A new treatment pioneered in France may prolong the life expectancy of people with amyotrophic lateral sclerosis (ALS), a fatal motor neurone disease.
The treatment involves injecting blood platelets straight into the brain through a very narrow pump and catheters.
An operation would take place to insert the catheters, taking around three hours, and patients would be able to leave the same day. Then, further injections could be made using the catheters in the following weeks.
“The main potential difficulty is the dose as I am very, very confident that the effects of the treatment will be positive,” said David Devos, 53, a neurologist, neuroscience researcher and professor of medical pharmacology from the Centre Hospitalier Universitaire de Lille, who is working on the new treatment.
The idea came about after chatting in the garden with his neighbour Thierry Burnouf, a blood specialist, around 12 years ago.
Through these discussions, Prof Devos understood that blood platelets, which are mostly known for making blood clots, also have other functions.
Prof David DevosDavid Devos / Inbrain Pharma
He theorised that the inside of platelets (removing the membrane which causes an inflammatory reaction that could be dangerous for patients) may be used to heal various illnesses, including ALS, thanks to their reparatory powers.
At first, other medical experts thought the idea was “crazy”.
“It was rewarding to be innovators but it also made things very difficult because no one believed in us or our work,” Prof Devos said.
Tests initially took place on mice which had ALS, Parkinson’s, mental trauma and similar issues, and the results proved the concept.
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“In the most severe cases of ALS, we were able to prolong life expectancy by 130% in the mice,” said Prof Devos.
To continue tests, he launched a start-up called InVenis Biotherapies and received €8.3million in funding.
Now, Prof Devos hopes to get further financial support to start clinical trials on humans and to industrialise production of the treatment.
Before that, toxicology reports of the treatment on animals such as pigs will need to be made to secure permission to start the clinical trials.
Prof Devos hopes the treatment will not only double the lifespan of those with ALS, but also improve their quality of life and give them more autonomy.
If everything goes well, the treatment could become widely available by 2040.
ALS (known as maladie de Charcot in France and as Lou Gehrig disease in the US after the baseball player who died from it) is a motor neurone disease which affects the nerve cells in the brain and spinal cord.
Often beginning with muscle twitching and weakness in arms or legs, it progresses very fast and the median life expectancy after diagnosis is three years. It typically leads to muscle atrophy.
Some 8,000 people are affected by the disease in France, estimates Prof Devos.
“It might seem rare, but every day around three people are diagnosed with it and three people die from it, leading to about a thousand deaths and new cases every year,” he told The Connexion.
There is no single “villain” when it comes to the causes of ALS, although genetic factors can make people more susceptible to it.
Possible risk factors also include exposure to lead, pesticides, and other environmental toxins.
“It is a combination of a genetic load and an environmental load that leads to the development of the illness,” said Prof Devos.
“The main obstacle to the treatment has not been medically related – it is because of funding and regulations.”
ALS became more widely known following the ice bucket challenge, which went viral on social media in 2014. This encouraged nominated participants to be filmed having a bucket of ice water poured on their heads and then nominating others to do the same to promote awareness of the disease and encourage donations to research.
