Thousands of people living with motor neurone disease could be given the chance to live longer, thanks to a new drug which has been shown to slow the progression of the most common form of the degenerative illness.
Amyotrophic lateral sclerosis (ALS) is the most common form of motor neurone disease, a muscle wasting condition progressively damages parts of the nervous system and is incurable.
It gradually stops patients being able to move, talk, swallow and even eat.
But now scientists believe they may be able to slow down the progression of the disease whilst preserving motor function and prolonging survival with a twice-daily pill called pridopidine.
The sigma-2 receptor (SIR) antagonist has previously been shown to play a role in stimulating multiple neuroprotective pathways implemented in neurodegenerative diseases such as ALS and Huntington’s Disease.
So far, clinical studies have demonstrated its safety and efficacy with data from more than 1,600 patients, some of which have received active treatment for seven years.
Yesterday, the manufacturers of the drug, Prilemia Therapeutics and Ferrer, announced their first enrollment in the pivotal study in patients with rapidly progressive ALS.
Whilst it’s currently not approved by any regulatory authority, researchers believe the global study, which will involve more than 500 participants, could pave the way for therapeutic treatments that slow down the progression of the disease.
Dr Sabrina Paganoni, co-director of the Mass General Brigham neurological clinical research institute, said: ‘Enrolling the first participant in this confirmatory study is a milestone in our search for potential new therapeutic options that may help to preserve function, maintain speech and prolong survival – key aims of early ALS therapy.’
MND charities have welcomed the next stage of the trial, highlighting the need for early diagnosis and treatment.
Kuldip Dave, Senior Vice President of Research at the ALS Association said: ‘The ALS community urgently needs new treatment options that can delay the disease’s relentless progression, and awaits the outcome of this study.
‘The earlier we can diagnose and treat ALS, the greater the potential to preserve function and maintain quality of life for longer, which are key to making ALS livable until we can cure it.’
The PREVAiLS study is set to take place in up to 60 leading ALS treatment centers across 13 countries including the US, EU and the UK.
The 48-week placebo study seeks to enroll patients with definite or probable ALS who are within 18 months of their symptoms first appearing.
ALS claimed the life of Grey’s Anatomy star Eric Danes at just 53-years-old earlier this year and the acclaimed scientist Stephen Hawking famously suffered from it.
Danes, who played the role of Dr Mark Sloan, announced he had been diagnosed with ALS less than a year before he passed away.
Grey’s Anatomy and Euphoria fans reacted with shock and sadness at the news that star Eric Dane has passed away with a form of deadly motor neurone disease
The TV hunk is best known being Dr Mark Sloan, above with Dr Derek Shepherd, played by Patrick Dempsey, on Grey’s Anatomy from 2006
It is not fully understood why MND occurs and there are currently no treatments to halt its cruel march – instead doctors focus on alleviating the worst of the symptoms.
Around 5,000 adults in the UK have the condition and there is a one in 300 risk of developing it over the course of a lifetime.
The disease causes muscle weakness that gets worse over a few months or years,
Symptoms typically first include stiff or weak hands, weak less and feet which may cause someone to trip over a lot, and twitches spasms or muscle cramps.
As MND gets worse, a sufferer may experience problems breathing, swallowing and speaking. Eventually they will no longer be able to walk or move.
Life expectancy for about half of those with the condition is between just two and give years from the onset of symptoms. But these can worsen rapidly.
Some people may live for up to 10 years, and, in rarer circumstances, even longer.
