‘Important new step’ in search for treatment for motor neuron disease, scientists reveal UK News
Researchers believe they have found an “exciting new potential therapy” for motor neuron disease (MND) with a drug commonly used for other health treatments.
MND is a group of rare diseases that destroy cells called motor neurons and cause patients to slowly lose function in their muscles.
Around 5,000 people are believed to be living with the incurable disease across the UK – the average life expectancy after symptoms appear is around three years.
Treatment could slow the progression of paralysis associated with MND.
Scientists said they made an “important step” with a drug currently used to treat prostate enlargement and high blood pressure.
Research at the University of Edinburgh showed that the drug terazosin protected against motor neuron death in zebrafish, mouse and stem cell models by increasing energy protection.
Together with partners at the University of Oxford, the experts wanted to determine whether the drug could also protect motor neurons from MND.
dr Helena Chaytow, senior postdoctoral fellow at the University of Edinburgh’s Euan MacDonald Center and first author of the study, said: “Our work shows that terazosin protects against motor neuron cell death in multiple models of MND, making it an exciting new potential therapy.
“The benefit of working with terazosin is that it’s already being prescribed for another medical condition, so we know it’s safe for people and could move quickly to the clinic.”
Professor Kevin Talbot, Professor of Motor Neuron Biology at the University of Oxford and co-leader of the study, added that the work “represents an important new step in the search for therapies”.
The research focused on an enzyme – an active molecule in cells – involved in energy production called PGK1.
“Terazosin delayed the progression of paralysis”
Motor neurons were grown in a dish and experts showed that terazosin protects these cells by increasing energy levels.
Terazosin also protected motor neurons in an MND mouse model, improving survival and delaying paralysis progression.
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Scientists believe this could slow the progression of the disease in humans and are considering starting a clinical trial.
Fifty patients were invited by the Oxford MND Care and Research Center to participate in a feasibility study examining the effects of terazosin on key indicators of disease progression.
A number of high profile British sportspeople have shared their experiences of MND in recent years, including rugby league’s Rob Burrow, rugby union’s Doddie Weir and footballer Stephen Darby.
Professor Stephen Hawking also had the disease but lived to be 55 years after being diagnosed at the age of 21 – despite being given two years to live.