DMD research

Disappointing findings in anti-inflammatory drug repurposing project

Duchenne UK is disappointed to announce that a project investigating the potential of montelukast, an existing anti-inflammatory drug for asthma, to treat DMD failed to show benefit in a pre-clinical study.

In 2017 we funded Drs. Dongsheng Duan and Bob McDonald from the University of Missouri to evaluate the use of montelukast, as a potential repurposed therapy for Duchenne muscular dystrophy. 

Drug repurposing can be an effective way to develop a drug with established safety data for one disease for another disease, minimising the time it takes to reach to clinical trials.

Montelukast, which is licensed as a treatment for asthma, is a type of medicine known as a leukotriene receptor antagonist.  It works by blocking the action of leukotrienes, which are chemicals released by the body as part of inflammatory reactions. 

The three-year programme we funded was aimed at evaluating the therapeutic efficacy of montelukast in young adult mdx mice, the most used mouse model for DMD. Given the current standard of care for DMD is corticosteroid, the project also evaluated montelukast in combination with prednisolone (a type of corticosteroid), to see if these together had a stronger therapeutic effect. The programme, which was due to complete through 2020, was initially delayed by significant technical challenges associated with the formulation of the drug and its delivery in the animal model, and subsequently by the COVID-19 pandemic.

Unfortunately, despite montelukast being an effective anti-inflammatory drug in asthma, in this initial research in DMD, it was not found to reduce muscle disease in mdx mice. This could be due to several factors, including the age of the mice used in the study, the duration of treatment, or the possibility that the biological pathway regulated by montelukast may not be a primary contributor to muscle inflammation in DMD.  At this stage, we can only conclude that within the conditions used in this study that a significant beneficial effect was not observed to justify further support from Duchenne UK.

The researchers at University of Missouri are planning more work in mice models of DMD to further elucidate the findings of the project.

We are sharing this disappointing news in the interests of openness and in the hope that all knowledge – whether the results are positive or not – can help progress understanding and the search for effective treatments in DMD.

Published on 7 March 2022

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