Targeting MDM2 to treat glioblastoma with normal (wild-type) TP53
MDM2 inhibitor therapy for TP53 wild-type GBM
This work tries a new drug that blocks MDM2 to help people with glioblastoma whose tumors have a normal (wild-type) TP53 gene.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Mayo Clinic Rochester NIH-funded |
| Lab location | 1 site (Rochester, United States) |
| Project ID | NIH-11189648 on NIH RePORTER |
What this research studies
If I were a patient, I'd hear that researchers are developing a drug called BI-907828 that frees the tumor-suppressor p53 to trigger cancer cell death. They are testing the drug in lab-grown human tumor samples implanted into animals (patient-derived xenografts) and combining it with radiation to see if tumors shrink and survival improves. The team measures drug effects by checking for stabilized p53 and increased activity of genes that cause cancer cells to die. Because the drug has limited entry into normal brain, they are using models with intact blood-brain barriers to understand how it might work in people and whether it could move toward clinical trials.
Who could benefit from this research
Good fit: Ideal candidates would be people with glioblastoma whose tumors retain a normal (wild-type) TP53 gene, especially those eligible for combined drug and radiation therapy trials.
Not a fit: Patients whose tumors have mutant or nonfunctional TP53 are unlikely to benefit from an MDM2-blocking approach.
Why it matters
Potential benefit: If successful, this approach could make radiation more effective and slow tumor growth in patients with TP53 wild-type glioblastoma, potentially extending survival.
How similar studies have performed: MDM2 inhibitors have shown promise in lab studies and some early clinical work in other cancers and in preclinical GBM models, but clear benefit in people with glioblastoma has not yet been proven.
Where this research is happening
Rochester, United States
- Mayo Clinic Rochester — Rochester, United States (Active)
Researchers
- Principal investigator: Sarkaria, Jann N. — Mayo Clinic Rochester
- Study coordinator: Sarkaria, Jann N.
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.