Chordoma

P4, N=60, West China Hospital , Sichuan University; West China Hospital , Sichuan University

P2, N=27, Active, not recruiting, Sichuan Baili Pharmaceutical Co., Ltd. | Recruiting --> Active, not recruiting | N=40 --> 27

P1/2, N=49, Active, not recruiting, Susan Chi, MD | Recruiting --> Active, not recruiting

Molecular insights have identified key drivers, including Brachyury [T-box transcription factor T (TBXT)], receptor tyrosine kinases, and emerging biomarkers that may guide future therapeutic selection. Integrated multimodal care is critical for improving survival and function in patients with sacral chordomas.

This review provides the first evidence-weighted map of intercellular signaling in chordoma. While RTK autocrine loops remain the most established, emerging cytokine- and CAF-mediated pathways highlight new biological mechanisms and potential therapeutic targets. Bridging single-cell discovery with functional validation and clinical translation will be essential to advance microenvironment-directed therapies in chordoma.

Our case highlights the potential for diverse morphology in chordomas and emphasizes the utility of an IHC panel, including anti-brachyury antibody, to aid in diagnosis. Our case also demonstrates the importance of evaluating the clivus during postmortem CNS examination.

P1/2, N=12, Active, not recruiting, Memorial Sloan Kettering Cancer Center | Recruiting --> Active, not recruiting | N=46 --> 12

Stromal myeloid cells showed increased CD47 and PD-1 expression. Our study identified distinct epigenetic profiles in sacral chordomas, which were associated with recurrence, and revealed expression of checkpoint markers TIM3, CD47 and PD-1, warranting further investigation through functional validation.

The tumor mutational burden was low overall, varied between patients and timepoints, and tended to be higher in recurrent cases. Quantitative nuclear morphometry, integrated with immunophenotyping and genomic profiling, captures recurrence-associated phenotypic remodeling in chordoma and may provide a quantitative framework for future digital pathology or AI approaches, pending validation in larger cohorts.

P=N/A, N=72, Recruiting, Mayo Clinic | Trial completion date: Aug 2028 --> Aug 2031

The immunosuppressive chordoma TME is associated with clinical outcomes and our data suggests OV infection reverses this deleterious immunosuppressive profile. These studies provide a framework for future clinical implementation amongst chordoma patients.