Patients have told us they want to know about research: What are scientists studying now? What are they finding out? And how do these findings contribute to progress towards new treatments? Our partner OptiStem, an EU-funded stem cell research project, has been working on a way to help answer these questions.

Summary 

A recent study has shown that muscle stem cells called mesoangioblasts can be grown in the laboratory from induced pluripotent stem cells (IPS cells). Scientists think that mesoangioblasts transplants may be an effective treatment for muscular dystrophy but currently these cells have to be taken from donor who is a tissue ‘match’ for the patient, which is relatively rare.As IPS cells are grown in the lab from a patient’s own muscle cells this could potentially overcome the problem of having to find a ‘matched’ donor.

Summary

There has been much effort by researchers to understand how skeletal muscle repairs itself and which cells are involved in this process. This article summarises a review by researchers in the group of Professor Giulio Cossu from the Stem Cell Research Institute, University of Milan from January 2010. The review discussed the different types of stem cells which could be used to repair muscles; as well as how therapies using these cells might work.

Summary

Recently it’s been shown that relief of muscular dystrophy symptoms is possible using stem cells. In Duchenne muscular dystrophy the protein dystrophin normally found in muscles is absent. Scientists of the San Raffaele Scientific Institute in Milan showed that giving muscles in mice the correct 'recipe' for dystrophin (it's gene) meant that the right protein could be produced.

Summary

Muscle dystrophies are heritable diseases that lead to muscle breakdown, weakness, inflammation, and in severe cases, can result in paralysis and even death. Nitric oxide, normally produced in the body, can activate satellite cells that are able to replace dying fibres with new healthy fibres. However, nitric oxide is not as active as it should be in patients with muscular dystrophy. This study tests NCX 320, a new drug that can release both nitric oxide and ibuprofen (an anti-inflammatory drug).

Summary

A scientific publication from Margaret Buckingham’s research group at Institute Pasteur in Paris identified a new factor that is important for the development of skeletal muscle.

Summary

A recent scientific study has shown that a type of muscle stem cell called ‘satellite cells’ are essential for muscle fibre repair and replacement. Scientists had already identified several different types of muscle cell that can contribute to the formation of new muscle tissue. They are now investigating what exactly each of these cell types do and how they work.

Summary

Research has shown for the first time that a non-muscle type of cell can switch to help make muscle during normal growth. These cells, called pericytes, which are normally found on small blood vessels, were shown to also make both muscle fibres and muscle stem cells. Whilst it has been shown that in extreme situations pericytes can contribute to muscle repair, it was not known whether this was also part of natural muscle growth. 

Summary

Muscular dystrophies cause muscle breakdown, weakness, and can lead to paralysis and death. The only current treatment that is effective is corticosteroids, shown to increase muscle strength. However, we do not know if it is effective in the long term, and there are side effects that limit its use. A combination of an anti-inflammatory drug, ibuprofen, and a nitric oxide delivery drug, isosorbide dinitrate, have been shown in mice to improve muscle health.

Summary

Adult muscle is maintained by a type of stem cell found in the muscle called a satellite cell. These satellite cells multiply and activate during normal muscle growth, and also when muscle is injured. This allows muscle to regenerate. Normally, satellite cells are able to renew themselves, but in muscular dystrophy repeated muscle damage occurs and the satellite cell supplies can run out. Recent research led by Emilio Clementi (from San Raffaele Scientific Institute and University of Milan in Italy) has shown that nitric oxide, a key molecule involved in cell signalling, can help maintain this supply of satellite cells.