Pre-Surgical Combination Therapy Enhances Survival in Rare Thyroid Cancer Patients

A recent study from The University of Texas MD Anderson Cancer Center has demonstrated that a pre-surgical combination treatment involving pembrolizumab, dabrafenib, and trametinib significantly improves outcomes in patients with BRAF V600E-mutated anaplastic thyroid cancer (BRAFm-ATC). This Phase II multicenter trial revealed that adding immunotherapy to targeted therapies not only increased the likelihood of tumor resection, with 74% of patients able to undergo surgery compared to just 5% with previous treatments, but also extended overall survival from an average of 14.5 to 18 months.

Anaplastic thyroid cancer is known for its aggressive nature, often presenting with tumors that are difficult to remove surgically. The study’s lead investigator, Dr. Mark Zafereo, noted that traditional treatments offered limited benefits, typically extending survival by only a few months. In contrast, incorporating pembrolizumab, an immunotherapy agent, alongside dabrafenib and trametinib—which inhibit the mutant BRAF protein—enabled better disease control.

Patients enrolled in the study between September 2021 and January 2025 received a median of four therapy cycles before surgery, with 30 out of 40 patients undergoing tumor removal. The combination therapy also doubled the progression-free survival, from approximately 6.7 months to over a year, indicating a delay in cancer progression.

While side effects such as kidney injury, sepsis, and intestinal tear were observed, the results suggest that this approach could become a new standard of care for BRAFm-ATC, especially for patients with the specific BRAF mutation. The study’s limitations include its small size, lack of a control group, and limited follow-up duration.

This research underscores the potential of combining immunotherapy with targeted treatments to improve surgical outcomes and prolong survival in aggressive thyroid cancers. Further studies are needed to confirm these findings and establish long-term benefits.

Source: https://medicalxpress.com/news/2025-06-pre-surgical-combination-therapy-boosts.html