Leiomyosarcoma

This report, representing the first documented case of LLMS from Central Asia, contributes to the limited global experience with this rare tumor. Our review identified four additional LLMS cases published between 2021 and 2024, totaling five recent reports including the present case. Collectively, these demonstrate persistent male predominance, glottic and supraglottic predilection, and survival outcomes consistent with previous observations. Complete surgical excision remains the cornerstone of therapy, while multidisciplinary-guided adjuvant treatment may benefit selected high-grade or high-risk patients. Continued accumulation and molecular characterization of cases are needed to refine prognostic assessment and optimize management strategies.

P1, N=85, Terminated, Boundless Bio, Inc. | Trial completion date: Mar 2027 --> Mar 2026 | Active, not recruiting --> Terminated | Trial primary completion date: Sep 2026 --> Mar 2026; Decision was made to halt the study based on overall clinical experience and market considerations. This decision was not driven by any safety signal.

P1, N=15, Not yet recruiting, M.D. Anderson Cancer Center

P2/3, N=345, Active, not recruiting, Pharma Mar S.A. | Recruiting --> Active, not recruiting

In xenografts, atorvastatin suppressed ULMS tumor growth by ~50% with minimal toxicity, as evidenced by normal serum ALT and creatinine levels and preserved organ histology. These findings identify protein geranylgeranylation as a novel therapeutic vulnerability in ULMS and support statin repurposing as a promising treatment strategy.

Our group has been interested in utilizing the synthetic toll-like receptor 4 (TLR4) agonist, glycopyranosyl lipid A in stable-emulsion formulation (GLA-SE), in patients with advanced STSs...Together, these findings demonstrate the potential of IT GLA-SE to elicit antigen-specific T cell clonal expansion as a component of immunotherapeutic strategies to combat metastatic STSs. Expanded studies are needed to explore the clinical and immunologic effects of IT GLA-SE, particularly in combination with other immunotherapies.

Through an epigenetic inhibitor screen, we identify and validate SMARCA4/2 inhibition as a promising therapeutic vulnerability for MES leiomyosarcomas. Together, this work defines two epigenetically driven, transcription factor-regulated, and clinically relevant states of leiomyosarcoma, revealing mechanistic underpinnings of tumor heterogeneity and uncovering actionable therapeutic strategies.

P1, N=25, Recruiting, University of California, Irvine

P1, N=18, Recruiting, Washington University School of Medicine | Trial primary completion date: Nov 2028 --> May 2031 | Trial completion date: Oct 2033 --> Apr 2036

RAF/BRAF-driven mesenchymal tumours possess a broader clinicopathologic spectrum than traditionally recognised, frequently affecting adults and deep/visceral sites. Their inherently variable immunophenotypes and the presence of high-grade morphologic features do not strictly predict an aggressive clinical trajectory. Comprehensive molecular profiling is essential to refine diagnostic criteria, accurately identify these neoplasms, and elucidate the genomic events associated with tumour progression.

MEF2C and THRA2 were associated with NCC markers. These findings define epigenomic features underlying lineage divergence and provide insight into NCC-related diseases such as leiomyosarcoma and neuroblastoma.

Pathology thus represented the diagnostic cornerstone. This case contributes to the limited literature describing MRI features of small bowel leiomyosarcoma presenting with adult intussusception and underscores the importance of a multimodal diagnostic approach, in which MRI serves as an adjunct to CT, while histopathology remains essential for definitive diagnosis.