Breakthrough in the fight against fatal motor neurone disease – thanks to a VITAMIN

Drugs that increase the amount of vitamin A in the body could help prevent fatal motor neurone disease (MND), according to promising new research.

Scientists in Scotland have discovered that the drugs, which target cells that activate vitamin A, could have therapeutic effects in diseases that cause brain decline, such as ALS, also known as ‘locked in syndrome’.

This rare and incurable condition attacks the brain and nerves, leaving patients unable to move, eat and eventually breathe.

Experts believe the findings could help identify new drugs that could lead to new treatments.

Professor Peter McCaffery, head of the research and head of medical sciences at the University of Aberdeen, said: ‘We found that these drugs bind to and activate the ‘retinoic acid receptor’, an important protein involved in the activation of vitamin A in the body.

Scientists in Scotland have discovered that the drugs, which target these specific receptors, could be therapeutic for diseases that cause brain deterioration, such as MND. Football legend Neale Daniher was diagnosed with MND in 2013

This rare and incurable condition attacks the brain and nerves, leaving patients unable to move, eat and eventually breathe. Pictured, Neale Daniher in June 2024

‘Our research is the first step towards identifying new drug targets, which could then lead to future therapies.’

Around 5,000 adults in the UK have ALS and the chance of someone developing the condition during their lifetime is one in 300.

The life expectancy of about half of people with the condition is only two to five years from the onset of symptoms. But they can worsen rapidly.

Muscle twitching and a weak grip are some of 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 offer treatments that can reduce the impact of the disease on a person’s life.

Football legend Neale Daniher was diagnosed with ALS (MND) in 2013 and is spending his remaining years raising awareness and money for the disease.

Every year he takes centre stage at the MCG for the Big Freeze game, which sees a raft full of celebrities take a dip in icy water to raise money for research and prevention.

Azita Kouchmeshky, a neuroscientist at the University of California and co-author of the study, recalls the research process like this: “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 the changes that occur in diseases such as ALS or MND.

‘Normally, these chemicals cause neurons to die. However, using 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 were found to produce changes that suggest they might be effective in the body as well.’

The research article was published in Frontiers in neuroscience.

Andy Whiting, Managing Director of Nevrargenics Ltd and Emeritus Professor at Durham University, designed and synthesised the drugs the team used.

He said: ‘There is a complete lack of truly disease-modifying drugs for neurodegeneration in general and ALS in particular.

‘We are committed to changing that and, first and foremost, offering hope to ALS patients.

“This is a next step towards new therapies for such globally challenging diseases.”