Liquid biopsies for rhabdomyosarcoma: A new way to improve rhabdomyosarcoma treatment

Project title: Prospective assessment of nucleic acids in liquid biopsies from frontline international rhabdomyosarcoma trial patients to determine clinical utility

Funded by The Little Princess Trust and administered by CCLG
Lead investigator: Professor Janet Shipley, The Institute of Cancer Research
Award: £210,885.00
Awarded July 2022

Rhabdomyosarcoma is the most common soft tissue cancer in children and young adults. Whilst most rhabdomyosarcoma patients can be successfully treated with chemotherapy, radiotherapy and surgery, there are many children whose disease remains or returns after treatment. Sadly, many of these patients do not survive.

Pieces of DNA can be released from cancers into the bloodstream. Work in other cancers and in small studies for rhabdomyosarcoma have shown that liquid biopsy blood tests looking for specific pieces of DNA, called markers, can indicate how a child’s cancer is responding to treatment or show if it has relapsed. To find out what works best for rhabdomyosarcoma patients, a clinical trial is needed. It will help discover the answers to questions such as how often liquid biopsies should be done and how best to test for the pieces of DNA.

Professor Janet Shipley and her team at the Institute of Cancer Research will use blood samples already collected as part of the ongoing FaR-RMS international clinical trial. They will look for genetic changes in samples from newly diagnosed RMS patients to see if there are any that could be used as markers or that play a role in disease progression. The team will then measure the amounts of DNA pieces in patients’ blood samples as they go through treatment as part of the FaR-RMS trial. They will use these measurements to see if the blood tests can show when treatments aren’t working, or when a patient has relapsed.

The researchers will be working with specialist centres in Europe using state-of-the-art technologies but will analyse the blood samples in their UK lab. They will have access to patients’ clinical information and scans so that they can hopefully identify patients who are having problems earlier than normal, without the need for multiple surgeries and scans.

Professor Shipley hopes that this research could help reduce healthy tissue damage when treatments aren’t also killing the cancer cells and create options for earlier interventions with more effective and personalised treatments.