Is the activation of AMPK a potential treatment of Duchenne muscular dystrophy?

Professor Dominic Wells at the Royal Veterinary College is investigating whether activating AMPK, an enzyme that plays a role in cell health, has a beneficial effect in dystrophic muscle. The findings from this study will determine if activating AMPK is a suitable approach for the treatment for Duchenne muscular dystrophy.

“This project will provide essential data to confirm whether AMPK activation could actually be a suitable therapy for Duchenne and related muscular dystrophies.”

What are the aims of the project?

AMPK is an enzyme that is present in all of our cells and plays an important role in maintaining the health of a cell. Therefore activating AMPK could be beneficial for dystrophic muscle. Previous studies have shown promising results from pharmacologically activating the AMPK enzyme in mdx mice, a mouse model for Duchenne muscular dystrophy. However, these studies used non-specific drugs, which may have acted on multiple enzymes, including AMPK. Therefore it is not clear if the beneficial results were obtained through increasing the activity of AMPK alone.

The aim of this project is to clarify whether the activation of AMPK has a beneficial effect in dystrophic muscle. Professor Dominic Wells and his team will do this by studying a new mouse model that lacks dystrophin and has an over-active AMPK enzyme. They will evaluate muscle health and function in these mice and compare them to control mice that lack dystrophin but have normal AMPK activity.

Why is this research important?

Drug development is a lengthy and expensive process, so it is vital for scientist to pinpoint and concentrate on targets that are likely to provide a benefit. It is currently not clear whether AMPK is a therapeutic target for Duchenne muscular dystrophy. This project will help clarify this, and will also improve our understanding of the role of AMPK in muscle.

How will the outcomes of this research benefit people with DMD?

Should Professor Wells find that AMPK is a suitable therapeutic target, he will be able to collaborate with pharmaceutical companies to investigate drugs that specifically activate AMPK. This work could lead to preclinical studies and potentially future clinical studies.

How might this research impact on other neuromuscular conditions?

AMPK activation could potentially be protective for dystrophic muscle in general. Therefore this project could have important implications for other muscular dystrophies.

Grant information

Project leader: Professor Dominic Wells
Institute: Royal Veterinary College
Condition: Duchenne muscular dystrophy
Duration: 1 year, starting 2017
Total cost (£): £23,773
Official title: AMPK activation for the treatment for Duchenne muscular dystrophy

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