Background

Fundamental limitations with the three approved therapies drive the need for new research that will benefit all people living with SMA.

Professor Boido, in collaboration with Professor Artero (University of Valencia, Spain) and Doctor Martinat (Inserm, I-Stem, France), will investigate if a drug that is already on the market for another condition, might benefit SMA, on the grounds that this drug has been shown to delay neurodegeneration and counteract muscle atrophy in different experimental SMA models. Importantly, as it is already a marketed drug, its long pharmacovigilance track record can be fostered to ensure safe use in SMA patients.

How will Professor Boido and her collaborators do this?

In order to support the use of this drug in humans, Professor Boido’s team and her collaborators will need to understand the molecular mechanism of its action. They will do this by looking at the relationship between dose and both the molecular and functional improvements. Importantly, they anticipate a more robust restoration of defects by combining this drug’s ability to increase SMN protein expression and risdiplam’s capability to improve splicing.

Why is it interesting to patients?

As it is already a well-known for the treatment of other conditions, bringing this drug forward for clinical trials should be prompt: this strategy is called drug repurposing.

By using a known drug, Professor Boido’s research could make substantial clinical, economic and social impacts.