DMD drugs20 Oct 2017 09:21
From the Muscular Dystrophy UK web site:
"Another potential therapy for DMD is to raise the levels of utrophin protein, as a functional substitute for dystrophin. The potential treatment has been developed in the laboratory of Professor Kay Davies in Oxford.
Despite excellent results in animal models, the first clinical trial in healthy volunteers of a first generation drug SMT C1100 lead to the delivery of insufficient amount of the drug to the muscles. SMT C1100 has since been improved and has recently successfully met its primary endpoint of safety and tolerability in a phase Ib clinical trial in DMD patients. On the basis of these findings, a second phase I trial tested the drug in boys with DMD when they are on a particular diet. The results showed that a balanced proportion of fats, proteins and carbohydrates increase the levels of SMT C1100 in the blood. A phase II open-label clinical trial to evaluate the safety and longer-term benefits of SMT C1100 on muscle health and function is currently underway.
There is now considerable effort ongoing in Oxford to develop second-generation drugs that have the potential to raise utrophin levels further. One such potential drug, SMT022357, has been shown to be distributed along the length of muscle fibres in the mdx mouse. Owing to its physical and chemical properties SMT022357 is likely to be more stable in the body than SMT C1100."
Hadn't appreciated that the next generation of these drugs was this far on. Sounds positive.