6-thio-dG, a telomerase-targeting drug for adult glioblastoma
6-thio-2'-deoxyguanosine in GBM: Evaluation of Pharmaco-dynamics, Effects of Prior Standard of Care and A Human Phase 0 Study
A short early human trial giving adults with glioblastoma a brain-penetrating drug called 6-thio-dG that targets telomerase and may trigger tumor-killing immune responses.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Duke University NIH-funded |
| Lab location | 1 site (Durham, United States) |
| Project ID | NIH-11164689 on NIH RePORTER |
What this research studies
This project develops a new drug, 6-thio-dG, that can cross the blood–brain barrier and is incorporated into cancer cell telomeres to cause DNA damage and immune activation. Researchers will complete lab and animal work to identify biomarkers that signal the drug is working and then run a Phase 0 human study to look for those same signs in patients. The Phase 0 part typically gives a small number of participants limited exposure before surgery so tumor tissue and blood can be tested for drug penetration and biological effects. The work aims to link the preclinical findings to real patient samples to guide later trials.
Who could benefit from this research
Good fit: Adults with confirmed glioblastoma who can travel to Duke University, are eligible for surgery, and are willing to provide tumor tissue and blood samples for biomarker testing are the best fit.
Not a fit: People not having surgery, children, or patients whose tumors lack telomerase activation should not expect direct clinical benefit from this early Phase 0 effort.
Why it matters
Potential benefit: If successful, this approach could trigger immune responses and tumor cell death in many telomerase-positive glioblastomas and guide development of more effective treatments.
How similar studies have performed: Preclinical studies show 6-thio-dG causes telomeric DNA damage and immune-mediated killing in telomerase-positive models, but human clinical experience with this approach is very limited and remains early-stage.
Where this research is happening
Durham, United States
- Duke University — Durham, United States (Active)
Researchers
- Principal investigator: Khasraw, Mustafa — Duke University
- Study coordinator: Khasraw, Mustafa
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.