ReveraGen BioPharma has announced the start of a Phase 1 clinical trial of a new steroidal drug, VBP15, for Duchenne muscular dystrophy (DMD). It is hoped that VBP15 will have at least the same benefits as the currently used corticosteroids, but fewer side effects.
The trial, being conducted in the USA, will involve only healthy volunteers. This is standard practice for phase 1 trials and will give the first information about the safety of the drug in humans.
If all goes well in the phase 1 trial, a phase 2 trial in boys with Duchenne MD should start later this year.
Research in mice, which was published towards the end of 2013, showed that VBP15 worked better than prednisolone without the harsh side effects. Read about these laboratory results here.
VBP15 may also prove to be a beneficial treatment for some other types of muscular dystrophy where inflammation is present in the muscle, but further clinical trials will be needed to determine this.
Corticosteroids such as prednisolone are currently the only medication known to help maintain muscle strength in boys with Duchenne muscular dystrophy. On average boys taking this medication are able to delay use of a wheelchair for three years. However, the harsh side effects have a significant impact on quality of life and prevents them being prescribed at the optimum age – before symptoms appear. Many patients also stop taking the medication due to the side effects which can include weight gain, mood changes, stunted growth, fragile bones, cataracts, high blood pressure, diabetes and increased chance of infections. You can read more about the pros and cons of steroids in our factsheet.
Why are only healthy volunteers included in the phase 1 trial?
Healthy volunteers are used in phase 1 studies because if side effects do occur then it is more straightforward to know that it is likely to be caused by the drug, not because of the symptoms of muscular dystrophy. There is also usually little or no physical benefit for the participants in phase 1 trials because the trials are so short, so it is better to ‘save’ the patients for the phase 2 trial which will be longer and will start to look at whether the treatment works.
• The Duchenne MD factsheet contains information about the condition and includes a research summary
• Read the press release about the start of the VBP15 clinical trial
• Clinical trials: your questions answered
• Read about the research MDA funds that aims to reduce inflammation in the muscles and improve muscle regeneration.
• You can get regular updates by becoming a friend on MDA’s Friends Facebook page, liking the MDA’s Company Facebook page
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Uploaded 25 February 2015