Drugs that increase vitamin A in the body could help stave off deadly motor neurone disease (MND), promising new research has suggested.
Scientists in Scotland discovered the medications, which target cells that activate vitamin A, may be therapeutic for diseases that lead to deterioration of the brain, like MND, sometimes called ‘locked in syndrome’.
This rare and incurable condition affects the brain and nerves, robbing sufferers of their ability to move, to eat and eventually breathe.
Experts believe the findings could help identify new drugs that could lead to fresh treatments.
Professor Peter McCaffery, study lead and medical sciences chair at the University of Aberdeen, said: ‘We discovered that these drugs bind and turn on the “retinoic acid receptor”, a key protein involved in activation of vitamin A in the body.
Scientists in Scotland discovered the medications, which target these specific receptors, may be therapeutic for diseases that lead to deterioration of the brain, like MND. Footy great Neale Daniher was diagnosed with MND in 2013
This rare and incurable condition affects the brain and nerves, robbing sufferers of their ability to move, to eat and eventually breathe. Pictured, Neale Daniher in June 2024
‘Our research provides the first steps to identify new targets for drugs that may then lead to future therapies.’
Around 5,000 adults in the UK have MND and there is a one in 300 risk of developing the condition over the course of a person’s life.
Life expectancy for about half of those with the condition is between just two and five years from the onset of symptoms. But these can worsen rapidly.
Muscle twitches and a weak grip are among the early signs of the condition, along with weakness in the leg or ankle, slurred speech and weight loss.
Some tremors are normal and can be caused by caffeine, stress and age.
There is no cure but doctors can provide treatments to help reduce the impact it has on a person’s life.
Footy great Neale Daniher was diagnosed with MND in 2013 and has dedicated his remaining years to raising awareness and money for the disease.
Every year he takes centre stage at the MCG for the Big Freeze game, in which a raft at celebrities take the plunge in ice cold water to raise money for research and prevention.
Recalling the research process, study co-author and neuroscientist at the University of California, Azita Kouchmeshky, said: ‘We tested these drugs in a series of studies on neurons grown in a dish.
‘Chemicals were added to the neurons that caused harm similar to the changes that occur in diseases such as MND or Amyotrophic lateral sclerosis (ALS).
‘Usually, these chemicals will cause the neurons to die. However, the application of the drugs that bind to the retinoic acid receptor significantly reduced the number of cells that died off.
‘The same drugs were also tested in mice and were found to induce changes that suggest they may also be effective in the body.’
The research paper was published in Frontiers in Neuroscience.
Andy Whiting, chief executive of Nevrargenics Ltd and emeritus professor at Durham University, designed and synthesised the drugs the team used.
He said: ‘There is a total lack of genuinely disease-modifying drugs out there for neurodegeneration in general, and ALS specifically.
‘We are committed to changing that and delivering hope for MND sufferers in the first instance.
‘This is one further step along the road to deliver new therapeutics for such globally challenging diseases.’
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