August 15th, 2022
Researchers have taken an “important new step” in finding a treatment for motor neurone disease after they found a drug commonly used for enlarged prostates and high blood pressure could treat the illness.
MND is a group of rare diseases that destroy cells called motor neurones and causes patients to slowly lose the function of their muscles.
There are currently more than 420 living with MND in Ireland and registered with the Irish Motor Neurone Disease Association. Broadcaster Charlie Bird was diagnosed with the condition last year.
Researchers at the University of Edinburgh have now demonstrated the drug terazosin protects against the death of motor neurones in zebrafish, mice, and stem cell models by increasing energy protection. Alongside partners at Oxford University, experts wanted to determine if the drug could also protect motor neurons from MND. They focused on an enzyme – an active molecule in the cells – involved in energy production called PGK1.
Motor neurones were grown in a dish and experts demonstrated that terazosin protects these cells by increasing energy levels. Terazosin also protected motor neurones in a mouse model of MND, improving survival and delaying the progression of paralysis.
They believe this could slow the progression of the disease in humans and are now looking into launching a clinical trial with 50 patients 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.
Motor neurones need to produce energy to carry the brain’s instructions to the muscles. If there is not enough energy, the messages cannot be transferred effectively and movement is affected. The research was funded by MND Scotland which says it is “delighted” to see a potential new therapy for the disease.
Dr Jane Haley, Director of Research for MND Scotland, 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.
Dr Helena Chaytow, senior postdoctoral researcher at University of Edinburgh’s Euan MacDonald Centre and first author of the study, said: “Our work shows that terazosin is protective of 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 is already prescribed for a different health condition, so we know that it is safe for humans and could quickly move to the clinic.”
Speaking from the Irish perspective, Professor Orla Hardiman said, “As we are beginning to dissect out the various different factors that contribute to MND, there is an evolving interest in testing drugs that are already available on the market on laboratory models of MND. However these studies are at a very early stage. We must remember that drugs that work in animal models are not always effective in humans, because MND is a human condition, and does not occur naturally in animals“.
She continued, “To protect us, all drugs, even those that are “repurposed”, must go through a rigorous testing process to make sure in the first instance that they do not cause harm, and to be sure that they are more effective that currently available treatments. This testing process is done in clinical research centres, is overseen in Ireland by the Health Products Regulatory Authority, and the National Research Ethics Committee.”
The Irish MND Research Group works closely with industry, and with specialist centres around Europe, and is actively involved in many clinical trials for MND. For more information please click on https://rmn.ie/clinical-trials-2/
Lillian McGovern, CEO IMNDA said, “The IMNDA welcomes the news on this drug and we will await to see how transferable the results are to clients who present with MND for medical treatment.”