Monday 28 May 2012
Phase 1 clinical trial of utrophin drug for Duchenne announced
The start of a phase 1 safety trial of a drug designed to treat Duchenne muscular dystrophy has been announced by Summit Corporation plc. The drug, currently being tested in healthy volunteers, is designed to increase levels of utrophin in the body which may be able to compensate for the lack of dystrophin in boys with Duchenne muscular dystrophy.
Oxford based drug discovery company Summit Corporation plc has announced the start of their phase 1 clinical trial to test a drug for Duchenne muscular dystrophy. During the trial, healthy volunteers are being given the drug in order to assess how safe and well tolerated it is at various different doses.
SMT C1100 was previously tested in a phase 1 clinical trial by US pharmaceutical company BioMarin, 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. In addition to testing the safety of the drug, this phase 1 trial will also evaluate how well this new formulation of the drug is absorbed.
It is anticipated that initial results from the trial will be available before the end of this year. A successful outcome from the phase 1 trial is expected to lead to a phase 2 study in Duchenne muscular dystrophy patients. 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.
The Muscular Dystrophy Campaign has funded Professor Dame Kay Davies' research into utrophin for more than 25 years and in collaboration with Summit they discovered and developed 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.
The start of this clinical trial is especially exciting because, unlike other approaches such as exon skipping, this drug has the potential to treat all boys with Duchenne muscular dystrophy, regardless of what genetic mutation they have.
Further information and links
Read about the ongoing utrophin research we are funding in Professor Davies laboratory.
Read the Summit Corporation plc press release.
If you have a question about this or any other research, please email us:
Read about the Duchenne muscular dystrophy research we fund.
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