Monday 19 December 2011
Summit secures funding for utrophin drug for Duchenne
Oxford based drug discovery company Summit Corporation plc has secured £1 million (US$1.5 million) worth of funding from several US-based charities. This will enable Summit to manufacture a new formulation of a potential drug for Duchenne muscular dystrophy and conduct a phase 1 clinical trial in healthy volunteers. The drug called SMT C1100 is designed to increase levels of utrophin in the body which may be able to compensate for the lack of dystrophin in these patients. It is thought that this therapeutic approach may also be applicable to individuals with Becker muscular dystrophy.
It is thought that utrophin, a protein naturally present in our body in small amounts, may be able to compensate for the lack of dystrophin in boys with Duchenne muscular dystrophy since both proteins are structurally similar and appear to have very similar functions
The Muscular Dystrophy Campaign has funded Professor Dame Kay Davies' research into utrophin for more than 25 years. In collaboration with Summit plc they found a potential drug to increase levels of utrophin which was named SMT C1100. Research has shown that treating a mouse model of Duchenne muscular dystrophy with SMT C1100 resulted in increased muscle strength and muscles that didn't tire as easily. This drug may also be applicable to individuals with Becker muscular dystrophy who have reduced levels of dystrophin in their muscles, but this has not been studied in detail as yet.
SMT C1100 was recently tested in a phase 1 clinical trial, but unfortunately even at high doses only small amounts entered the bloodstream. Summit plc has since reformulated SMT C1100 into a form that should be better absorbed and therefore reach the muscles at the levels thought to be required to improve muscle function. The planned phase 1 trial will evaluate if this is the case in humans.
The funding secured by Summit comprises of US$750,000 from the Muscular Dystrophy Association ('MDA'), US$250,000 from Parent Project Muscular Dystrophy ('PPMD') and US$500,000 from a group of four independent Duchenne foundations: Charley's Fund, Cure Duchenne, Foundation to Eradicate Duchenne and the Nash Avery Foundation.
Further information and links
Read about the ongoing utrophin research we are funding in Professor Davies laboratory.
Read the Summit Corporation plc press release.
More information about Duchenne and Becker muscular dystrophies.
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