Developing New Chemical Compounds to Target DMD

Our Partnership

This research, headed by Professor Dame Kay Davies of Oxford University, aimed to find chemical compounds which could increase levels of a protein called utrophin. It is especially important, as this type of treatment could help all people with Duchenne muscular dystrophy, whatever their mutation.

We also co-funded a project working in the same area, led by Dr Angela Russell from Professor Davies' team.

What Is Utrophin?

Utrophin is a protein found in adult muscle, which is similar to the dystrophin protein lacking in those with DMD. The hope is that increasing its levels might compensate for the lack of dystrophin and improve muscle function. Professor Dame Kay Davies has already developed one drug with this same aim, SMT C1100, which has been successful in a Phase Ib clinical trial. This project aims to discover new follow-on compounds that can increase utrophin levels more effectively than SMT C1100.

Professor Davies, Professor Angela Russell and their team have created a new, more sensitive drug screen to search for these potential therapeutic compounds. New compounds have been identified that increase levels of utrophin in muscle cells grown in the lab. 

What Is Happening About Clinical Trials?

The aim is to find new chemical compounds which increase the levels of utrophin in a mouse model of Duchenne muscular dystrophy. If this proves successful, these compounds will be developed for clinical trials on humans, as has been achieved with SMT C1100.

The projects were funded through the Duchenne Forum. The other members of the Duchenne Forum are MDUK, The Duchenne Research Fund, Harrison's Fund and Alex's Wish.


Published on 4 May 2016

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