Grant Call Summary

Scleroderma and Raynaud's UK (SRUK) is the only UK charity dedicated to improving the lives of people with scleroderma and Raynaud's phenomenon. We envision a world where no-one has their life limited by scleroderma and Raynaud's. The World Scleroderma Foundation (WSF) promotes and supports global research into all aspects of scleroderma and is committed to improving the quality of life for scleroderma sufferers and their families.  

SRUK and WSF are pleased to work together once again to promote innovative and high-quality research by new and established investigators in fields related to scleroderma. 

The focus of this joint call for grants is Stratified Medicine of the Future. Systemic sclerosis is a highly complex and heterogeneous condition in terms of clinical presentation, disease progression, organ involvement and patient outcomes. On an individual level, this complexity presents a challenge both in predicting disease progression and prognosis. This should ensure that patients have timely access to the most effective treatments before irreversible organ damage has occurred. On a wider level, the ability to stratify patients is therefore of intrinsic value in the design of clinical studies and trials to minimise ambiguity in treatment effects and demonstrate the efficacy of tailored treatments for SSc.  

This £100,000 ‘winner-takes-all’ award will pump-prime research into future methods of patient stratification, enabling the winner to develop preliminary data or concepts which could be used to support larger consortia-based applications.  

Project Title: Advanced in vitro test systems with integrated multi-“OMICS” to define pathway activation and treatment response scores for patient stratification towards personalised medicine in systemic sclerosis.

• Lead Investigator: Professor Jörg Distler
• Co-Investigators: Professor Christopher Denton (University College London), Dr Andrea-Hermina Györfi (University Hospital Erlangen-Nürnberg)
• Lead Institution: University Hospital Erlangen-Nürnberg
• Amount Funded: £97,600
• Research Strategy Area: Precision Medicine

Project summary

Systemic sclerosis is a highly complex and heterogeneous condition in terms of clinical presentation, disease progression, organ involvement and individual patient outcomes. This complexity presents a problem both in assigning patients to the ‘best treatment for them’ a process which is often trial and error involving lengthy follow up to arrive at the appropriate therapeutic.

This project will develop a novel in vitro toolset which will allow the molecular stratification of patients to their most appropriate therapy by looking at markers of early treatment response. This novel system will involve in vitro exposure of small slices of patient skin derived from biopsy to treatment candidates and will combine molecular screening, biostatistics and machine learning to generate scores which predict the likelihood of an individual patient responding to a specific treatment.