Teaching An Old Drug New Tricks: Repositioning Strategies for Spinal Muscular atrophy

Abstract

Spinal muscular atrophy (SMA) is a childhood disorder caused by loss of the SMN gene. Pathological hallmarks are spinal cord motor neuron death, neuromuscular junction dysfunction and muscle atrophy. The first SMN genetic therapy was recently approved and other SMN-dependent treatments are not far behind. However, not all SMA patients will reap their maximal benefit due to limited accessibility, high costs and differential effects depending on timing of administration and disease severity. The repurposing of commercially available drugs is an interesting strategy to ensure more rapid and less expensive access to new treatments. In this mini-review, we will discuss the potential and relevance of repositioning drugs currently used for neurodegenerative, neuromuscular and muscle disorders for SMA.

Keywords::

• drug repositioning
• motor neuron
• neuromuscular disorders
• neuromuscular junction
• skeletal muscle
• spinal muscular atrophy
• survival motor neuron
• therapies

Financial & competing interests disclosure

JM Hoolachan is funded by a studentship from the Keele University School of Medicine. ER Sutton is funded by a studentship from Muscular Dystrophy UK. M Bowerman receives funding from SMA Angels Charity, Association Française contre les Myopathies, Action Medical Research and Muscular Dystrophy UK for SMA research. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

No writing assistance was utilized in the production of this manuscript.

Additional information

Funding

JM Hoolachan is funded by a studentship from the Keele University School of Medicine. ER Sutton is funded by a studentship from Muscular Dystrophy UK. M Bowerman receives funding from SMA Angels Charity, Association Française contre les Myopathies, Action Medical Research and Muscular Dystrophy UK for SMA research. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.