Alice’s Arc invests a further £130K for a work package relating to the development of a CAR T-cell treatment for rhabdomyosarcoma

Professor Janet Shipley and Dr Stella Man from the Institute of Cancer Research will work with Dr Karin Straathof at UCL Great Ormond Street Institute of Child Health. This new work package forms a critical part of the overall research project ‘Identification and evaluation of foetal immune targets in rhabdomyosarcoma.’ It builds upon the work of Dr Sam Behjati, Wellcome Sanger Institute, analysing RMS cells and how they differ from normal cells

The overall goal of this work is to ‘progress in silico findings into experimental models leading towards clinical relevance.’ This analysis of developmental targets, combining data analysis with experimental work in model systems, may reveal novel avenues for RMS treatment. The foetal roots of RMS biology remain relatively unexplored so there is a real opportunity here, which the team at the ICR are determined to harness. This approach may also have impact and application beyond RMS by defining a blueprint for similar work into other childhood cancers.

The targets identified by Dr Karin Straathof and team will now be explored in model systems of RMS organised and grown by Dr Stella Man at the ICR. Model systems will include those derived from tumour samples from patients after growing them in mice (called patient-derived xenografts (PDX)) and cell lines that have been developed to be resistant to standard treatment, or genetically engineered to switch the fusion gene ‘on’ or ‘off’.
Research questions
  • What are the processes driving tumour progression?
  • What characteristics do cells have that make them resistant to current treatments?
  • What are the differences between cells at diagnosis and relapse?
  • How do the molecular profiles of PDX cells models compare with those obtained directly from patient tumours
  • What molecular features can we therapeutically target?


How will the funds (£130K) from Alice’s Arc be used?

The funds will be used to fund the salary of Dr Stella Man and associated consumables required. Stella will focus on the preparation of RMS models and the sequencing analyses with Dr Karin Straathof. The molecular targets identified from the analysis of the sequencing data will be tested in the RMS models.

Project partners

This project builds on a collaboration initiated with an Innovation Award from Cancer Research UK in conjunction with Children with Cancer UK.


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