RESEARCHERS at the University of Edinburgh have taken an “important new step” in finding a treatment for motor neurone disease (MND).
They found a drug commonly used for enlarged prostates and high blood pressure which could treat the illness.
MND is a group of rare diseases that destroy cells called motor neurons which causes patients to slowly lose the function of their muscles.
Researchers were able to demonstrate that the drug terazosin can help protect against the death of motor neurons in zebrafish, mice and stem cell models by increasing energy protection.
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Alongside partners at Oxford University, experts wanted to determine if the drug could also protect motor neurons from MND.
Director of research for MND Scotland Dr Jane Haley said: “We are delighted that, as a result of the study, the drug will move to a feasibility study in Oxford, involving people living with MND.
“This is a wonderful example of researchers, clinicians and MND charities working together to try and speed up the search for new treatments for MND – because it’s about time we found a cure.”
Researchers focused on an enzyme – an active molecule in the cells – involved in energy production called PGK1.
Motor neurons were grown in a dish and experts demonstrated that terazosin protects these cells by increasing energy levels.
Terazosin also protected motor neurons in a mouse model of MND, improving survival and delaying the progression of paralysis.
Fifty patients have been invited from the Oxford MND Care and Research Centre to participate in a feasibility study which will examine the impact of terazosin on key indicators of disease progression.
About 5000 people are thought to be living with the disease across the UK with the average life expectancy after the onset of symptoms around three years.
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Motor neurons need to produce energy to carry the brain’s instructions to the muscles but if there is not enough energy, then they cannot be transferred effectively and movement is affected.
Senior postdoctoral researcher at the University of Edinburgh and first author of the study Dr Helena Chaytow said: “Our work shows that terazosin is protective of motor neuron cell deaths in multiple models of MND, making it an exciting new potential therapy.
“The benefit of working with terazosin is that it is already prescribed for a different health condition, so we know that it is safe for humans and could quickly move to the clinic.”
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