A molecular approach to predict recurrence or spread of cancer that will lead to improving outcomes for children with rhabdomyosarcoma

Project title: MG-fnRMS and CINSARC gene expression signatures to predict relapse in fusion gene negative rhabdomyosarcomas: assessing an approach to improve patient outcomes

Funded by CCLG and CCLG Special Named Funds Angus' Door, Jacob's Join, Pass the Smile For Ben Crowther, Ollie's Star, Super Ruby's Rhabdo Raiser, Team Jake and a donation from Elliott's Trust
Lead investigator: Prof Janet Shipley, The Institute of Cancer Research
Award: £99,864
Awarded December 2020

Rhabdomyosarcomas are major cause of death from cancer in childhood. Whilst around two-thirds of young people with rhabdomyosarcoma survive, the treatments often have long-lasting toxic effects on quality of life and the remaining third lose many years from their expected lifespan.

Two molecular tests show potential to predict rhabdomyosarcoma recurrence or spread:

  1. MG-fnRMS test: We previously identified a set of 5 genes, called MG-fnRMS, through analyses that showed a difference in rhabdomyosarcoma samples from patients who subsequently did or did not relapse. Analysis of a relatively small number of rhabdomyosarcoma patient samples with MG-fnRMS showed promise but requires validation.
  2. CINSARC test: A set of 67 genes, called CINSARC, derived from adult sarcomas can be analysed to predict outcome in different types of adult sarcomas and also other types of adult cancers. Due to molecular similarities between adult sarcomas and childhood rhabdomyosarcomas, we propose to assess this test for the first time in rhabdomyosarcomas.

We will use these tests on a large series of samples from rhabdomyosarcoma patients treated on previous clinical trials to assess how well, together with other factors, they can predict relapse and poor outcome. The best will be taken forward into testing patients in the newly established Front-line and Relapse-RhabdoMyoSarcoma (FaR-RMS) international clinical trial through the European paediatric Soft Tissue Sarcoma Study Group (EpSSG).

Better prediction of disease recurrence will enable sparing future low-risk rhabdomyosarcoma patients unnecessary treatment related toxicities. High-risk rhabdomyosarcoma patients may ultimately benefit from closer monitoring, more intense or new and more targeted cancer treatments that may prevent or effectively treat recurrence or disease spread. An exciting future prospect is to base new targeted therapeutic strategies on our molecular understanding of the roles for genes in the most successful test. Together, this is anticipated to improve outcomes for children with rhabdomyosarcoma.

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