^
    19h
    Characteristics of the cytogenetic variants of alveolar rhabdomyosarcoma (PubMed, Arkh Patol)
    Four cytogenetic patterns were observed: classical translocation, translocation with amplification, translocation with deletion, and normal signal distribution. Alveolar rhabdomyosarcomas exhibit genetic heterogeneity and a diversity of cytogenetic profiles. The frequency ratio of PAX3/PAX7::FOXO1 variable transcripts is 1:1. Approximately 20% of cases of alveolar RMS do not have cytogenetic signs of rearrangements of the FOXO1 gene.
    Journal
    |
    MYOD1 (Myogenic Differentiation 1) • PAX3 (Paired Box 3) • PAX7 (Paired Box 7)
    1d
    Desmoplastic Small Round Cell Tumors of the Gastrointestinal Tract. (PubMed, Cancers (Basel))
    The prognosis of these tumors is highly dependent on factors such as tumor location and stage at diagnosis, and given their aggressive nature, a multidisciplinary approach may be required that combines surgical resection, chemotherapy, and radiation therapy, among other options. In this review, we provide a synopsis of the pathophysiology of DSRCTs and the latest diagnostic advancements, including the utility of molecular profiling and novel biomarkers.
    Review • Journal
    |
    WT1 (WT1 Transcription Factor) • EWSR1 (EWS RNA Binding Protein 1) • VIM (Vimentin)
    3d
    AK112-US-01: Ivocizumab in the Treatment of Multiple Advanced Tumors (clinicaltrials.gov)
    P2, N=100, Recruiting, Fudan University | Not yet recruiting --> Recruiting
    Enrollment open • Metastases
    |
    Yidafan (ivonescimab)
    4d
    RMST: A long noncoding RNA involved in cancer and disease. (PubMed, J Biochem)
    While current findings are promising, further research is needed to fully elucidate the functions and translate these insights into clinical applications of RMST. This review underscores the significance of RMST in cellular processes and disease pathogenesis, highlighting its potential as a novel target for diagnostic and therapeutic interventions.
    Journal
    |
    SOX2
    6d
    Molecular-Functional Imaging of Hypoxia in Childhood Sarcomas (clinicaltrials.gov)
    P1, N=18, Enrolling by invitation, The Hospital for Sick Children | Trial completion date: Dec 2024 --> Dec 2025 | Trial primary completion date: Dec 2023 --> Jun 2025
    Trial completion date • Trial primary completion date
    8d
    Treatment of Pediatric, Adolescent, and Young Adult Patients With Fusion-Positive Alveolar Rhabdomyosarcoma Infiltrating Regional Lymph Nodes in the European CWS-2002P and RMS 2005 Studies and the Soft Tissue Sarcoma Registry. (PubMed, Pediatr Blood Cancer)
    Patients with fusion-positive N1 ARMS carry a poor prognosis. Adequate local treatment of LN improved survival.
    Journal
    |
    FOXO1 (Forkhead box O1)
    |
    doxorubicin hydrochloride • ifosfamide • vincristine • dactinomycin
    10d
    Primary intracranial DICER1-mutant sarcoma: a clinicopathological analysis of seven cases (PubMed, Zhonghua Bing Li Xue Za Zhi)
    Primary intracranial DICER1-mutant sarcoma is a rare tumor. Understanding its morphological characteristics, immunohistochemical and molecular markers and differential diagnosis is crucial to avoid misdiagnosis and to improve diagnostic accuracy of this tumor.
    Retrospective data • Journal
    |
    TP53 (Tumor protein P53) • ATRX (ATRX Chromatin Remodeler) • DICER1 (Dicer 1 Ribonuclease III) • SALL4 (Spalt Like Transcription Factor 4)
    |
    TP53 mutation
    12d
    Schwann Cell-Mediated M2-Like Macrophage Polarization in Rhabdomyosarcoma. (PubMed, Oral Dis)
    Our study provides valuable insights into the microenvironment and immunological characteristics of RMS, offering crucial information for further research and potential therapeutic strategies.
    Journal
    |
    CD74 (CD74 Molecule)
    13d
    ARST1431: Combination Chemotherapy With or Without Temsirolimus in Treating Patients With Intermediate Risk Rhabdomyosarcoma (clinicaltrials.gov)
    P3, N=325, Active, not recruiting, National Cancer Institute (NCI) | Trial completion date: Oct 2024 --> Oct 2025
    Trial completion date
    |
    FOXO1 (Forkhead box O1) • PAX3 (Paired Box 3) • PAX7 (Paired Box 7)
    |
    cyclophosphamide • irinotecan • Torisel (temsirolimus) • vincristine • vinorelbine tartrate • dactinomycin
    14d
    Dysregulation of the p53 pathway provides a therapeutic target in aggressive pediatric sarcomas with stem-like traits. (PubMed, Cell Oncol (Dordr))
    Our results indicate that restoring canonical p53 activity provides a promising strategy for developing improved therapies for pediatric sarcomas with unfavorable stem-like traits.
    Journal
    |
    KLF4 (Kruppel-like factor 4) • SOX2
    |
    TP53 mutation • TP53 wild-type
    17d
    GEFT inhibits the GSDM-mediated proptosis signalling pathway, promoting the progression and drug resistance of rhabdomyosarcoma. (PubMed, Cell Death Dis)
    Overall, our investigation demonstrated that nigericin and dactinomycin play therapeutic roles in tumours by promoting RMS cell pyroptosis. Interference with GEFT and drug combination can exert a great inhibitory effect on tumours.
    Journal
    |
    CASP3 (Caspase 3) • NLRP3 (NLR Family Pyrin Domain Containing 3) • GSDME (Gasdermin E)
    |
    dactinomycin
    18d
    Icariin ameliorates Coxsackievirus B3-induced viral myocarditis by modulating the S100 calcium binding protein A6/β-catenin/c-Myc signaling pathway. (PubMed, Phytomedicine)
    Our findings indicate that ICA provides significant protection against CVB3 infection by modulating the S100A6/β-catenin/c-Myc signaling pathway, suggesting its potential use as a novel drug against CVB3 infection in clinical application.
    Journal
    |
    CD8 (cluster of differentiation 8) • TNFA (Tumor Necrosis Factor-Alpha) • CD4 (CD4 Molecule) • CCL2 (Chemokine (C-C motif) ligand 2) • CXCL2 (C-X-C Motif Chemokine Ligand 2) • IL1B (Interleukin 1, beta) • S100A6 (S100 calcium binding protein A6)
    20d
    Evolutionary Therapy for Rhabdomyosarcoma (clinicaltrials.gov)
    P2, N=28, Recruiting, H. Lee Moffitt Cancer Center and Research Institute | Trial completion date: Dec 2027 --> Aug 2027 | Trial primary completion date: Dec 2026 --> Aug 2026
    Trial completion date • Trial primary completion date • Metastases
    |
    FOXO1 (Forkhead box O1)
    |
    doxorubicin hydrochloride • cyclophosphamide • irinotecan • vincristine • vinorelbine tartrate • dactinomycin • Navelbine oral (vinorelbine tartrate oral)
    22d
    Malignant Mixed Mullerian Tumor (Carcinosarcoma) in the Female Genital Tract: A Retrospective Study From a Single Center in North India. (PubMed, Cureus)
    Clinical and histological features resemble high-grade endometrial carcinoma, necessitating an extensive IHC panel to narrow down differentials and confirm the diagnosis. This study highlights the histological and IHC features and emphasizes that early diagnosis plays a crucial role in disease management.
    Retrospective data • Journal
    |
    VIM (Vimentin)
    |
    VIM expression
    22d
    Flavored, Oral Irinotecan VAL-413 (Orotecan®) Given With Temozolomide for Treatment of Recurrent Pediatric Solid Tumors (clinicaltrials.gov)
    P1, N=20, Recruiting, Valent Technologies, LLC | Trial completion date: Jun 2024 --> Jun 2025 | Trial primary completion date: Jun 2024 --> Jun 2025
    Trial completion date • Trial primary completion date
    |
    temozolomide
    22d
    PEPN2112: Elimusertib for the Treatment of Relapsed or Refractory Solid Tumors (clinicaltrials.gov)
    P1/2, N=31, Active, not recruiting, National Cancer Institute (NCI) | N=23 --> 31 | Trial completion date: Dec 2024 --> Nov 2025 | Trial primary completion date: Dec 2024 --> Sep 2024
    Enrollment change • Trial completion date • Trial primary completion date
    |
    BRCA1 (Breast cancer 1, early onset) • BRCA2 (Breast cancer 2, early onset) • ATM (ATM serine/threonine kinase) • MSH2 (MutS Homolog 2) • CDK12 (Cyclin dependent kinase 12) • WT1 (WT1 Transcription Factor) • ATRX (ATRX Chromatin Remodeler) • CHEK2 (Checkpoint kinase 2) • RAD51 (RAD51 Homolog A) • FANCA (FA Complementation Group A) • POLD1 (DNA Polymerase Delta 1) • FOXO1 (Forkhead box O1) • XRCC2 (X-Ray Repair Cross Complementing 2) • ATF1 (Activating Transcription Factor 1) • PAX3 (Paired Box 3)
    |
    elimusertib (BAY 1895344)
    25d
    Dedifferentiated parosteal osteosarcoma with a novel low-grade leiomyosarcomatous and high-grade conventional osteosarcomatous differentiation: Case report and review of literature. (PubMed, Int J Surg Case Rep)
    This case presenting a distinctive pattern of dedifferentiation is, to our knowledge, the first documented case of low-grade leiomyosarcoma occurring in parosteal osteosarcoma. As it enhances the clinical understanding by broadening the range of dedifferentiated components identified in parosteal osteosarcoma and highlights the diagnostic difficulties associated with these uncommon bone tumors.
    Review • Journal
    |
    MDM2 (E3 ubiquitin protein ligase)
    27d
    Silenced long non-coding RNA RMST ameliorates cardiac dysfunction and inflammatory response in doxorubicin-induced heart failure in C57BL/6 mice via the modulation of the microRNA-10b-5p/TRAF6 axis. (PubMed, J Physiol Biochem)
    RMST silencing could attenuate inflammatory response and cardiomyocyte apoptosis and upregulate cardiac function in mice with doxorubicin-induced HF by modulating the miR-10b-5p/TRAF6 axis. The study provides novel therapeutic targets for HF treatment.
    Preclinical • Journal
    |
    TNFA (Tumor Necrosis Factor-Alpha) • RMST (Rhabdomyosarcoma 2 Associated Transcript) • MIR10B (MicroRNA 10b) • TRAF6 (TNF Receptor Associated Factor 6)
    |
    miR-10b overexpression
    |
    doxorubicin hydrochloride
    27d
    IL-18R supported CAR T cells targeting oncofetal tenascin C for the immunotherapy of pediatric sarcoma and brain tumors. (PubMed, J Immunother Cancer)
    Our study identifies the C domain of the ECM protein TNC as a promising CAR T-cell therapy for pediatric solid tumors and brain tumors. While we focus here on pediatric cancer, our work has relevance to a broad range of adult cancers that express C.TNC.
    Journal • CAR T-Cell Therapy • IO biomarker
    |
    IL18 (Interleukin 18)
    29d
    Extraosseous Ewing's Sarcoma Masquerading as Adolescent Neck Mass-a Case Report and Review of Literature. (PubMed, Indian J Otolaryngol Head Neck Surg)
    Despite the typically benign nature of extraosseous Ewing tumors, they can rarely metastasize in less than 20% of cases, as exemplified by this rare case. Accurate diagnosis requires a combined clinic-radio-immunohistochemical approach, and general practitioners should be aware of this clinical entity in neck masses considering its variable clinical presentation and poor prognosis in certain patient's population.
    Review • Journal
    |
    PAX5 (Paired Box 5)
    1m
    Intracranial solitary fibrous tumour with rhabdomyosarcomatous differentiation: A diagnostic challenge of a rare presentation. (PubMed, Int J Surg Case Rep)
    This case report presents a unique instance of an intracranial dedifferentiated SFT with rhabdomyosarcomatous dedifferentiation, highlighting the significant diagnostic challenges posed by this rare entity.
    Journal
    |
    BCL2 (B-cell CLL/lymphoma 2) • VIM (Vimentin) • STAT6 (Signal transducer and activator of transcription 6) • CD99 (CD99 Molecule) • GFAP (Glial Fibrillary Acidic Protein)
    1m
    Targeting NFE2L2/GPX4 signaling pathway: Therapeutic potential of arsenic sulfide-induced ferroptosis in combating rhabdomyosarcoma. (PubMed, Int Immunopharmacol)
    No abstract available
    Journal
    |
    GPX4 (Glutathione Peroxidase 4)
    1m
    Clinicopathological Analysis of a European Cohort of MYOD1 Mutant Rhabdomyosarcomas in Children and Young Adults. (PubMed, Pediatr Blood Cancer)
    This MYOD1 mutant cohort demonstrates increased MYOD and reduced MYF4 immunostaining, high risk of local failure and poor survival in agreement with other studies. Increased treatment intensity and improved local control should be considered for these patients.
    Journal
    |
    FOXO1 (Forkhead box O1) • MYOD1 (Myogenic Differentiation 1) • PAX3 (Paired Box 3)
    1m
    Alveolar Paratesticular Rhabdomyosarcoma in an Adult Patient With PAX3-FOXO1 Fusion and Unfavorable Evolution. (PubMed, Cureus)
    Here, We report a case of paratesticular alveolar rhabdomyosarcoma in an adult patient who initially complained of increased scrotal volume for two years and presented with a PAX3-FOXO1 fusion. This emphasizes the dire prognosis of the disease, reinforcing the need for thorough and directed diagnostic efforts.
    Journal
    |
    FOXO1 (Forkhead box O1) • PAX3 (Paired Box 3)
    |
    PAX3-FOXO1 fusion
    1m
    Patterns of expression of VEGFR2, PDGFRs and c-Kit in pediatric patients with high grade non-rhabdomyosarcoma soft tissue sarcoma. (PubMed, Front Oncol)
    Our findings indicated that tyrosine kinase receptors are upregulated in NRSTS and exhibited a distinct expression pattern within various subgroups. High expression of VEGFR2 and PDGFRα significantly correlated with reduced survival and may guide targeted therapy approaches for this poor prognosis group of patients.
    Journal
    |
    KIT (KIT proto-oncogene, receptor tyrosine kinase) • PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • KDR (Kinase insert domain receptor) • PDGFRB (Platelet Derived Growth Factor Receptor Beta)
    |
    KDR overexpression • KDR expression • KIT expression • PDGFRA expression
    1m
    Radiation Therapy to Treat Musculoskeletal Tumors (clinicaltrials.gov)
    P2, N=202, Active, not recruiting, St. Jude Children's Research Hospital | Trial completion date: Oct 2024 --> Oct 2029
    Trial completion date
    1m
    Ivocizumab in the Treatment of Multiple Advanced Tumors (clinicaltrials.gov)
    P2, N=100, Not yet recruiting, Fudan University
    New P2 trial • Metastases
    1m
    Multi-cohort, Single-arm Phase II Study of Albumin-paclitaxel, Ifosfamide, and Cisplatin in the Treatment of Rare Advanced Tumors (clinicaltrials.gov)
    P2, N=100, Not yet recruiting, Fudan University
    New P2 trial • Metastases
    |
    cisplatin • albumin-bound paclitaxel • cyclophosphamide • ifosfamide
    1m
    ARST2031: A Study to Compare Early Use of Vinorelbine and Maintenance Therapy for Patients With High Risk Rhabdomyosarcoma (clinicaltrials.gov)
    P3, N=118, Recruiting, Children's Oncology Group | Active, not recruiting --> Recruiting
    Enrollment open
    |
    FOXO1 (Forkhead box O1)
    |
    cyclophosphamide • vincristine • dactinomycin • Navelbine oral (vinorelbine tartrate oral)
    1m
    Alpha/Beta CD19+ Depleted Haploidentical Transplantation + Zometa for Pediatric Hematologic Malignancies and Solid Tumors (clinicaltrials.gov)
    P1, N=22, Recruiting, University of Wisconsin, Madison | Suspended --> Recruiting | Trial completion date: Dec 2025 --> Dec 2026 | Trial primary completion date: Dec 2024 --> Dec 2025
    Enrollment open • Trial completion date • Trial primary completion date
    |
    melphalan • zoledronic acid
    1m
    ASAP1 and ARF1 regulate myogenic differentiation in rhabdomyosarcoma by modulating TAZ activity. (PubMed, Mol Cancer Res)
    This study shows that ASAP1 and ARF1 are necessary for myogenic differentiation, providing a deeper understanding of differentiation in FN-RMS and illuminating an opportunity to advance differentiation therapy. Implications: ASAP1 and ARF1 regulate MEKi-induced differentiation of FN-RMS cells by modulating WWTR1 (TAZ) activity, supporting YAP1/TAZ inhibition as a FN-RMS differentiation therapy strategy.
    Journal
    |
    YAP1 (Yes associated protein 1) • WWTR1 (WW Domain Containing Transcription Regulator 1) • ASAP1 (ArfGAP With SH3 Domain)
    |
    RAS mutation
    1m
    Irinotecan and Carboplatin as Upfront Window Therapy in Treating Patients With Newly Diagnosed Intermediate-Risk or High-Risk Rhabdomyosarcoma (clinicaltrials.gov)
    P2, N=65, Active, not recruiting, Memorial Sloan Kettering Cancer Center | Trial completion date: Oct 2024 --> Oct 2025 | Trial primary completion date: Oct 2024 --> Oct 2025
    Trial completion date • Trial primary completion date
    |
    carboplatin • doxorubicin hydrochloride • cyclophosphamide • ifosfamide • etoposide IV • irinotecan • vincristine • Neupogen (filgrastim) • dexrazoxane
    2ms
    FollowTheDIN: Chemo-immunotherapy in Patients Under 18 Years of Age With Bone and Soft Tissue Sarcomas (clinicaltrials.gov)
    P3, N=40, Recruiting, N.N. Petrov National Medical Research Center of Oncology
    New P3 trial
    |
    carboplatin • docetaxel • temozolomide • doxorubicin hydrochloride • irinotecan • Qarziba (dinutuximab beta)
    2ms
    Palbociclib in Treating Patients With Relapsed or Refractory Rb Positive Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With Activating Alterations in Cell Cycle Genes (A Pediatric MATCH Treatment Trial) (clinicaltrials.gov)
    P2, N=23, Completed, National Cancer Institute (NCI) | Active, not recruiting --> Completed
    Trial completion • Metastases
    |
    RB1 (RB Transcriptional Corepressor 1)
    |
    Ibrance (palbociclib)
    2ms
    Chemical Carcinogen (3-Methylcholanthrene)-induced Pleomorphic Rhabdomyosarcomas in Fanconi Anemia Fancd2-/-, Fancg-/- (C57BL/6), Fancd2-/- (129/Sv) Mice. (PubMed, In Vivo)
    These transplantable FA mouse tumor cell lines should be valuable for testing effects of new radiation therapy protocols including FLASH high dose rate radiation delivery, immunotherapies, and combined radiation and chemotherapy treatments for radiosensitive FA patients.
    Preclinical • Journal • IO biomarker
    |
    HRD (Homologous Recombination Deficiency) • FANCD2 (FA Complementation Group D2) • FANCG (FA Complementation Group G)
    2ms
    Improving Adolescent and Young Adult Self-Reported Data in ECOG-ACRIN Trials (clinicaltrials.gov)
    P=N/A, N=400, Recruiting, Eastern Cooperative Oncology Group | Trial completion date: Nov 2024 --> May 2027 | Trial primary completion date: Nov 2024 --> May 2027
    Trial completion date • Trial primary completion date
    2ms
    Emerging role and function of Hippo-YAP/TAZ signaling pathway in musculoskeletal disorders. (PubMed, Stem Cell Res Ther)
    The efficacy and feasibility of Hippo pathway-related drugs for targeted therapy of musculoskeletal diseases are also discussed in our study. These endeavors offer novel insights into the application of Hippo signaling in musculoskeletal disorders, providing effective therapeutic targets and potential drug candidates for treating such conditions.
    Review • Journal
    |
    YAP1 (Yes associated protein 1) • WWTR1 (WW Domain Containing Transcription Regulator 1) • TAFAZZIN (Tafazzin)
    2ms
    Vincristine and Temozolomide in Combination With PEN-866 for Adolescents and Young Adults With Relapsed or Refractory Solid Tumors (clinicaltrials.gov)
    P1/2, N=64, Recruiting, National Cancer Institute (NCI) | Not yet recruiting --> Recruiting
    Enrollment open • Combination therapy
    |
    UGT1A1 (UDP glucuronosyltransferase family 1 member A1)
    |
    UGT1A1*28 • UGT1A1*1*1
    |
    temozolomide • vincristine • locnartecan (PEN-866)
    2ms
    Dependence of PAX3-FOXO1 chromatin occupancy on ETS1 at important disease-promoting genes exposes new targetable vulnerability in Fusion-Positive Rhabdomyosarcoma. (PubMed, Oncogene)
    We additionally show that, in some FP-RMS, KDM3A also increases PAX3-FOXO1 levels. Together, our studies illuminate mechanisms of action of the KDM3A/ETS1 regulatory module, and reveal novel targetable mechanisms of PAX3-FOXO1 chromatin complex regulation, in FP-RMS.
    Journal
    |
    FOXO1 (Forkhead box O1) • ETS1 (ETS Proto-Oncogene 1) • PODXL (Podocalyxin) • FGF8 (Fibroblast Growth Factor 8) • IL4R (Interleukin 4 Receptor) • KDM3A (Lysine Demethylase 3A) • PAX3 (Paired Box 3)
    |
    PAX3-FOXO1 fusion
    2ms
    Primary Gastric Alveolar Rhabdomyosarcoma: A Potent Mimicker of Neuroendocrine Carcinoma. (PubMed, Cureus)
    Primary gastric ARMS is an extremely rare condition. Better awareness of this entity and its similarity to NEC is necessary for appropriate diagnosis and treatment.
    Journal
    |
    NCAM1 (Neural cell adhesion molecule 1) • SYP (Synaptophysin) • OLIG2 (Oligodendrocyte Transcription Factor 2)
    2ms
    Vemurafenib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With BRAF V600 Mutations (A Pediatric MATCH Treatment Trial) (clinicaltrials.gov)
    P2, N=4, Active, not recruiting, National Cancer Institute (NCI) | Trial completion date: Sep 2024 --> Sep 2025
    Trial completion date • Metastases
    |
    BRAF (B-raf proto-oncogene)
    |
    BRAF mutation • BRAF V600
    |
    Zelboraf (vemurafenib)
    2ms
    Embryonal rhabdomyosarcoma of prostate combined with prostatic abscess in an adult patient: a case report and literature review. (PubMed, Transl Androl Urol)
    Due to its non-specific presenting symptoms, prostate ERMS can be easily misdiagnosed, which can lead to treatment delay. Multimodality approaches, including neoadjuvant/adjuvant chemotherapy, radiotherapy, and radical resection, can improve the survival rate and reduce ERMS patient mortality.
    Review • Journal
    |
    CRP (C-reactive protein)