Professor Dame Kay Davies at Oxford University is aiming to identify biomarkers that can monitor the effectiveness of utrophin up-regulators. These will provide a more objective measure for future clinical trials and will also help to accelerate the development of utrophin up-regulators.
What are the aims of the project?
Utrophin is a protein which is similar to dystrophin and is naturally found at very low levels in the body. Increasing the amount of utrophin can compensate for the lack of dystrophin in people with Duchenne and Becker muscular dystrophies.
This PhD studentship project fits into a larger research programme focussed on the identification of small molecules that can increase the amount of utrophin in the body (called utrophin up-regulators). One of these is currently in clinical trial for people with Duchenne muscular dystrophy (see our Breaking Research News for more information on Ezutromid).
This particular project aims to identify biomarkers that will assist in evaluating the effectiveness of utrophin up-regulators in clinical trials. The researchers will assess what molecular changes occur when utrophin levels are increased in mouse models of the condition. This will allow them to identify molecules which could serve as potential biomarkers.
Why is this research important?
At the moment, when a new treatment is being tested, researchers use a variety of ways to measure whether the treatment has had a positive effect. However, these measures suffer from limitations and are not always very good at showing small changes and improvements in symptoms. There is a need for more objective measures such as biomarkers, which this project will help to identify.
How will the outcomes of this research benefit patients?
Biomarkers identified from this research will help to assess the effectiveness of utrophin up-regulators, therefore accelerating their development. This could benefit all people with Duchenne muscular dystrophy, regardless of their genetic mutation, as well as people with Becker muscular dystrophy. Identifying non-invasive biomarkers would provide even greater benefits, as there would be no need to take muscle biopsies from people receiving the utrophin up-regulator.
How might this research impact on other neuromuscular conditions?
This project may provide fundamental insights into what is going on at the molecular level inside muscles that are wasting (dystrophic muscle). This knowledge could improve understanding of muscular dystrophies in general, which could be important in the development of future treatments.
Project leader: Professor Dame Kay Davies
Institute: Oxford University
Condition(s): Duchenne & Becker muscular dystrophies
Duration: fours years, starting 2016
Total cost: £115,671
Official title: Biomarkers in utrophin modulation therapy development
Find out more about the importance of biomarkers
Read about our other Duchenne muscular dystrophy research projects
Read our research news stories on Duchenne muscular dystrophy
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