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Researchers test old drugs for potential treatment in spinal muscular atrophy (SMA)

Researchers in the UK and Germany have used specific molecular tools to identify drugs previously approved for use in other diseases that might be useful for treating spinal muscular atrophy.

Dr Bill Duddy writes…

Using cell studies to find new ways to use old drugs

The cells in our body are filled with molecular complexity. Every cell is like a mini universe made up of many thousands of different protein machines interacting (a way of communication) with millions of copies of themselves and other proteins, and many billions of other molecules, such as DNA, fats, and sugars. When a drug molecule affects a cell, it rarely changes just one part of this universe, it more often stirs up lots of the cell’s processes, disturbing many aspects of what the cell creates, or how it controls itself.

Researchers can measure these effects, for not just one drug but for many. By using modern scientific tools that track, or profile, the quantities (amounts) of many different molecules at the same time, the effects of thousands of drugs have been carefully catalogued.

Using this knowledge, researchers can sometimes predict which drugs could help treat a certain condition. If one drug has already been found to have a positive effect on cells from people with a particular condition, and if the molecular changes caused by that drug have been profiled, the catalogue can be checked for other drugs that have a similar effect on another condition. These other drugs then might turn out to be even better than the first drug at treating the condition.

This is one type of an approach known as drug repurposing, or drug repositioning, where drugs that have already been tested and approved for clinical use in one condition, are repurposed to treat another condition.

Testing new drugs for spinal muscular atrophy (SMA)

In this research study partly funded by Muscular Dystrophy UK, researchers at Keele University and the University of Oxford in the UK, and at Hannover Medical School in Germany, have used a drug repositioning approach for spinal muscular atrophy (SMA).

SMA is a genetically inherited condition that causes progressive weakness and wasting of the muscle, and exists in several forms affecting children, young people, and adults. Although powerful treatments have recently emerged for SMA, they are not complete cures, and repurposed drugs could complement them while avoiding some of the costs of developing new drugs from scratch.

The researchers had previously shown that a drug called prednisolone could improve signs of the condition in mice. They have now tracked the molecular changes caused by prednisolone, and used computational methods to identify other drugs that might cause similar changes which have already been approved for clinical use in other diseases. They checked which of these had previously been used safely in young patients and could be taken orally, finding two (metformin and oxandrolone) to take forward for further testing. They found that both metformin and oxandrolone had positive effects on an experimental worm model of SMA, but only oxandrolone helped survival in mice, and its effects were not as beneficial as those of prednisolone.

The significance of this research

This work is important because it helps us understand the molecular changes that happen in SMA, and whether drugs like prednisolone can correct those changes. Although the researchers tested only two additional drugs, they identified many more that could be explored as potential treatments for the condition. The findings will help to improve future SMA drug repositioning studies, making management of this condition less challenging.


Please note, you should not take prednisolone without it being prescribed by your doctor. Every drug taken must be suggested by your doctor and carefully monitored as they come with various side effects, especially with long-term use.

‘What’s new in research’ is our monthly blog series featuring recent advancements in research into muscle wasting conditions. Each month, we choose a research article that will be summarised for you by our research communications volunteers, all of whom have backgrounds in various fields of research.

This piece is written by Bill Duddy. Bill is a lecturer in the neuromuscular research team at Ulster University School of Medicine. He lost his brother to Duchenne muscular dystrophy, has devoted his career to the understanding and treatment of neuromuscular conditions, and enjoys explaining science.

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