New combination treatment for SDH-deficient gastrointestinal stromal tumors
National Succinate Dehydrogenase-deficient GIST Translational Research and Clinical Trial Consortium
This project combines the chemotherapy temozolomide with a new drug that activates the DR5 death receptor to try to better treat people with SDH-deficient GIST.
Quick facts
| Grant type | U01 cooperative agreement |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California, San Diego NIH-funded |
| Lab location | 1 site (La Jolla, United States) |
| Project ID | NIH-11303253 on NIH RePORTER |
What this research studies
This consortium brings together multiple centers to focus on SDH-deficient GIST, a type of tumor that often does not respond to standard medicines like imatinib. The team uses patient-derived tumor cell cultures and a biorepository to guide treatment choices and laboratory studies. Based on lab findings that temozolomide increases DR5 on tumor cells and a new DR5 agonist triggers tumor cell death, researchers are running a multicenter clinical trial testing the two drugs together. The project also collects tumor samples and clinical data to learn which patients benefit and why.
Who could benefit from this research
Good fit: People with SDH-deficient GIST—including adolescents and adults whose tumors lack SDH function and who may have failed standard therapies—are the intended candidates.
Not a fit: Patients whose tumors are not SDH-deficient or who cannot safely take temozolomide or the DR5 drug are unlikely to benefit.
Why it matters
Potential benefit: If successful, the combination could control tumors better and offer a new option for patients whose SDH-deficient GIST does not respond to current therapies.
How similar studies have performed: Small patient reports showed temozolomide can help some SDH-deficient GIST patients, and lab studies show promising synergy with DR5 agonists, but the combined approach has not yet been proven in larger clinical trials.
Where this research is happening
La Jolla, United States
- University of California, San Diego — La Jolla, United States (Active)
Researchers
- Principal investigator: Sicklick, Jason Keith — University of California, San Diego
- Study coordinator: Sicklick, Jason Keith
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.