Drugs that increase vitamin A in the body could help prevent the deadly motor neurone disease (MND), promising new research suggests.
Scientists in Scotland have discovered that drugs that target cells that activate vitamin A may be therapeutic for diseases that lead to brain deterioration, such as ALS, sometimes called “locked-in syndrome.”
This rare and incurable disease affects the brain and nerves, depriving those affected of their ability to move, eat and eventually breathe.
Experts believe the findings could help identify new drugs that could lead to new treatments.
Professor Peter McCaffery, who led the study and is chair of medical sciences at the University of Aberdeen, said: ‘We found that these drugs bind to and activate the ‘retinoic acid receptor’, a key protein involved in activating vitamin A in the body.
Scientists in Scotland have discovered that drugs that act on these specific receptors may be therapeutic for diseases that cause brain deterioration, such as ALS. Football star Neale Daniher was diagnosed with ALS in 2013.
This rare and incurable disease affects the brain and nerves, depriving those affected of their ability to move, eat and ultimately breathe. Pictured, Neale Daniher in June 2024
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‘Our research provides the first steps toward identifying new drug targets that could then lead to future therapies.’
Around 5,000 adults in the UK have ALS and there is a one in 300 risk of developing the disease over a person’s lifetime.
For about half of those with the disease, the life expectancy is between two and five years from the onset of symptoms, but these can worsen rapidly.
Early signs of the condition include muscle spasms and a weak grip, along with weakness in the leg or ankle, difficulty speaking 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.
Football great Neale Daniher was diagnosed with ALS in 2013 and has dedicated his remaining years to raising awareness and funds for the disease.
Every year, it takes centre stage at the MCG for the Big Freeze match, where a raft of celebrities is submerged in freezing water to raise money for research and prevention.
Recalling the research process, study co-author and University of California neuroscientist 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 damage similar to changes that occur in diseases such as ALS or amyotrophic lateral sclerosis (ALS).
‘Normally, these chemicals cause the death of neurons. However, the application of drugs that bind to the retinoic acid receptor significantly reduced the number of cells that died.
‘The same drugs were also tested in mice and found to induce changes that suggest they might also be effective in the body.’
The research article was published in Frontiers in neuroscience.
Andy Whiting, chief executive of Nevrargenics Ltd and emeritus professor at Durham University, designed and synthesised the drugs used by the team.
He said: ‘There is a complete lack of truly disease-modifying drugs for neurodegeneration in general, and for ALS in particular.
‘We are committed to changing that and providing hope to ALS patients in the first instance.
“This is another step towards developing new therapies for diseases that pose a global challenge.”
