Blood-based biomarker

Developing a more informative measure of disease progression and response to new treatments

Although Duchenne has been studied for many years, classic measures of disease progression rely on basic observations of movement, such as the six-minute walk test. These measures pose a significant challenge for researchers because incremental disease improvement may go unnoticed. In addition, these measures don’t apply to non-ambulant patients, who may benefit from new treatment candidates, and who are currently not eligible for clinical trials.

With the funds raised in 2016, we are funding a project that involves analysing blood-based samples with the aim of establishing molecular signatures that correlate with stages of Duchenne, which can then be measured through blood tests. If successful, this technique may provide an alternative, more informative measure of therapeutic efficacy in clinical trials. The project is being managed by Solid Biosciences.

Blood samples

Pre-clinical research

£140,000 contribution

Funded in 2016-17

Cambridge, MA, USA

What is a biomarker?

In medicine, a biomarker is a biological feature that can be used to measure the presence or progress of a particular disease. Biomarkers can be used to assess the effectiveness of a particular treatment in helping to alleviate the effects of a disease. We are helping fund development of new biomarkers for Duchenne that aim to monitor the progression of Duchenne more effectively and measure patients’ response to new treatments in clinical trials.

Why are we supporting this project?

Despite the significant acceleration of therapeutic developments in the field of Duchenne in the last few years, a number of issues remain to be solved and require dedicated support and attention. We need better and more reliable outcome measures for boys with Duchenne, which is an area that has been insufficiently explored in the field so far. Characterisation of better outcome measures will enable the field to monitor disease progression and benefits of any therapeutic strategies in a more coherent and reliable manner. The newly funded projects supported by the Duchenne Research Fund will address those shortcomings and I am confident that the successful completion of those will make seminal contributions to the field of Duchenne.

Professor Ronald Cohn

Head of the DRF Scientific Advisory Board; Chair, Department of Paediatrics, University of Toronto; Paediatrician-in-Chief, The Hospital for Sick Children, Toronto

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