New therapy offers hope to spinal muscular atrophy patients
This image shows the results of a new therapy technique for Spinal Muscular Atrophy patients developed by academics at the University of Sheffield. The study has led to the restoration of the SMN protein in motor neurons, as shown in this image. Credit: Professor Mimoun Azzouz, from the University of Sheffield's Department of Neuroscience
Children who suffer from the devastating disease Spinal Muscular Atrophy are set to benefit from a new breakthrough in therapy developments by researchers at the University of Sheffield.
The research, which was published in Science Translational Medicine, has shown that a novel gene transfer system has the potential to provide an effective therapeutic treatment for SMA patients.
SMA is a devastating motor neuron disease which affects children. It is caused by an abnormal survival motor neuron (SMN) gene, which leads to a reduction of SMN protein levels. The disease is currently incurable, and patients often require prolonged medical care as no effective treatments to alleviate the condition, currently exist.
The research team at Sheffield, led by Professor Mimoun Azzouz, tested whether a novel gene replacement therapy through a single injection was the most efficient way to treat the disease in a group of mice. They found that the injection, which expressed the SMN gene, successfully restored the SMN protein to normal levels and alleviated symptoms in the SMA model.
The new technique has the potential to develop a simple injection, without any requirement for risky and costly surgical interventions, and has achieved the highest therapeutic effects reported in the field to date. This has significant implications for the future treatment of SMA.
Professor Mimoun Azzouz, from the University’s Department of Neuroscience, said: “I am delighted by the outcome of several years of efforts to tackle this devastating disease. These results bring us one step closer to a successful gene therapy treatment for patients with SMA.”
More information: The paper, entitled ‘Systemic delivery of scAAV9 expressing SMN prolongs survival in a model of spinal muscular atrophy’ was published in Volume 2, Issue 34 of Science Translational Medicine: Chiara Valori, Ke Ning, Matthew Wyles, Richard J Mead, Andrew J Grierson, Pamela J Shaw, Mimoun Azzouz.
Provided by University of Sheffield.
SOURCE: www.physorg.com
on June 12, 2010 at 7:03 pm
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Nice content. Thank you for your information.
on June 13, 2010 at 6:19 am
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Thank you too for visiting my website & for checking this useful information.
on June 14, 2010 at 10:58 am
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That sounds so promising! Thank you Syed for the great news
on June 14, 2010 at 12:25 pm
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Yes Andrea, this sounds promising. Lets have our hopes high
on June 15, 2010 at 12:19 am
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Is there a way to find out when this treatment will become available to patients? Thanks.
on June 15, 2010 at 2:45 am
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Hi Matt, its good to see that the latest research is giving so much of hopes to find a cure to SMA. I am sure one such wonderful cure will be available to patients once they are pretty sure to go ahead with it. Thank you.
on July 13, 2010 at 9:20 pm
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What a great resource!
on August 21, 2010 at 10:07 pm
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Hi Syed !
If the treatment of SMA start in a cuple of years, is it going to be late for our son, who is at age of 16 months today ?
on August 22, 2010 at 9:02 am
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Dear Sladjana, its never late when something good happens finally. Lets pray God that the cure is so sooner, so many SMA families will smile back finally.
on October 18, 2010 at 12:24 pm
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Very nice post!
on October 29, 2011 at 6:18 am
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Great news! Thanks Doc. I am a 64 y/o type IV. Diagnosed 4 years ago. I have weakness in both calves and ankles. I walk 3 miles twice weekly. No weakness above knees. I am a bodybuilder. Will someone my age hope for a reversal with the new drugs. charles
on October 29, 2011 at 4:38 pm
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Hi Charles, nice to know about you. Lets hope for the best that there will be something soon to make sure the cure is on its way.