Scoping out new treatments for SMA from existing drugs

Published Date

The exciting speed of research into treatments for spinal muscular atrophy (SMA) over the last few years has given lots of people living with the condition hope. Top of that list has been Spinraza, which MDUK continues to vigorously campaign for so that all those in the UK who could benefit can access it.

But what other treatments are needed to support people with SMA through their whole lives – and what about other options we need to create for people who don’t respond to Spinraza?

Dr Melissa Bowerman, who works at Keele University, said:

“We know the therapeutic landscape has changed – two years ago there was no treatment, but now we’re in a position where in a few years most patients could have received one of several gene therapies that are being developed.

“With Spinraza we know that although the results are spectacular, it’s not a cure. There are also a small number of patients that do not respond as well to the treatment, and older patients for whom the priority may be to maintain a good quality of life.

“As a community we know we have to keep developing more therapies.”

Repositioning existing treatments

Finding new treatments can be a long and difficult process, but an existing drug has been identified by Dr Bowerman that could be interest. The treatment, which affects metabolism, is already being tested for other muscle-wasting conditions including Charcot Marie Tooth disease (CMT), as well as a whole host of other conditions including cancer, diabetes and Alzheimer’s. That means there’s lots more research on its use to draw on.

“That’s a core aspect of our research programme – repositioning drugs that already exist,” says Dr Bowerman. “If we get really positive results in an animal model, it’s a lot easier to transfer to the clinic.”

The potential for this new drug was found because of work Dr Bowerman’s team has previously done on what was going wrong inside the cells of SMA animal models.

With that problem isolated, it was possible to see what other treatments were in use for other conditions that have similar effects.

The project will now take about a year to work with cells to make sure the drug is safe and effective, before moving on to tests in animal models.

Ultimately, Dr Bowerman hopes to test the drug with genetic therapies for SMA to see if they can work together to have an even greater effect.

To find out more about the science, read the lay summary for the project.

Treatments working together

Dr Kate Adcock, Director of Research and Innovation at Muscular Dystrophy UK, said:

“SMA can be such a devastating condition so it’s fantastic to see innovative treatment possibilities such as this new use for an old drug. This is early-stage research but we are really excited to see how it could work with other treatments to offer real hope for people living with SMA in the future.”

Dr Bowerman said:

“I myself have run in the charity’s Town and Gown event and have seen how many fantastic fundraisers are there supporting MDUK, and so I really appreciate the hard work that goes into fundraising for research like this. We look forward to sharing the results with the entire SMA and MDUK community once it is complete.”

Read more about all of Muscular Dystrophy UK’s 2018 research grants, covering 10 projects and totalling £1.2m – including a project looking at ways to monitor the progression of SMA.

For more information on these grants or any aspect of research into muscle-wasting conditions, contact our Research Line on and 020 7803 4813.

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