CHRISTMAS has come early for one scientist hoping to develop a pioneering therapy to help people with a rare spinal condition.
Professor Matthew Wood, of Oxford University, has begun a study – part-funded by an £80,000 grant from the Muscular Dystrophy Campaign (MDC) and The SMA Trust – into ways of helping those with spinal muscular atrophy (SMA).
And it is work like his which is supported by the annual Spirit of Christmas charity concert performance held in Oxford.
Money raised from the festive spectacular is used to invest in research projects such as those undertaken by Prof Wood.
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He will investigate how “molecular patches” can be used to restart the production of a vital protein needed by nerve cells.
They were first used to treat severe forms of muscular dystrophy, but it is believed adapting them could help up to 2,500 children and adults in the UK.
Prof Wood said: “With this important project we aim to develop a treatment for SMA and also develop much improved methods for getting these drugs directly to the brain and spinal cord, where they are most needed.”
Zoe Williams, MDC special events co-ordinator, said: “Professor Wood’s study is one of ten exciting new projects to help fight muscle-wasting conditions that the Muscular Dystrophy Campaign has been able to fund this year.
“Purchasing a ticket for an event like the Spirit of Christmas is a great way to support us.
“It’s going to be a wonderful evening and just by coming along with friends or family and enjoying getting into the Christmas spirit you will be helping us to keep this kind of vital research going.”
There are several types of SMA, which is a rare and genetic condition.
It can affect crawling and walking ability, as well as hand, arm, neck and head movement, breathing and swallowing.
The cause of the condition is a fault in the SMN1 gene – which carries information needed to create the “survival motor neuron” protein. A lack of that information stops the creation of the protein, meaning nerve cells are unable to properly control muscles.
However a second gene, SMN2, can also produce the required protein but in much smaller amounts.
The molecular patches – short fragments of DNA – can be used to alter the way the SMN2 gene is read so that larger amounts of protein are produced.
It is hoped the patches can also be applied to the difficult-to-reach regions of the spinal cord and brain without the need for injections.
Prof Wood and his research team hope to accomplish this by testing short protein fragments – known as peptides – which can link to the patches and allow them to reach the spinal cord and brain.
MDC director of research Dr Marita Pohlschmid said: “Today this therapy is in advanced clinical trials for that condition. It gives us great encouragement that it could be adapted to treat SMA.”
The MDC will be holding its annual Spirit of Christmas event, supported by the Oxford Mail’s sister paper The Oxford Times, on December 3.
The annual festive musical evening, to be held in the Sheldonian Theatre, will feature the Dragon School Choir and is also expected to attract guest stars.
Newsnight journalist Katie Razzall has already signed up to speak at the event.
Previous years have included Dominic West, Alison Steadman and Miranda Richardson.
- Ticket prices for the event start at £10. Visit musculardystrophy.org or call 020 7803 2856.
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