Brain-penetrating drugs that target SUMO1 for glioblastoma
Development of BBB-permeable SUMO1 small molecule degraders for glioblastoma therapy.
New oral drug candidates aim to enter the brain and remove a protein called SUMO1 that helps glioblastoma grow, for people with this aggressive brain tumor.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Indiana University Indianapolis NIH-funded |
| Lab location | 1 site (Indianapolis, United States) |
| Project ID | NIH-11308690 on NIH RePORTER |
What this research studies
Researchers discovered that a protein called SUMO1 helps glioblastoma stem cells survive and regrow tumors. They screened chemical libraries and found small molecules that tag SUMO1 for destruction inside tumor cells, then improved those compounds to better cross the blood–brain barrier. The team is optimizing lead compounds for oral use and testing them in patient-derived tumor cell cultures (neurospheres) and mouse tumor models to check tumor shrinkage, safety, and how the drug behaves in the body. The goal is to produce a candidate with the right brain penetration and safety profile to move toward human testing.
Who could benefit from this research
Good fit: People with glioblastoma, especially those whose tumors are resistant to standard chemotherapy or who have recurrent disease, would be the eventual candidates for therapies developed from this work.
Not a fit: People without glioblastoma or with brain tumors not driven by SUMO1 are unlikely to benefit from these specific drugs.
Why it matters
Potential benefit: If successful, this could lead to a new brain-penetrant oral therapy that more effectively shrinks glioblastoma and could improve survival compared with current options.
How similar studies have performed: Protein-degrading drugs (degraders) have shown promise in other cancers, but developing BBB‑penetrant SUMO1 degraders for glioblastoma is a newer and largely preclinical approach.
Where this research is happening
Indianapolis, United States
- Indiana University Indianapolis — Indianapolis, United States (Active)
Researchers
- Principal investigator: Hao, Chunhai Charlie — Indiana University Indianapolis
- Study coordinator: Hao, Chunhai Charlie
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.