cell ALL remains largely untested in patients.
Where this research is happening
Morgantown, United States
- West Virginia University — Morgantown, United States (Active)
Researchers
- Principal investigator: Bobbala, Sharan Kumar Reddy — West Virginia University
- Study coordinator: Bobbala, Sharan Kumar Reddy
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.