What the trial was testing
The trial enrolled 53 patients with thyroid cancer. The study was sponsored by Dana-Farber Cancer Institute and tracked outcomes across the full group of patients who matched the trial's eligibility profile.
It was initial testing (phase 2). Trials at this stage are designed to produce evidence regulators and physicians can act on — not just observations to follow up later.
What the results showed
3 of 10 people with anaplastic thyroid cancer responded to the combination immunotherapy.
JAMA oncology · 2024 · NCT03246958
These findings — that tumors shrank with the two-drug immunotherapy combination — were published in the JAMA oncology and represent the headline result of the study.
Researchers tracked outcomes across 53 patients enrolled in the trial. The result was consistent enough across the group that the team felt confident reporting it.
What this means for patients
For patients with thyroid cancer, this result changes the calculus on what to ask their care team about. Whether it changes day-to-day care depends on factors like disease subtype, prior treatments, and where the patient is in their care journey.
What you can do now
This was mid-stage testing and the drug combination is not FDA-approved for thyroid cancer. The study didn't meet its main goal, but doctors saw some responses in rare, aggressive forms. If you have advanced thyroid cancer, ask your oncologist about approved options like lenvatinib or cabozantinib, or whether any immunotherapy trials might be right for you.
Eligibility for the treatments mentioned above depends on specific test results and clinical history. Bring this summary, the trial name, and your most recent labs or pathology report to your next visit.
Open thyroid cancer trials
Phase II Trial of Tunlametinib in NRAS-Mutant Advanced Thyroid Cancer
This phase II trial evaluates Tunlametinib (MEK inhibitor) ± PD-1 in NRAS-mutant advanced thyroid cancer. Key Objectives: Assess efficacy (ORR by RECIST v1.1) Evaluate safety profiles Study Design: Single-arm, single-center 4 cohorts based on: * Histology (differentiated vs. poorly/undifferentiated) * Prior therapy status Treatment: * Cohorts 1-2: Tunlametinib monotherapy (12mg BID) * Cohorts 3-4: Tunlametinib + PD-1 (commercially available) Key Procedures: Screening: NRAS testing + full staging (CT/MRI/PET) Monitoring: q3-week labs, q9-week imaging Follow-up: 30-day safety visit + q3-month survival tracking Endpoints: Primary: ORR Secondary: Safety (CTCAE), PFS, DoR Unique Aspects: First study targeting NRAS in thyroid cancer with MEK+PD-1 Includes rare aggressive subtypes (poorly/undifferentiated)
Agnostic Therapy in Rare Solid Tumors
The ANTARES study is a phase II basket trial designed to evaluate the tissue-agnostic efficacy of the monoclonal anti-PD1 antibody, nivolumab, in patients with advanced or metastatic rare tumors. The study aims to treat rare malignancies with PD-L1 expression (CPS ≥ 10), regardless of the tumor's tissue type or location. Patients who have not responded to standard treatments will be included, and treatment will last for up to 12 months. The study will assess objective response, progression-free survival, and biomarkers such as PD-L1, ctDNA, and microvesicles, in a multicenter collaborative effort to provide innovative therapeutic options for this underrepresented population