New Research Project Funded: Stratified Medicines of the Future

Last summer, SRUK announced a new grant call ‘Stratified Medicine of the future’ to be delivered in partnership with the World Scleroderma Foundation.  This grant call was a winner-takes-all competition which would award £100,000 of funding to an UK/ International project team allowing them to test their scientific concept and acquire data which could be used to further develop larger funding applications which would see their idea being translated to the clinic. Today the winning team will be announced at the 7^th World Scleroderma Congress.

What is patient stratification and how can it help those with systemic sclerosis?
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.

Stratified Medicine is an approach used to divide a group of patients with a particular medical condition into ‘subgroups’ based on their disease subtype or their likelihood to respond to a particular therapy. It aspires to direct patients to the best available treatment, one which is most likely to work in controlling their condition with the least side effects.  When a method of stratifying patients has been developed, it ensures patients get the right treatment at the right time.  

Can you imagine a future where we will be able to eliminate trial and error in prescribing medications by predicting the best treatment for a person’s scleroderma before they even receive it!

The winning proposal of the SRUK/ WSF Stratified Medicine of the Future Award aspires to make this a reality.  In their one-year project titled ‘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.’ Professor Jorg Distler and Dr Andrea-Hermina Györfi from the University Hospital Erlangen-Nürnberg, together with Professor Chris Denton from the Royal Free Hospital in London will do just this.

The patient experience of scleroderma is very unique. Patients differ through the organs affected down to unseen molecular differences which may determine how a person will respond to various treatments. This coupled with the fact there is no short term measure which can tell a clinician whether a treatment is working for a patient. This is often judged by studying the patient looking for signs of improvement or progression through a trial and error. A patient might be prescribed several drugs before arriving at what might be considered to be the ‘right’ treatment.   

Professor Distler and his team are aiming to break down these two barriers to allow patients to receive the optimal treatment quicker. In their project they will develop a novel technique to allow the ‘molecular stratification’ of patients to their most appropriate therapy by looking at molecular markers of ‘early treatment response’.  To do this they will use a system they are developing which uses small slices of a patient’s skin derived from skin biopsy and exposing these slices in the lab to scleroderma treatment candidates. Through using molecular biology techniques and machine learning the team will develop a scoring system which will be able to predict the likelihood of an individual patient responding to a specific treatment.

This test is expected to give an answer on which treatment is best for the patient in less than a week meaning that if this research is successful and can be translated to clinical practice a clinician might in future be able to give a patient a treatment which he or she knows will work. The team also hope that their ‘toolkit’ will allow patients to be better selected to clinical trials of new treatments.

We wish the project team all the best with their exciting project and hope to update you as this work progresses!

If you’d like to read about other work that SRUK have funded, take a look at our Recently Funded Projects | SRUK page!