A new drug combination for specific types of thyroid cancer
Phase II trial of avutometinib plus defactinib in RAF dimer-driven thyroid cancers
This research is testing a combination of two medicines, avutometinib and defactinib, for people with rare and aggressive forms of thyroid cancer that have specific genetic changes.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Sloan-Kettering Inst Can Research NIH-funded |
| Lab location | 1 site (New York, United States) |
| Project ID | NIH-11193449 on NIH RePORTER |
What this research studies
Two rare types of thyroid cancer, radioiodine-refractory differentiated thyroid cancer and anaplastic thyroid cancer, currently have very poor outcomes. While some targeted treatments exist for other genetic changes, therapies for tumors driven by RAF dimers (like RAS, NF1, and non-V600 BRAF mutations) are still needed. This study uses avutometinib, a new type of MEK inhibitor, which has shown promise in RAS-mutant tumors. However, it can lead to resistance, so researchers are combining it with defactinib to block a pathway called FAK that contributes to this resistance.
Who could benefit from this research
Good fit: Ideal candidates are patients with radioiodine-refractory, recurrent, or metastatic differentiated thyroid cancer, or anaplastic thyroid cancer, whose tumors are driven by RAF dimer-activating mutations like RAS, NF1, or non-V600 BRAF.
Not a fit: Patients whose thyroid cancers do not have these specific RAF dimer-activating genetic mutations may not benefit from this particular treatment approach.
Why it matters
Potential benefit: If successful, this drug combination could offer a new and more effective treatment option for patients with these aggressive and hard-to-treat thyroid cancers.
How similar studies have performed: Avutometinib alone has shown promising activity against RAS-mutant tumors, but this combination with defactinib is designed to overcome resistance mechanisms observed with single-drug therapy.
Where this research is happening
New York, United States
- Sloan-Kettering Inst Can Research — New York, United States (Active)
Researchers
- Principal investigator: Ho, Alan L. — Sloan-Kettering Inst Can Research
- Study coordinator: Ho, Alan L.
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.