MISSION

EORTC has been involved in a number of large practice-changing clinical trials in the field of Sarcoma, leading to the registration of drugs to treat sarcomas including gastrointestinal stromal tumors (GIST).

PRACTICE CHANGING RESEARCH

Understanding Sarcomas: 

Sarcomas, a rare group of cancers affecting soft tissue and bone, present diagnostic and treatment challenges due to their low global incidence and limited research interest. There are over 70 forms of sarcoma, complicating efforts to enhance care. Pharmaceutical companies’ reluctance to invest in new drugs and limited funding sources worldwide add to the challenge. Treatment options have historically been limited to chemotherapy, with poor outcomes for patients in advanced stages.

EORTC researchers have made significant contributions in the past two decades, leading to the development of targeted therapies for sarcomas, including gastrointestinal stromal tumours (GIST). The following compounds were investigated within this group (STBSG):

Imatinib for gastrointestinal stromal tumour (GIST)
Study Coordinator: Prof Jaap Verweij		 A breakthrough treatment for inoperable patients and/or patients with advanced disease. The EORTC STBSG helped to show the benefit of imatinib for patients, and to establish the different dosing regimens. Imatinib received worldwide approval for the treatment of adult patients. The availability of imatinib dramatically changed the course of this disease by prolonging life.14 15 16 17
Trabectedin for advanced soft tissue sarcoma
Study Coordinator: Prof Axel le Cesne		 The results of the EORTC Phase 2 study using trabectedin at 1.5 mg/m² regimen contributed to the accelerated approval* of trabectedin by the European Union for advanced soft tissue sarcoma and then other countries. 18

*Approvals obtained by both the European Medicine Agency (EMA) and the Food and Drug Administration (FDA) in the USA
Pazopanib for certain forms of soft-tissue sarcoma
Study Coordinator: Prof Winette van der Graaf		 The results of the EORTC Phase 3 Palette trial led to worldwide approval of pazopanib as a new treatment option for certain forms of soft-tissue sarcoma, at an advanced stage of the disease that was previously treated with chemotherapy. 19 20
Eribulin for certain forms of soft-tissue sarcoma
Study Coordinator: Prof Patrick Schöffski		 The EORTC phase 2 trial showed promising outcomes for certain forms of sarcoma at an advanced stage. These results provided sufficient evidence to proceed with a practice-changing trial, ultimately leading to the worldwide approval of the drug. 21
Crizotinib in several types of rare cancers
Study Coordinator: Prof Patrick Schöffski		 The CREATE trial of crizotinib included patients suffering from advanced rare cancers (including certain rare forms of soft-tissue sarcoma) carrying certain biological alterations called ALK or MET and showed a benefit of the treatment for some forms of soft-tissue sarcoma. 22 23 24 25

14 Van Oosterom, A. T. et al. Safety and efficacy of imatinib (STI571) in metastatic gastrointestinal stromal tumours: A phase I study. Lancet 358, 1421–1423 (2001).

15 Verweij, J. et al. Imatinib mesylate (STI-571 Glivec, Gleevec) is an active agent for gastrointestinal stromal tumours, but does not yield responses in other soft-tissue sarcomas that are unselected for a molecular target: Results from an EORTC Soft Tissue and Bone Sarcoma Group phase II study. Eur J Cancer 39, 2006–2011 (2003).

16 Zalcberg, J. R. et al. Outcome of patients with advanced gastro-intestinal stromal tumours crossing over to a daily imatinib dose of 800 mg after progression on 400 mg. Eur J Cancer 41, 1751–1757 (2005).

17 Verweij, J. et al. Progression-free survival in gastrointestinal stromal tumours with high-dose imatinib: Randomised trial. Lancet 364, 1127–1134 (2004).

18 Sleijfer, S. et al. Pazopanib, a multikinase angiogenesis inhibitor, in patients with relapsed or refractory advanced soft tissue sarcoma: a phase II study from the European organisation for research and treatment of cancer-soft tissue and bone sarcoma group (EORTC study 62043). J Clin Oncol 27, 3126–3132 (2009).

19 Van Der Graaf, W. T. A. et al. Pazopanib for metastatic soft-tissue sarcoma (PALETTE): a randomised, double-blind, placebo-controlled phase 3 trial. Lancet 379, 1879–1886 (2012).

20 Schöffski, P. et al. Activity of eribulin mesylate in patients with soft-tissue sarcoma: A phase 2 study in four independent histological subtypes. Lancet Oncol 12, 1045–1052 (2011).

21 Schöffski, P. et al. Long-term efficacy update of crizotinib in patients with advanced, inoperable inflammatory myofibroblastic tumour from EORTC trial 90101 CREATE. Eur J Cancer 156, 12–23 (2021).

22 Schöffski, P. et al. Crizotinib achieves long-lasting disease control in advanced papillary renal-cell carcinoma type 1 patients with MET mutations or amplification. EORTC 90101 CREATE trial. Eur J Cancer 87, 147–163 (2017).

23 Schöffski, P. et al. The tyrosine kinase inhibitor crizotinib does not have clinically meaningful activity in heavily pre-treated patients with advanced alveolar rhabdomyosarcoma with FOXO rearrangement: European Organisation for Research and Treatment of Cancer phase 2 trial 90101 ‘CREATE’. Eur J Cancer 94, 156–167 (2018).

24 Schöffski, P. et al. Crizotinib in patients with advanced, inoperable inflammatory myofibroblastic tumours with and without anaplastic lymphoma kinase gene alterations (European Organisation for Research and Treatment of Cancer 90101 CREATE): a multicentre, single-drug, prospective, non-randomised phase 2 trial. Lancet Respir Med 6, 431–441 (2018).

25 Schöffski, P. et al. Activity and safety of crizotinib in patients with advanced clear-cell sarcoma with MET alterations: European Organization for Research and Treatment of Cancer phase II trial 90101 ‘CREATE’. Annals of Oncology 28, 3000–3008 (2017).

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