Pre-clinical testing of a new treatment approach for rhabdomyosarcoma

01 June 2012
Dr Janet Shipley, The Institute of Cancer Research, London

Around 70 children are diagnosed with rhabdomyosarcoma every year in the UK. It is a cancer that is difficult to treat and remains a major cause of death from cancer in childhood, with little progress having been made in recent decades. The aim of this project is to take forward a new approach to the treatment of this disease.

Amount of grant: £113,684*  |  Date of award: June 2012

The team
Dr Janet Shipley & Professor Andrew Pearson, The Institute of Cancer Research; Dr Julia Chisholm & Dr Suzanne Gatz, The Royal Marsden Hospital.


Rhabdomyosarcoma (RMS) is the most common type of soft tissue sarcoma to occur in childhood, with around 70 new cases every year in the UK.

RMS resembles primitive muscle cells and can occur in almost any part of the body but most commonly develops in the head and neck, the bladder or the testes.

RMS is a difficult cancer to treat, particularly in children with disease that has recurred or has spread to other parts of the body. There has been no significant increase in survival in the last three decades, despite rigorous clinical trials.

Attention has recently focused on a type of protein known as Fibroblast Growth Factors (FGFs). FGFs are involved in various cell functions and their actions are mediated by FGF receptors on the surface of cells.

Clinical investigations in a range of adult cancers, looking at the effects of blocking these receptors, using drugs known as FGFR inhibitors have shown great promise. No clinical testing in children has so far been carried out.

Project description

Dr Janet Shipley is the Team Leader of the Sarcoma Molecular Pathology Team at the Institute of Cancer Research in London. She is an internationally recognised expert in paediatric RMS and is working closely with clinical colleagues at the Royal Marsden Hospital and national and European RMS study groups to take this work forward.

Work carried out in Dr Shipley’s laboratory suggests that FGF receptors are a promising treatment target in RMS. Pharmaceutical companies have drugs that inhibit FGF receptors – known as FGFR inhibitors - at various stages of pre-clinical and clinical development. The project team has carefully selected four such FGFR inhibitors to undergo pre-clinical investigation in laboratory models of RMS. This work will enable them to select the best, most appropriate inhibitor to take forward to clinical trial in children.

 ‘Dr Shipley is an internationally recognised expert in paediatric rhabdomyosarcoma and has assembled an outstanding research team.’ Independent reviewer.

What difference will this project make?

No real improvement in survival rates for RMS have been seen in the last three decades and new treatment approaches are desperately needed.

This work will enable Dr Shipley to select and take forward a new drug into clinical trials for testing in young patients with RMS. Ultimately, the hope is that this work will result in the adoption of a new, more effective drug into the standard treatment of patients with RMS to save patients that are failed by existing therapies.

* this project is being funded in collaboration with Great Ormond Street Hospital Children’s Charity, with each charity contributing 50% of the total cost of £227,368.

Read more: Soft tissue sarcomas | Patient story, Ross Anderton

Where your money goes

Hover over a segment for details