What are the aims of the project?
The aim of this project is to investigate whether a commercially available drug that targets the body’s metabolism could be a potential treatment for SMA. This could be used alongside therapies that boost production of the SMN protein (called SMN-specific therapies).
Why is this research important?
Recent advances in SMA research have led to the exciting development of genetic therapies that increase the levels of the SMN protein. These SMN-specific therapies, such as SPINRAZA and gene therapy, have shown very promising results in animal studies and clinical trials. However, research has shown that the success of these therapies is also dependent on age and severity of the condition. Therefore, developing non-SMN specific therapies that could be used in combination with genetic therapies could have an even greater effect.
This project will aim to develop a potential non-SMN specific therapy by building on Dr Bowerman’s previous research. She has shown that a hormone involved in regulating the body’s metabolism is elevated in a mouse model of SMA. Reducing the activity of this hormone improved the health of the mice. Dr Bowerman and her student will now determine whether a commercially available drug will have the same effect.
How will the outcomes of this research benefit people with SMA?
The drug being tested in this project has already gone through clinical trials for other health conditions, so researchers already know a lot about its safety and how it works. If it’s shown to be beneficial for SMA, it could potentially be taken forward to clinical trials faster than a new drug.
What will the researchers do?
First, Dr Bowerman and her student will assess how safe and effective the drug is in different cell models (e.g. muscle, heart, neurons and fat cells) before testing it in healthy mice. Next, they will assess the drug’s therapeutic potential in mice with SMA.
Finally, the researchers will treat the SMA mice with a gene therapy – that will increase the levels of the SMN protein –and the experimental drug. This will inform the researchers whether combining the two therapies has an even greater effect than using gene therapy alone.
Grant information
Project leader: Dr Melissa Bowerman
Institute: Keele University
Condition: Spinal muscular atrophy
Duration: PhD Studentship – four years
Total cost (£):108700
Official title: Therapeutic potential of a glucocorticoid receptor antagonist for the treatment of spinal muscular atrophy.
For further information
Find out more about Dr Bowerman’s research in our feature article
If you would like further details about this research project, please contact the MDUK Research Line on 02078034813 or email research@musculardystrophyuk.org
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