Truly remarkable’ drug can reverse decline caused


A truly remarkable’ drug can reverse the decline caused by motor neuron disease

Scientists say they’ve slowed and even reversed some of the devastating effects of motor neuron disease the treatment works in only 2 percent of patients but the team at the University of Sheffield has described it as truly remarkable at a moment of hope for the.


whole disease MND association set that walls are mounting confidence in the therapy. Other research was carried out in part by the neuroscience institute at Sheffield university in

England its director is professor Dame Pamela shal thank you very much for coming to the program. My pleasure so any are you read 0 good news could you just talk us through exactly how this works in the simplest possible terms. So as you mentioned 2 percent.


of patients with MND have a slight fall in this SO do warm salt one gene and what’s the toughest some therapy done us it has to be injected to rile a lumbar puncture into the

cerebral spinal fluid and it lowers the level of the sub-one protein and the toxic effects on motor neurons that arise from that protein. The treatment has to be given at monthly intervals. I say I’m this kind of technology approach that helps just a small.


proportion what kind of confidence come we get from it can it be broadened out do we know it definitely works all the time was the kind of confidence levels around. So we know

that there are at least 13 genes that can cause or predispose to motor neuron disease SO 21 was the first gene found back in 1993 so I think it’s a really important proof of principle for the first rogue gene that can cause an injury so that approach can be applied to.


other genetic subgroups but also a very important thing in this trial is that we’ve now got proteins called biomarkers that we can measure to show that the treatment is working so

we could measure the reduced level of the SOD 1 protein in the spinal fluid we could measure the reduction of numerous filament proteins that are released as motor neurons get injured and degenerate. How for some treatment lowering the level of those proteins so.


that’s a Monaco that we’ve got that will enable us to tell if an experimental treatment is working as an early stage was overseas but yet potentially very useful that. early stage I

think it just pulls and just reminds people what exactly is M. and T. and what are some of the devastating impacts often. So we have 2 sets of motion on your own promoting your arms in the front part of the brain and low notes in your own soul the way down the spinal cord.


in your back and what they do those precious cells, in a nutshell, is form a connection between the brain and the muscles so it is that they all the vehicle if you like by which the

the brain controls movements and asks those cells to get damaged and degenerate than the connection between the brain and the muscles are lost and the muscles become weak and wasted and then there is this cruel creeping paralysis that happens to patients and it can. affect the


muscles of the arms and legs the speech and swallowing muscles the muscles that control breathing but certain muscle groups the ones that move the eyes for example are relatively

spending the disease is interesting to use the word cruel that that is exactly how it seems and feels to somebody it is such a devastating impact on people’s lives and is it a physical service a particularly complicated area of science why have we taken so long to get.


where we are now or is this actually an example of we have enough funding and I’m the research is going well just give us a scale and put it into some kind of context for us so I think

the nervous system is undoubtedly more difficult than some other systems in the body to get effective treatments you know to get effective treatment since the number system is more difficult we come we haven’t been able until recently to study the nervous system until.


For MND patients in life if your skin doctor can take a biopsy of the diseased skin and study it in the bar tree we obviously can’t do that to live living human beings but

nowadays we can take a small. A sliver of skin cells and reprogram those cells in the lab or treat to become motor neurons well the neighborhood cells that fit around the motor neurons in the nervous system and protect them so we can Montel the disease much better.


but that is only happened in the last few years hi Sir so we use those models to try and understand the mechanisms of disease and also identify new treatment targets our next

talk for some has emerged from just step process. I’m afraid that is a huge complicated area and I wish we could have more time to go into it because it is so tricky but thank you so much for explaining it’s okay professor Dame public show thank you.


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