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There are over 60 different types of muscle-wasting conditions and they can affect people in different ways. These conditions, which affect children and adults, cause muscles to weaken and waste over time. Many of these conditions are genetic and, while they may be inherited, they can sometimes occur without a family history of the condition. For other conditions, the cause is unknown.
Some conditions affect multiple organs in the body, not just the muscles like those in our arms and legs. If some potential treatments are to be as effective as possible, they need to reach the different parts of the body. The treatments need to get not only to the muscles but also to other parts of the body, such as the heart muscle, diaphragm or nerves. That’s why we’re funding research into improving the delivery of treatments; our goal is to develop potential treatments that could stop muscle-wasting conditions in their tracks.
Researchers around the world are working hard to develop potential treatments and technologies to ensure that they are delivered safely and effectively. As part of this effort, MDUK is funding projects in which researchers are linking a type of potential therapy in development, called antisense oligonucleotides (molecular patches) to short fragments of protein called peptides, which can help the treatment get inside cells, including the muscle and heart. Another project is linking molecular patches to antibodies that could help deliver the treatment to nerves. In some cases, new delivery methods may lead to safer, simpler, and less invasive ways to deliver treatments than those currently available. We’re also funding research to improve the design of potential treatments to make sure they are effective in these different organs once they get there.
Another challenge in the delivery of some potential treatments, such as AAV gene therapy, is that the body may generate an immune response to the treatment. This means it may destroy the treatment before it’s had a chance to act. So we’re also funding research that aims to overcome these challenges, for example by developing different technologies that aim to shield potential therapies from the immune system and deliver them to the muscles or act as an alternative delivery method.
The knowledge researchers gain from this research and the technologies they’re developing will be invaluable. It could help in the design and delivery of potential therapies for many different muscle-wasting conditions.
We’re excited to be funding a number of different research projects into pioneering technologies to improve the delivery of potential therapies and their function in different organs in the body. You can read more about them here:
- Improving the delivery of molecular patches for spinal muscular atrophy
- Developing an advanced molecular patch therapy for myotonic dystrophy type 1
- Using molecular patches to prevent heart muscle disease in Duchenne muscular dystrophy
- Developing new delivery methods for microdystrophin gene therapy
- Understanding heart failure in Duchenne and Becker and developing an improved gene therapy
We also fund research into improving our understanding of muscle-wasting conditions, developing potential treatments and improving the quality of life for people living with muscle-wasting conditions. You can find out more about these projects here.