(Rhabdomyo)sarcoma

Soft-tissue sarcomas are rare cancers arising in connective tissues of the body and often have a poor prognosis. Despite surgical, radiotherapeutic- and/or systemic treatment of high-grade sarcomas, more than half of the patients relapse in distant organs within two years of initial diagnosis. Because of the rarity and heterogeneity of the disease, setting up large clinical trials for drug testing is difficult. Advanced genetic analyses of sarcomas have so far not been able to identify upfront biomarkers predictive of treatment. Patients with inoperable, metastatic or high-risk disease will receive systemic treatment, but upon disease progression or relapse, there are currently no standardized therapeutic approaches available. Treating physicians, together with patients, currently must choose from a number of chemotherapy regimens based on scarce studies available with no guarantee this choice will be effective in a specific patient. Often consecutive regimens of chemotherapy, each with severe treatment related toxicities, are explored and their effectiveness can only be evaluated after several months of treatment. This iterative process poses a significant burden to the patient, suffering from unwanted treatment side-effects. Rhabdomyosarcoma, one of the more common subtypes, has a survival rate below 30% in patients with metastatic disease. Therefore, we aim to advance targeted therapy options for pediatric (rhabdomyo)sarcoma.