Motor neurone disease -- helping the body to help itself | Action Medical Research

Touching Lives - December 2003

Motor neurone disease -- helping the body to help itself

Symptoms include swallowing difficulties, slurring of speech and shortness of breath, all of which lead to severe disability and premature death. There is currently no treatment for MND. Professor Jackie de Belleroche and her team intend to change that.

Jackie’s previous projects — two of which have also been funded by Action Medical Research — have discovered the potentially lifesaving role of Heat Shock Protein 27 (HSP27). Heat Shock Proteins are found in most cells and have two functions: they act as ‘molecular chaperones’ transporting proteins around the cell, and they are induced in response to injury as a part of the cell’s natural defence mechanism.

HSPs were first detected because they occurred as a response to increases in temperature — but ^they can cope with any challenge, and in our brain they come into action to counter any threat to health^.

“We have been characterising HSPs in cell culture and other models,” Jackie told me in her Charing Cross Hospital laboratory. “We’ve seen HSPs induced in epilepsy and stroke models. The HSP removes aberrant proteins so that the bad ones don’t build up and cause the cell to die — they are part of the normal neuro-protective mechanism, and there are several HSPs that complement each other’s actions. We now know that HSPs are acutely relevant for coping with damaged proteins.”

Jackie and her team believe they can protect cells by inducing the over-expression of the uniquely valuable HSP27, and have already demonstrated that HSP27 can significantly reduce seizures.

Now they are turning their attention to motor neurones in the spinal cord (the nerve cells that transmit impulses to the muscles telling them what to do, and the degeneration of which lead to the symptoms of MND). The team want to boost the HSP27 available to protect these motor neurone cells which are so vitally important.

Jackie continues, “We need to show in the laboratory that HSP27 can stop the progression of MND. If it does unequivocally we can go forward ideally with a drug treatment. It would be perfectly possible to deliver the HSP to the hippocampus region in the brain which is the vital area for memory and cognitive function. So in parallel with trying to prove that HSP27 can stop motor neurone degeneration, we’re testing drugs that would induce the expression of HSP27. We know HSPs are neuro-protective but we want to prove unequivocally that they can stop motor neurone degeneration.

London-based team

The London-based team is leading the field in this hugely exciting and potentially lifesaving research, which could easily see spin-off benefits for other neuro-degenerative conditions such as Alzheimer’s and Huntingdon’s disease. But Jackie is modest about her team’s work, as you would expect any self-respecting scientist to be!

“We’re just taking advantage of the natural cell function,” she says. “We’re all capable of producing our own HSPs but we’re giving HSP production a boost, which can make all the difference. We’re testing three or four drugs at the moment so if we prove the point about HSP’s effectiveness we can move quite quickly on getting a treatment out there.”

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