An exclusive license agreement has been signed between SATT Nord and the startup Invenis Biotherapies for the development of a new innovative biotherapy for the treatment of neurodegenerative diseases, particularly amyotrophic lateral sclerosis.
Amyotrophic lateral sclerosis (ALS), also known as Charcot's disease, is a serious and debilitating neurodegenerative disease caused by the progressive death of motor neurons. It results in progressive paralysis of the muscles involved in voluntary movement. It is a fatal disease with an average survival time of 3 to 5 years and affects more than 150,000 people worldwide. The incidence is very high in France, where there are 5 new cases and 5 deaths per day. In most cases, it is the damage to the respiratory muscles that causes the death of patients.
The research effort dedicated to it in recent years has significantly advanced knowledge about its genetics and biology.
To date, there is no truly curative treatment available, and the only treatment available is primarily aimed at slowing progression, relieving symptoms, and improving patients' quality of life, with a very modest effect on paralysis and life expectancy.
Most therapeutic strategies have failed in clinical trials, probably due to extensive damage to the central nervous system that cannot be accessed with local, single-site transplantation, or are ineffective, particularly biotherapies based on modified stem cells, as they are generally based on a single molecule targeting a single signaling pathway, whereas the underlying pathophysiological mechanisms of neurodegeneration are multiple and complex.
This suggests the need to move towards a therapeutic strategy that acts on a large number of targets (pleiotropic treatment).
Recent work resulting from a collaboration between David Devos, neurologist and pharmacologist at Lille University Hospital, professor at the University of Lille, director of the INSERM DVCD INSERM research team at the LILNCOG laboratory (Lille Neuroscience & Cognition) and Professor Thierry Burnouf, researcher at the Graduate Institute Biomedical Materials and Tissue Engineering at Taipei Medical University, has demonstrated the therapeutic potential of platelet derivatives for treating chronic neurodegeneration, such as ALS, Parkinson's disease, or neurodegeneration induced by trauma or stroke, and have enabled the development of a powerful biotherapy based on the natural repairing power of human blood platelets.
These platelet derivatives, obtained from healthy donors, from platelet concentrates not used in transfusions, consist of the secretome of blood platelets, an abundant physiological source of trophic factors (growth factors (NTF, VEGF, PDGF, BDNF, etc.), neurotransmitters, neuromodulators, anti-inflammatory and antioxidant proteins, etc.) that regulate the development, maintenance, function, and plasticity of the central nervous system, could protect neurons from death.
After more than 10 years of interdisciplinary and translational research, a unique platelet lysate preparation (HPPL, Human Platelet Pellet Lysate) has been developed that is safe and compatible with the central nervous system, using a patented clinical-grade process.
HPPL, now recognized as a novel pleiotropic biotherapy, is unique in the world because it overcomes all the challenges that make other platelet preparations incompatible with administration into the central nervous system, while providing significant neuroprotection in animal models of Parkinson's disease, stroke, and ALS.
Continuous, controlled, and safe intracerebroventricular administration, using a very fine catheter placed in a cerebral ventricle (small pocket of water) connected to a pump placed under the skin in the abdomen, will allow complete biodistribution of the central nervous system over time and overcome the problems of action time and targeting of modified stem cells.
Thanks to the recent award of €8.3 million in RHU (University Hospital Research) "SECRET-GIFT" grant of €8.3 million, the current objective is to demonstrate the feasibility, safety, and initial efficacy data of HPPL biotherapy with continuous i.c.v. administration in the first 12 patients with early-stage ALS.
"For the first time, we have a pleiotropic therapeutic approach that has the potential to slow neurodegeneration in the long term and provide effective treatment for millions of patients with neurodegenerative diseases, particularly those affected by ALS." Prof. David Devos.
This project was supported and financed by SATT Nord to the tune of €430,000, enabling the recruitment of a development engineer and the implementation of an intellectual property and market access strategy. It was licensed to the startup Invenis Biotherapies in 2023.
The HotGift project, led mainly by professors David Devos and Thierry Burnouf, was monitored from the outset by Matthieu Fisichella, then health project manager at SATT Nord, and resulted in three patent applications being filed.
The quality of the project, its clinical interest, and the relationship of trust that had developed over the months with Professor David Devos quickly convinced Matthieu Fisichella to create Invenis Biotherapies in June 2021 as its founder and president. On July1, 2023, Boris Molle, former head of technology transfer at SATT Nord, was called upon to succeed Matthieu Fisichella as CEO of Invenis Biotherapies. François-Xavier Denimal, Head of the Health Business Unit at SATT Nord
The preclinical results obtained on various neurodegenerative models (Parkinson's disease, ALS, traumatic brain injury, etc.) demonstrated the ability of platelet lysate to target the various signaling pathways (oxidative stress, neuroinflammation, cell survival, etc.) that lead to neurodegeneration.
"Platelet-based therapeutic applications are becoming increasingly widespread, whether for cell therapy as a culture medium that is gradually replacing FCS (fetal calf serum), regenerative medicine (tissue or bone repair), or even the treatment of dry eye syndrome. Invenis Biotherapies is the only company developing a therapy based on platelet derivatives for the treatment of neurodegeneration." Prof. Thierry Burnouf.
"The growing demand for platelet lysates heralds a change in the blood products industry, comparable to that of the 1980s when the plasma derivatives production chain was organized, characterized by the optimal use of whole blood donations and the possible implementation of dedicated platelet collection for the production of platelet lysates for the treatment of degenerative diseases. We aim to play a key role in this evolution, enabling every blood collection to be put to good use, in accordance with established ethical rules. We make it a point of honor to contribute significantly to the development of innovative biotherapies for the treatment of neurodegeneration, using platelet derivatives and ensuring ethical and responsible management at every stage of the process," Boris Molle, CEO of Invenis Biotherapies.