New drug to be tested for use in the muscular dystrophies

Published Date
Julia Ambler

Researchers at the Royal Veterinary College, University of London have received a grant from the Medical Research Council to investigate an experimental drug for its ability to prevent the formation of scar tissue in muscular dystrophy.

Prof Dominic Wells, and Dr Susan Brown, at the Royal Veterinary College in London recently received a grant from the Medical Research Council (MRC) as part of an innovative scheme that saw the MRC partnering with pharmaceutical company AstraZeneca. AstraZeneca made 22 of its chemical compounds available free-of-charge to scientists, who were encouraged to apply for MRC funding to use them in medical research with the ultimate aim of benefiting patients. AstraZeneca had conducted early trials of these compounds and validated their use for future research, but had put them on hold for further development. The aim of this partnership was to extend the possible application of these compounds for use in new areas.

Prof Wells and Dr Brown will be testing a drug called AZD1236 for its ability to prevent scar tissue formation in muscular dystrophy.  AZD1236 was designed to block the activity of an enzyme called matrix metallopeptidase-9 (MMP-9) which is known to be involved in the formation of scar tissue. In the muscular dystrophies, there is a gradual breakdown of the muscle which leads to muscle weakness.  As the muscle breaks down it is replaced by fat and scar tissue further reducing the ability of the muscles to work properly. It is this process that the researchers hope to slow using AZD1236.

The researchers will use the drug, originally developed by AstraZeneca to treat a type of lung disease known as chronic obstructive pulmonary disease, in two different mouse models of muscular dystrophy – Duchenne muscular dystrophy and limb girdle muscular dystrophy to see if they can slow progression of the disease. If successful, the study will provide the evidence they need to plan human trials of the drug to test whether it has a benefit for individuals with muscular dystrophy.

Prof Dominic Wells said,

This novel collaboration between the MRC and AstraZeneca allows unprecedented access to drugs that have previously been tested in man. In our case, if the MMP-9 inhibitor proves effective in mouse models of muscular dystrophy, this drug can be fast-tracked into clinical trials.

Further information and links

Find out more about the partnership between the MRC and AstraZeneca

Read about the research we are funding in Prof. Wells’ and Dr. Brown’s laboratories


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