MT-125: a brain-penetrant treatment to slow glioblastoma spread and growth
Official title: Phase 0/I dose escalation trial of MT-125 monotherapy in recurrent high-grade gliomas
Funded at Myosin Therapeutics INC. · NIH-11387631 · Data updated 2026-11-12
A new drug, MT-125, taken by people with recurrent glioblastoma, targets two motor proteins to slow cancer spread and growth inside the brain.
Quick facts
| Grant type | Sbir 2 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Myosin Therapeutics INC. NIH-funded |
| Lab location | 1 site (Jupiter, United States) |
| Project start | April 1, 2023 |
| Project end | July 31, 2027 |
| Funding year | 2026 |
| Award (this year) | $2,241,448 |
| NIH project number | 5R44CA278293-05 |
| Project ID | NIH-11387631 on NIH RePORTER |
Who could benefit from this research
✓Could help
Adults 21 years and older with recurrent high-grade gliomas, including glioblastoma, who meet specific health criteria and can participate safely in a phase 0/I dose-escalation study.
✕Less likely to help
Patients who do not tolerate the drug or who have tumors not driven by NMIIA/IIB pathways may not benefit.
What this research studies
This project tests MT-125, a brain-penetrant drug designed to block two non-muscle myosin II motors that control invasion and proliferation of glioblastoma cells. In preclinical work, MT-125 was shown to slow tumor spread and extend survival, and it works well with a current brain-penetrant therapy. The trial will start with careful dose escalation to find a safe, effective dose for patients with recurrent high-grade gliomas. Researchers will monitor safety, brain drug levels, and tumor response to determine whether MT-125 could become a new option for GBM patients.
Why it matters
Potential benefit: If successful, MT-125 could become a safer and more effective therapy to slow tumor growth and improve survival for people with recurrent glioblastoma.
How similar studies have gone: Preclinical studies showed MT-125 blocks invasion and proliferation and extends survival, supporting progression to human testing.
Where this research is happening
-
Myosin Therapeutics INC.
Jupiter, United States Active
Researchers
- Principal investigator: Brownstein, Michael J — Myosin Therapeutics INC.
- Study coordinator: Brownstein, Michael J
Related research
Other NIH-funded projects on the same topics.
-
How immune myeloid cells affect aggressive childhood brain tumors
Researchers are looking at whether immune cells called macrophages and neutrophils inside and around aggressive childhood brain tumors are linked to tumor behavior and survival in children with high-grade gliomas.
-
MT-125 treatment for recurrent high-grade brain tumors
Adults with recurrent high-grade gliomas will receive the brain-penetrant drug MT-125 to find safe doses and check whether it can slow tumor invasion and growth.
-
Investigating the role of immune cells in childhood brain tumors
This study is looking at how certain immune cells in the brain, called macrophages and neutrophils, affect a type of brain cancer in kids, and it hopes to find new ways to help treat these young patients better.
-
Understanding how a specific histone mutation affects brain tumor development in children
This study is looking at how a specific change in a gene related to brain development might lead to brain tumors in kids, with the hope that understanding this could help find better treatments for these tough-to-treat tumors.
-
Developing mRNA therapies for a deadly pediatric brain tumor
This study is exploring a new way to treat a tough brain tumor called diffuse midline glioma in kids by using special particles to deliver gene therapy directly to the tumor, aiming to improve how we fight this disease.
-
Using oncolytic viruses to deliver genes for treating high-grade gliomas
This study is exploring a new way to treat aggressive brain tumors in kids by using special viruses that can target and destroy cancer cells, while also boosting the body's immune system to help fight the cancer better, and some patients may have the chance to try these exciting new treatments in clinical trials.
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.