biomedicine shows “130% increase in survival”

Why doctor: Why is your research program, SECRET-GIFT, carried out thanks to the ACT4ALS-MND ​​Network (F-CRIN), a source of hope for patients suffering from amyotrophic lateral sclerosis (ALS)?

Pr David Devos : Amyotrophic lateral sclerosis is the most terrible of brain diseases because if the neurologist is not mistaken in the diagnosis, which is very rarely the case, we are doomed with a life expectancy of 3 years on average. We have been looking for so-called “neuroprotective” treatments for 50 or 60 years, not to cure it, but at least to significantly slow down its progression. Currently, there is only one treatment in the world that is capable of this: Riluzole. It increases survival by three months. We also have some hope with Tofersen but it only works for a very small group of patients who have a mutation in the SOD1 gene. This disease is therefore considerably lacking in treatments.

As a doctor-researcher, I took a different reasoning because I do not think that by acting on the Y receptor or the X pathway I will succeed in reversing a disease where everything is destroyed in two years.

How is this new therapy, which you patented with Professor Burnouf, innovative?

Currently, we do a lot of targeted therapy, that is to say we take a receptor and a molecule to try to treat patients. As a doctor-researcher, I took a different reasoning because I do not think that by acting on the Y receptor or the X pathway I will succeed in reversing a disease where everything is destroyed in two years. I had the idea of ​​working on this acellular biotherapy 14 years ago now, through a meeting with Professor Thierry Burnouf who was working on platelets. By discussing together, we realized their power: platelets are not only used for blood coagulation, they also have a function of protecting and repairing tissues thanks to the secretome. And we had the idea of ​​working on this repair system and creating a platelet lysate that could be injected into the brains of ALS patients. We started doing cell culture in 2012 and found that it worked very well in two classic ALS mouse models. Since then, we have of course continued the trials, we patented our innovation and launched the start-up InVenis Biotherapies, a spin-off from the Lille campus, in 2022 to carry out a first clinical trial on people.

How do you make this biomedicine, HPPL platelet lysate (“Human Platelet Pellet Lysate”)?

The idea is to recover platelets from healthy donors who reach the expiration date, i.e. on the 9th day, when transfusion in hospital is no longer possible. These platelets then undergo several freezing and thawing treatments in order to break the membranes. We also centrifugate and heat the product until we obtain a very clear liquid containing blood platelet growth factors and which is compatible with human administration into the central nervous system.

During our animal tests, we observed very strong neuroprotection each time we administered the right dose of product.

How would treatment be taken for people with ALS?

It is a long-term treatment. The idea is to inject the treatment directly into the patient’s brain using a very thin catheter placed behind the head and which only penetrates 3 cm into the brain. The system is connected to a small pump placed under the skin of the stomach to administer a specific dose. This requires intervention, but the risks of hemorrhage or infection are very low, as we have seen in patients with Parkinson’s disease who are treated in this way for the administration of dopamine.

What are the first results?

During our animal tests, we observed very strong neuroprotection each time we administered the right dose of product. We saw, in the most severe model of the disease, a 130% increase in survival, which is exceptional.

If all goes well, when could we expect to see this treatment on the French market?

We must launch the first clinical trial on 12 people in 2026-2027. Then, if all this goes well, we will launch a second one. But you should know that a well-done therapeutic trial always takes at least 3 to 5 years, because you have to be very careful, find the right dose for each patient, etc. So, in the best case scenario, I would say it will be in a little over 2 years for the first patients and 10 years for everyone who wants it.

Do you already know the characteristics of the patients who will be able to participate in this first trial?

With the ACT4ALS network research team, we already have an idea of ​​the criteria for these patients, but we are still in discussion. It’s quite complex because we have to be able to properly evaluate the drug and if we define criteria that are too strict, we will have more difficulty finding patients, but if it is very broad, the patients will be too heterogeneous… A criterion on which we are unanimous in, is that we need patients “at the beginning” of the disease, who progress at the same speed, and forms of ALS which are neither too fast nor too slow.

-

-

PREV Kenya tragedy: Man confesses to murder of 42 women
NEXT To lower electricity prices, the next government will have to change the rules