MSDAVENIR and Inserm sign a strategic framework agreement

Cyril Schiever, Chairman of the Board of MSDAVENIR, Professor Yves Lévy, Chairman and CEO of Inserm and Pascale Augé, President of the Executive Board of Inserm Transfert, today signed a strategic framework agreement to support French medical research.This event took place under the patronage and in the presence of Marisol Touraine, French Minister for Social Affairs and Health, and Thierry Mandon, Secretary of State for Higher Education and Research.

signature convention Inserm MSDAvenir©Philippe Chagnon – Essop

The framework agreement between Inserm and MSDAVENIR: a public-private partnership approach to support health research
It will help support research projects of excellence covering:
– oncology, personalized medicine and translational research
– Inserms’s Major Transversal Programs (GPT): aging, microbiota, genomic variability in patients and its contribution to the natural history of human diseases

This framework agreement reflects the desire of MSDAVENIR to make a significant commitment to medical research in France. “This agreement cements the founding ambition of MSDAVENIR: to invest differently in France to further scientific and medical knowledge. Through this collaborative approach with Inserm, MSDAVENIR forms part of a dynamic for ‘open research and innovation’, driven and supported by the French government. We are particularly honored today by the presence of the Minister for Health and the Secretary of State for Research, an indication of their support for this endeavor” declared Cyril Schiever, Chairman of MSDAVENIR.

“The agreement we are signing today with MSDAVENIR is fully in line with three of Inserm’s major objectives: foster the medical and scientific attractiveness of France; develop public-private partnerships within a framework of open innovation; and focus efforts onto domains of excellence in fundamental and translational research serving new precision medicine” added Yves Levy, Chairman and CEO of Inserm.

“It’s a great honor for Inserm to witness today the culmination of this framework agreement in the presence of the Minister of Social Affairs and Health and the Secretary of State for Higher Education and Research.”

“France is currently the leader in terms of innovation in health. Everywhere I look I see great enthusiasm, the dynamism of start-ups, and a shared ambition to innovate in order to improve patient wellbeing. Signing this framework agreement will help us further expand this movement” declared Marisol Touraine, Minister of Social Affairs and Health.

“We must strengthen partnerships between academia and the pharmaceutical world; this way, our research will progress and be more highly valued, therefore increasing our attractivity” said Thierry Mandon, the Secretary of State for Higher Education and Research.

See the first partnership with the Marseille Immunopôle to reinforce the leadership of French research in immuno-oncology


MSDAVENIR is a research support fund in the life sciences that was set up in March 2015 and endowed with €75 million for a three-year period. Through the signing of partnerships, this fund aims to drive research in both scientific areas and societal fields related to research, education or health.
Contact: Stéphanie Martel – moc.kcrem@letram.einahpets

About INSERM –
Set up in 1964, the National Institute for Health and Medical Research (INSERM) is a Public Scientific and Technical Research Establishment supervised jointly by the Ministry for Higher Education and Research and the Ministry for Health. The vocation of its scientists is study all diseases, from the most common to the most rare, through their work in biological and medical research and population health.
Contact: Priscille Rivière – rf.mresni@esserp – +33 (0)1 44 23 60 97

About Inserm Transfert –
Inserm Transfert SA is a private subsidiary of Inserm founded in 2000 that is responsible for the institute’s valorization mission. It manages the entire transfer of technologies and knowledge from Inserm’s research laboratories towards health industries – from invention disclosure and the maturation of innovative projects to the signing of industrial partnerships.
Contact: Céline Cortot – rf.trefsnart-mresni@totroc.enilec – +33 (0)1 55 03 01 68

An effective drug for myoclonus-dystonia, a rare disease of the nervous system

A team coordinated by Prof. Emmanuel Flamand-Roze from Pitié-Salpêtrière Hospital, AP-HP, has tested, at the clinical investigation centre of the Brain and Spine Institute (Inserm /CNRS/UPMC) , the efficacy of zonisamide, a drug currently used to treat certain forms of epilepsy, in 23 patients with a rare disease of the nervous system, myoclonus-dystonia. The promising results from this trial, which was funded by AP-HP (the Paris Public Hospitals), are the subject of a publication in the journal Neurology on 6 April 2016.

Healthy Brain Pills

(c) Fotolia

Myoclonus-dystonia is a rare disease that results from poor control of movements by the brain, leading to abnormal muscle contractions. It features two types of symptoms: jerky muscular movements (myclonus), and an abnormal posture of some parts of the body (dystonia). The unpredictable muscular jerks that accompany every one of the patient’s movements are the most disabling symptom. They are usually strongest in the upper limb and neck regions. The mobility problems associated with this disease can seriously impede the activities of daily living. These highly visible disorders often lead to stigmatisation, loss of self-esteem, and social withdrawal among patients. There is presently no effective drug for this disease. There is, however, a neurosurgical treatment that gives good results, but it is invasive, and restricted to severe forms of the disease.

The scientific team, made up of physicians and researchers, conducted a randomised, double-blind, placebo-controlled trial, to test the efficacy of zonisamide in 23 patients with myoclonus-dystonia. Zonisamide is a drug that has been used in Europe for the last ten years to treat certain forms of epilepsy. It is well tolerated by most patients who use it for this purpose.

The results of this trial show that zonisamide brings about a highly significant reduction in the myoclonus and associated disability. The patients’ dystonia is also alleviated by this treatment.

“This is the first trial to demonstrate the efficacy of a drug against this disease,” explains Prof. Flamand-Roze. “Zonisamide may therefore be offered to patients with mild and moderate forms of myoclonus-dystonia, and to all patients who choose not to have, or who cannot have neurosurgical treatment.”

Jumping genes: all guilty?

Transposable elements, also known as “jumping genes” are DNA fragments that can move or copy themselves from one location to another on the chromosomes. They have invaded the genomes of most living organisms, from bacteria to humans, via the plants. When they jump, they bring about complex modifications in genes near which or in which they insert themselves, and can thereby alter or abolish their function. This phenomenon contributes to the evolution and adaptation of species.

However, in the shorter term, “jumping genes” can have harmful effects. In humans, the only currently active transposon family, the LINE-1 type retrotransposons, causes new cases of genetic diseases, such as haemophilia and muscular dystrophy. This is why their activity is normally under tight control. However, in nearly half of epithelial cancers, they manage to escape the many cellular defence mechanisms that protect our DNA, and jump actively, contributing to the emergence and progression of cancers. Moreover, in studies they are often used as tumour biomarkers for diagnostic or prognostic purposes.

DNA Structure

(c) Fotolia

One of the main problems raised by the study of “jumping genes” is related to their extremely repetitive nature. Our DNA contains thousands of copies of them, almost identical to one another, and every individual contains hundreds of copies that are not listed in the reference map of the human genome. Furthermore, until now, it had been impossible to know whether activation of “jumping genes” results from a general disruption, leading to massive mobilisation of all copies, or whether, on the contrary, only a small number of them manage to escape the protective controls. Through a new approach, published in the journal eLife, involving high-throughput sequencing, genomics, epigenomics and bioinformatics, the team led by Gaël Cristofari, Inserm Research Fellow, and his collaborators at Unit 1081, “Institute for Research on Cancer and Aging, Nice (IRCAN),” has succeeded in measuring the activity of the “jumping genes” in normal and cancer cells with unprecedented resolution.

According to their results, only a small number of copies are really guilty: those located in permissive regions of our chromosomes. And these regions vary depending on the type of cell. Furthermore, all these active copies are not present in all individuals!

“The important concept here is that the small group of LINE-1 transposons that escapes control is different from one cell type to another: in certain cancers, one group is important, in another type of cancer, it will be another group of copies. This observation suggests that behind each LINE-1 group, there is a mechanism and signals specific for a particular type of organ or tissue,” explains Gaël Cristofari.

These results provide a better understanding of how new mutations may emerge, they suggest the existence of genetic factors behind this phenomenon, and they provide new data for the rational use of LINE-1 retrotransposons as biomarkers in oncology, by focusing on the active copies in a given type of cell.


This work was made possible by financial support from ARC French Foundation for Cancer Research, Fondation pour la Recherche Médicale, Cancéropôle PACA, the European Research Council, the French National Research Agency (ANR) (Labex Signalife), and the Research Group on Mobile Genetic Elements (CNRS, GDR 3546).