The latest research published in the Journal of Clinical Oncology strengthens the promise of the first-ever treatment developed for low-grade serous ovarian cancer (LGSOC). The findings expand the potential reach of the drug combination avutometinib and defactinib, bringing hope to more people facing this historically hard-to-treat disease.

FDA Approval & Expanding Options for LGSOC

In May, the U.S. Food and Drug Administration (FDA) approved the drug combination avutometinib (a RAF/MEK inhibitor) and defactinib (a FAK inhibitor) for the treatment of recurrent LGSOC in patients with a KRAS mutation. Now, new results from the phase II RAMP 201 clinical trial reveal the duo may also benefit patients without the KRAS mutation—known as KRAS “wild type.”

​Key Findings from the RAMP 201 Study

Researchers found significant improvements in both objective response rate (ORR) and progression-free survival (PFS) for patients treated with avutometinib and defactinib—regardless of KRAS status:

Why This Matters: Traditional Treatments Fall Short

LGSOC is rare—fewer than 10% of all ovarian cancers—and typically affects younger women, with an average diagnosis age of 45. Unlike high-grade serous ovarian cancer, LGSOC generally grows slowly but can be resistant to standard chemotherapy.

Standard frontline treatment includes surgery and chemotherapy, often followed by hormone therapy. But in recurrence, traditional chemotherapy yields only a 13% response rate. In contrast, this new drug combination produced robust tumor shrinkage in nearly a third of patients and extended time before progression.

Safety: A Well-Tolerated, Lower-Toxicity Option

The RAMP 201 trial showed the avutometinib/defactinib combo to be generally well-tolerated. The common side effects included fatigue, swelling, rashes, nausea, vomiting, and diarrhea—typical for MEK and FAK inhibitors—with just 10% of patients discontinuing due to side effects. This is a promising, less toxic alternative to conventional chemotherapy.

The Future: Toward a New Standard of Care

With a phase III trial already underway to evaluate the combination in patients with and without a KRAS mutation, avutometinib and defactinib are pushing the boundaries in LGSOC treatment—bringing new hope to patients who haven’t responded to previous therapies.

Supporting Progress: STAAR Low-Grade Serous Ovarian Cancer Foundation

Progress in LGSOC research wouldn’t be possible without advocacy and funding. The STAAR Low-Grade Serous Ovarian Cancer Foundation is the first U.S. nonprofit dedicated exclusively to low-grade serous ovarian cancer. Since 2020, when it was started by three women with LGSOC, STAAR has helped fund more than $1 million into advancing research targeted at this rare cancer.

To read more, get involved, or support ongoing LGSOC research, visit staaroc.org