A drug which has shown promise as a new therapy for motor neuron disease (MND) will move to a feasibility study involving people living with MND.

MND Scotland was the key funder of a study which found that terazosin, a drug typically used to treat enlarged prostates and high blood pressure, could also help people living with MND.

MND is a rapidly progressing terminal illness which destroys nerve cells known as motor neurons, causing patients to slowly lose function of their muscles. The average life expectancy is currently just 18 months from diagnosis.

In studies using zebrafish, mice and stem cell models, experts have demonstrated that terazosin protects against the death of motor neurons by increasing their energy production.

Researchers say the drug could help slow the progression of MND, which affects around 400 people in Scotland at any given time.

If the new feasibility study into the drug’s effect in MND patients yields positive results, this could pave the way for a full clinical trial in the future.

It is still unclear why motor neurons die, but experts know that a decrease in their energy production takes place at an early stage of the disease.

Motor neurons 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.

Researchers funded by MND Scotland, at the University of Edinburgh and the University of Oxford, targeted the energy production of motor neurons as a potential therapeutic strategy for treating MND.

Using terazosin, which has previously been shown to be effective at increasing energy production in models of stroke and Parkinson’s disease, the team wanted to determine if this 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.

Zebrafish models of MND showed that either genetically increasing the amount of PGK1 in the zebrafish or treating them with terazosin to increase PGK1’s activity improved the growth of motor neurons.

Terazosin also protected motor neurons in a mouse model of MND, improving survival and delaying the progression of paralysis.

The team also grew motor neurons in a dish and demonstrated that terazosin protects these cells by increasing energy levels.

To investigate this further the teams at the Universities of Edinburgh and Oxford are inviting fifty 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.

The study is published in eBioMedicine. It was funded by MND Scotland and the My Name’5 Doddie Foundation.

Dr Jane Haley MBE, Director of Research for MND Scotland, said: “As key funders of this research study, we are delighted to see a potential new therapy for MND on the horizon.

“The results of this study show us that terazosin, a drug currently used to treat enlarged prostates and high blood pressure, may be able to protect motor neurons. We are delighted that, as a result, 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.

“As a charity, our work is only possible because of the incredible fundraisers who go the extra mile to help fund vital research. Thank you to everyone who is doing their bit to make time count.”

Dr Helena Chaytow, senior postdoctoral researcher at the University of Edinburgh 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.”

Professor Tom Gillingwater, Professor of Anatomy at the University of Edinburgh and study co-lead, said: “We are excited about the potential for terazosin to impact on the breakdown of motor neurons in MND. The current work illustrates the importance of bringing together scientists and clinicians in order to identify new targets for therapy suitable for taking forward into studies in human MND patients”.

Professor Kevin Talbot, Professor of Motor Neuron Biology at the University of Oxford and study co-lead, said: “We urgently need to accelerate the way drugs are developed from laboratory models into trials in patients. Our work uses a combination of approaches to increase the confidence that drugs will actually work in people with MND and significantly slow disease progression. It represents an important new step in the search for therapies”.

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