25 June 2021

International project Alice's Arc jointly funded uncovers genetic changes underlying the development of rhabdomyosarcoma

The work has identified genetic changes that link to the aggressiveness, early age onset and location in the body.

We jointly funded an international project for the ICR in 2018 and here are the exciting findings. The ICR discovered a series of genetic changes underlying the development of rhabdomyosarcoma which link to the aggressiveness, early age of onset and location in the body. The researchers analysed DNA from 641 patients and found:

- in fusion-negative rhabdo children whose tumours had faults in the genes MYOD1 and TP53 had a poorer response to treatment.

- in fusion positive rhabdo children faults in the genes TP53, CDK4 and MYCN were linked to a poorer outcome.

The implications of this research will lead to changes to the current system of categorising child to the existing risk categories. It also may lead to the development of new drugs to treat these specific gene faults.

Please read the attached link to Health Europa who reported the story. The findings were published in the Journal of Clinical Oncology and news coverage also appeared in Pharma Times, Yahoo News and the Yorkshire Post.

Many thanks to all our supporters who have enabled this research to forge ahead. This is what it's all about. Making the changes needed to help children in the future.