2 UK Nonprofits Fund PhD Student to Study SMA Mechanisms, Treatments

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by Lindsey Shapiro, PhD |

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A couple of nonprofits in the U.K. will jointly sponsor a PhD student’s research into the mechanisms underlying nerve degeneration and potential treatment approaches for spinal muscular atrophy (SMA).

Totaling £113,022 (nearly $150,000) and funded evenly by Spinal Muscular Atrophy UK (SMA UK) and Muscular Dystrophy UK (MDUK), the grant will be awarded to a graduate student of Lyndsay Murray, PhD, a professor at the University of Edinburgh in Scotland.

The studentship is to cover four years of salary and some research supply costs for a student training in Murray’s laboratory, who will work on a project titled “Understanding and supporting motor neurons following compensatory sprouting using mouse models.”

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“We are delighted to be working in partnership with SMA UK to fund the study at the University of Edinburgh,” Kate Adcock, PhD, MDUK’s director of research and innovation, said in a press release. “Not only will we be funding an excellent study, but we’ll be helping to support a new researcher begin their career in this exciting field.”

SMA is characterized by the progressive loss of motor neurons — the nerve cells that provide instructions to muscles to control voluntary movements — resulting in muscle weakness and atrophy. In patients, motor neurons and the muscles they connect to, together called a motor unit, are enlarged.

These enlarged motor units then experience high levels of oxidative stress — an imbalance between highly reactive “free radical” molecules and the antioxidants the body uses to defend against them. This cellular stress is thought to negatively affect motor neuron survival and contribute to disease progression.

One goal of the project is to use a technique called transcriptional profiling to gain a sense of which proteins have altered levels after motor unit enlargement in healthy mice. This could provide clues as to which molecular pathways are involved in motor neuron degeneration and inspire new therapeutic approaches, according to SMA UK.

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A second goal will be to screen therapies that might improve motor neuron survival by preventing oxidative stress in a mouse model of SMA. These mice will also be treated with Biogen’s approved SMA therapy, Spinraza (nusinersen).

“SMA UK are delighted to be working with MDUK to fund this important work,” said Angela Smith-Morgan, CEO of SMA UK. “It is an interesting area of research which will add to our understanding of muscle development in people living with SMA.”