MDF Newsletter Content Issue 46 April 2015

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Research As soon as further information is available regarding the recruitment process we will make a further announcement. Summit Corporation plc said it would announce the results of this trial in mid- 2015. If successful, it will be followed by a Phase 2 trial to test the benefit of SMT C1100 and to monitor its safety over a longer period of time. Glyn Edwards, Chief Executive Officer of Summit, said: We believe it is possible to enhance absorption of SMT C1100 through dietary means and this new patient trial is designed to test this so that we can confidently evaluate the efficacy of utrophin modulation in subsequent clinical trials. We believe that utrophin modulation has the opportunity to benefit all boys with DMD [Duchenne muscular dystrophy] and we are working to ensure this molecule has the best chance to reach the market through a well-designed clinical path. The Muscular Dystrophy Campaign works closely with the Utrophin Alliance. This strategic partnership between Summit Corporation plc and Oxford University was set up to speed up the development of treatments for Duchenne muscular dystrophy. The charity currently funds research projects in Professor Kay Davies’ and Dr Angela Russell’s laboratories in the search for more efficient drugs with the potential to raise utrophin levels. Background information The Muscular Dystrophy Campaign has funded Professor Kay Davies’ research into utrophin for more than 25 years. 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. In collaboration with Summit, Professor Davies’ laboratory discovered and developed SMT C1100 to increase levels of utrophin in the body. The results of the trial are encouraging, as the drug has the ability to treat so many boys. Unlike other approaches 40 such as exon skipping, SMT C1100 may have the potential to treat all boys with Duchenne or Becker muscular dystrophy, regardless of their genetic mutation. Article online at http://www.musculardystrophy.org/research/news/8031 Follistatin improves walking ability Tuesday 30 December 2014 Dr. Jerry Mendell’s group from Nationwide Children’s Research Institute, USA recently published the results of a clinical study for Becker muscular dystrophy. The study demonstrated that injecting a modified virus carrying the follistatin gene into leg muscles of people with Becker muscular dystrophy was not only safe, but also improved muscle strength. The results of the clinical study are encouraging and provide initial evidence that increasing the levels of follistatin in leg muscles of individuals with Becker muscular dystrophy can increase their ability to walk a longer distance. More extensive clinical studies are necessary to confirm the initial results and also test whether walking ability is maintained until later in life. The clinical trial followed encouraging tests in animals showing that delivery of the follistatin gene, through a harmless virus called adeno-associated virus, was safe and improved muscle strength. This could also be achieved in a mouse model for Duchenne muscular dystrophy where dystrophin is missing. The follistatin gene carries the information for a protein produced in a number of different tissues in the human body. In the muscle it is known to inhibit a protein called myostatin, which is responsible for the reduction of muscle mass and strength. Increased levels of follistatin therefore lead to reduced myostatin activity, which results in increased muscle mass and strength. The strength of the quadriceps (a leg muscle) is one of the factors that has an impact on the ability of individuals with Becker muscular dystrophy to walk. The rationale of the clinical study was to test whether the delivery of the follistatin gene to this particular leg muscle could strengthen it and improve the individual’s ability to walk. Six participants with Becker muscular dystrophy were enrolled on the clinical trial. They had to be able to complete the six-minute walk test (6 MWT), which assesses the distance people are able to walk in six minutes. Three participants received a low dose of the virus, while the other three received a high dose. After a year, the two individuals receiving the low dose showed an improvement in their walking ability. They could walk 58m and 125m further in the 6 MWT, respectively. The third individual did not show any improvement in his walking ability. The same was observed in the group that received the high dose: two individuals showed an improvement of 108m and 29m in the 6MWT respectively, while the third did not show any improvement in his walking. The researchers used magnetic resonance imaging (MRI) and took muscle biopsies to visualise the structure of the muscles. This showed that muscle cells had increased in size but also showed that fibrosis was stronger in those in whom walking ability had not improved. They therefore concluded that a greater amount of fibrosis might prevent the muscles from getting stronger. Most importantly, the clinical study showed that injections of the modified adeno-associated virus were safe and no complications were observed. The researchers will also test whether the same approach could work in people with other muscle-wasting conditions and they have already started a clinical study including individuals with sporadic inclusion body myositis. They are also planning a study in individuals with Duchenne muscular dystrophy where they will inject the virus into various muscles. Both studies will take place in the US and will include only a small number of participants who will be invited by the investigators. Article online at http://www.musculardystrophy.org/research/news/8063
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MDF Newsletter Content Issue 46 April 2015

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