Possible treatment for muscular dystrophy
About 100 boys are born with muscular dystrophies every year in the UK. All dystrophies cause progressive muscle weakness and in Duchenne muscular dystrophy this leads to an early death.
One approach to treatment is to find molecules that boost muscle strength. After a mass screening programme that tested chemicals on muscle tissue in culture, a chemical called SMTC1100 was tested on a mice that have muscular dystrophy.
The mice given SMTC1100 were able to run 50% further in exercise trials and when SMTC1100 was given in combination with prednisolone, the steroid commonly taken by boys with Duchenne muscular dystrophy, fatigue was almost completely prevented and the mice were able to run 3.5 times further than with no treatment at all.
SMT C1100 has already been tested in a phase 1 clinical trial on a small number of healthy volunteers and there were no safety concerns. However, the results of the trial were disappointing because insufficient amounts of the drug were being absorbed into the bloodstream and reaching the muscles. The drug is now being re-formulated before new clinical trials.
Find out more about muscular dystrophy here.