A new study has found a repurposed drug could help patients with their motor neuron disease treatment.
University of Edinburgh researchers have found a drug typically used to treat enlarged prostates and high blood pressure shows promise as a new motor neuron disease treatment. Motor neuron disease is a group of rare diseases that destroy nerve cells, this causes patients to lose function of their muscles.
There is currently no cure for motor neurone disease, but treatments can alleviate some of the burdens. Motor neurone disease treatments include physiotherapy, medicines for muscle stiffness, and emotional support. However, this new research could slow disease progression and extend the life expectancy of patients.
Protection against death of motor neurons
The researchers used zebrafish, mice, and stem cell models to test the effectiveness of the drug terazosin and found that it protects against the death of motor neurons by increasing their energy production.
They are progressing to a feasibility study into whether the drug would be an effective motor neuron disease treatment in humans. If the results are positive, they will launch a full clinical trial.
Currently, no clear research has uncovered why motor neurons die, but experts know a decrease in their energy production takes place at an early stage of the disease. Motor neurons require energy to carry the brain’s instructions to the muscles but without enough energy, the messages cannot be transferred effectively, and movement is affected.
This spurred researchers from the University of Edinburgh and partners from the University of Oxford to target energy production as a potential therapeutic strategy for motor neuron disease treatment.
Terazosin: a new motor neuron treatment?
Terazosin has previously been shown to be effective at increasing energy production in the models of stroke and Parkinson’s disease, the team wanted to see if the drug could also act as a motor neuron disease treatment. They focused on an enzyme – an active molecule in the cells – involved in energy production called PGK1.
When the researchers used zebrafish models of motor neuron disease, they found that when genetically increasing the amount of PGK1 in the fish or treating them with terazosin to increase PGK1’s activity, improved the growth of motor neurons.
Terazosin protected motor neurons in a mouse model of motor neuron disease, improving their survival and delaying the progression of paralysis.
The researchers then grew motor neurons in a dish and showed how the drug protects these cells by increasing energy levels, which could act as a motor neuron disease treatment.
The researchers plan to invite 50 patients from the Oxford MND Care and Research Centre to participate in a feasibility study to understand whether this drug is a suitable motor neurone disease treatment.
The study is published in eBioMedicine.