How miR-155 and RUNX affect plexiform neurofibroma growth and treatment
MiR-155 and RUNX function in neurofibroma tumorigenesis and therapy
This project looks at how two cell molecules (miR-155 and RUNX) and cell stress pathways might be targeted to develop better treatments for people with NF1-related plexiform neurofibromas.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Cincinnati Childrens Hosp Med Ctr NIH-funded |
| Lab location | 1 site (Cincinnati, United States) |
| Project ID | NIH-11143101 on NIH RePORTER |
What this research studies
If you or a loved one has NF1 and plexiform neurofibromas, researchers at Cincinnati Children’s are studying why these benign nerve tumors grow and return after treatment. They compare human tumor samples with mouse models and lab-grown cells to see how miR-155, RUNX, and endoplasmic reticulum (ER) stress pathways drive tumor growth. The team tests whether blocking specific ER stress proteins (like PERK or VCP), alone or combined with the MEK drug selumetinib, slows tumor cell growth or reduces tumors in mice. Their work is preclinical now but aims to point the way to new drug combinations or trials that could help patients in the future.
Who could benefit from this research
Good fit: People with neurofibromatosis type 1 (NF1) who have plexiform neurofibromas, especially those interested in tissue donation or future clinical trials, are the most relevant candidates.
Not a fit: People without NF1 or whose tumors are not plexiform neurofibromas are unlikely to see direct benefits from this research in the near term.
Why it matters
Potential benefit: If successful, this work could point to new drug targets or drug combinations that shrink plexiform neurofibromas more effectively or keep them from coming back after treatment.
How similar studies have performed: The MEK inhibitor selumetinib already shrinks many plexiform neurofibromas but tumors often regrow after stopping, and early lab work (PERK knockdown in mice and VCP plus MEK inhibition in cells) suggests ER stress targeting may help but remains experimental.
Where this research is happening
Cincinnati, United States
- Cincinnati Childrens Hosp Med Ctr — Cincinnati, United States (Active)
Researchers
- Principal investigator: Wu, Jianqiang — Cincinnati Childrens Hosp Med Ctr
- Study coordinator: Wu, Jianqiang
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.