New treatments for relapsed rhabdomyosarcoma

Project title: Novel models and new treatments for relapsed rhabdomyosarcoma patients

Funded by CCLG and CCLG Special Named Funds including Angus’ Door, The Jenni Clark Fund, Ollie’s Star, Team Jake, Pass The Smile for Ben, Jacob’s Join, Hattie’s Rainbow of Hope Appeal and Be More Ruby
Lead investigator: Professor Janet Shipley, The Institute of Cancer Research
Award: £99,398.00  
Awarded December 2021

Of all the children diagnosed with rhabdomyosarcoma, a third will have their cancer spread or return after their initial treatment (otherwise known as a relapse). Unfortunately, these patients often have a low chance of survival and need more effective treatments for their relapsed rhabdomyosarcoma.  

Scientists use disease models to assess whether new treatments work or to develop new treatments. For rhabdomyosarcoma, these models might be the same as the tumour genetically, physically or experimentally. This study aims to develop and test new models of rhabdomyosarcoma in order to find better treatments for relapsed patients. The researchers will use tumour samples taken from relapsed patients in European countries enrolled onto the existing FaR-RMS international clinical trial. Cancer cells will be grown flat in petri dishes and in 3D, as well as in zebrafish, as models of relapsed rhabdomyosarcoma.  

These models will be used to screen and test available drugs, such as agentregorafenib, which is being introduced for relapsed patients into the FaR-RMS trial. A new drug called regorafenib will be used alongside traditional chemotherapy in the new models at the same time as they are being used to treat patients. This means that the models’ reaction can be compared to patients’ tumours’ reactions so that the researchers can see which models are most like real tumours. 

This research team, led by Professor Janet Shipley, will try to identify potential treatments for further investigation in the models. Together with molecular characterisation of these models in a parallel project, this will provide better understanding of the biology of relapsed RMS that respond to particular drugs. From the models, tumour biomarkers can be found that show how a tumour is responding to treatment. New treatments identified for children with relapsed rhabdomyosarcoma could also be introduced into the FaR-RMS trial.