Investigating new treatments for a specific type of gastrointestinal stromal tumor (GIST).
Official title: National Succinate Dehydrogenase-deficient GIST Translational Research and Clinical Trial Consortium
Funded at University of California, San Diego · NIH-11049088 · Data updated 2026-11-12
This study is looking at a new way to treat gastrointestinal stromal tumors (GIST) caused by specific gene changes, by combining a chemotherapy drug called temozolomide with a new treatment that targets a protein to see if it helps patients feel better.
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 start | March 5, 2024 |
| Project end | February 28, 2029 |
| Funding year | 2025 |
| Award (this year) | $637,789 |
| NIH project number | 5U01CA278470-02 |
| Project ID | NIH-11049088 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with SDH-deficient gastrointestinal stromal tumors, particularly adolescents and young adults.
✕Less likely to help
Patients with GISTs that do not have SDH deficiencies or those who do not meet the trial criteria may not benefit from this research.
What this research studies
This research focuses on gastrointestinal stromal tumors (GIST) that are caused by mutations affecting the succinate dehydrogenase (SDH) complex. It aims to explore the effectiveness of combining the chemotherapy drug temozolomide (TMZ) with a new therapeutic agent that targets death receptor 5 (DR5) to improve treatment outcomes for patients with SDH-deficient GISTs. The study includes a multicenter phase II trial and utilizes a unique biorepository of patient-derived cell cultures to test these treatments. Patients may have the opportunity to participate in clinical trials that assess the safety and efficacy of this combined approach.
Why it matters
Potential benefit: If successful, this research could provide a more effective treatment option for patients with SDH-deficient GISTs, potentially improving survival rates.
How similar studies have gone: Previous studies have shown promising results with temozolomide in small cohorts, indicating potential for success with this combined treatment approach.
Where this research is happening
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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.