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The Consortium – Individual Members
Trophos S.A.
Trophos is a biopharmaceutical company committed to the discovery and development of novel therapeutics to treat neurodegenerative diseases. It was founded in 1999 by a group of entrepreneurs and academic scientists as a spin off of the laboratory of Professor Christopher Henderson, a world expert in motor neuron biology and diseases. The Trophos drug discovery strategy involves the phenotypic screening of small molecules to identify those that confer a survival benefit in models of primary neuron subject to a physiopathological relevant stress. In essence, compounds are selected for their ability to improve survival of “sick” neurons. Once “hits” are validated for their efficacy they are use to identify their molecular targets and mechanism of action. This ‘black box’ approach has proven particularly effective in the case of TRO19622 olesoxime which is the most advanced clinical product at Trophos, having successfully completed phase 1 clinical trials. Through its discovery platform and the recruitment of a team of senior managers coming from the pharmaceutical industry, Trophos has acquired considerable experience of drug discovery and development in the neurodegenerative domain mostly for orphan indications such as SMA, ALS & HD.
Department
In vitro pharmacology
Clinical research
Preclinical development
Role
Coordinator WP6, WP7, WP8 and WP9 leader
Contact
Julien Veys
+33 4 91 82 82 82
Université de la Méditerranée
The Mediterranean Institute of Neurobiology (INMED) located in Marseille, France, belongs to the Université de la Méditerranée and the French Medical Research Council (INSERM). Inaugurated in 2004, the INMED gathers 15 research teams (over 1 000 reserachers) with all the facilities required to perform post-genomic studies on brain development, synaptic plasticity, brain oscillations, as well as neurological disorders including neurodegenerative disorders as AD, PD and MND.
Professor Brigitte Pettmann is senior scientist in the INMED. Her team belongs to Cédric Raoul’s group which has been particularly associated with discoveries in the fields of motoneuron biology and neuronal death during development and in pathologies (ALS and SMA). The group showed that motoneurons can use a specific death pathway triggered by the activation of the death receptor Fas, and that motoneurons from mice models of ALS show an increased sensitivity to this death pathway, achieved through an amplification loop. Activation of the pathway has since been demonstrated to occur in ALS patients including sporadic forms.
Professor Jean Pouget is Head of the Neurology and Neuromuscular Diseases Department at the University Hospital Centre of the Université de la Méditerranée – La Timone. Prof. Pouget and his team are one of the French reference centres for the treatment of Amyotrophic Lateral Sclerosis. Prof. Pouget was investigator in a number of clinical trials in ALS including phase 1b study of TRO19622 olesoxime in collaboration with Trophos and Prof. Vincent Meininger (APHP-PSL).
Department
INMED - Motoneuron pathology: death signalling and gene therapy perspectives team
APHM – Service de neurologie et des maladies neurologiques
Role
WP4 leader, B. Pettmann Head of BRSC Clinical trial’s principal investigator in Marseille
University of Sheffield
Professor Pamela Shaw is Director of the Sheffield Care and Research Centre for Motor Neuron Disorders, part of the Academic Neurology Unit. Professor Shaw’s group consists of a multidisciplinary team of basic and clinical scientists investigating molecular mechanisms of neurodegeneration in disorders of the human motor system, particularly ALS and HSP. Current areas of research activity include the following: molecular pathology including SOD1 related familial ALS, genetics of human motor system disorders, characterisation of the cell specific molecular features of motor neurones, cellular models of ALS and exploration of related mitochondrial dysfunction.
The Sheffield Care and Research Centre for Motor Neuron Disorders is also one of the reference centres for the treatment and clinical research on ALS in the UK. Clinical research is focused on i) the study of CSF biochemical markers, aiming to define potential biochemical subgroups of patients and to correlate these parameters with clinical response to therapy with the neuroprotective anti-glutamate agent riluzole, and ii) the evaluation of new therapeutic agents in patients with ALS. Recent multinational trials have included evaluation of riluzole, xaliproden and intrathecal brain derived neurotrophic factor.
Department
Academic Neurology Unit
School of Medicine and Biomedical Sciences
Role
WP1 leader (Andrew Grierson) WP3 leader (Pamela Shaw) Clinical trial’s principal investigator in Sheffield
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Eberhard Karls Universität Tübingen
The Department of Medical genetics is among the largest departments of medical genetics in Germany and comprises three basic sections: (i) clinical genetics, (ii) cytogenetics and (iii) research. The department has moved to cutting edge equipped new facilities in 2001 and employs currently a full time staff of more than 60 persons.
Under the leadership of Prof. Olaf Riess, the Department of Medical Genetics has coordinated and performed outstanding worldwide-recognised research in the field of neurodegenerative diseases such as Huntington's disease, Parkinson's disease, dystonias and spinocerebellar ataxias. For all of these diseases, a wide range of different transgenic and/or knock out models (more than 30 models) have been created. This team has been using these models for investigation of the pathogenesis, for therapeutic trails and for studying gene expression networks of the brain by Affymetrix whole genome expressioon arrays. Complementary, a large sample of patients with the respective diseases is being investigated for specific expression changes in the blood (profiles) which might be useful for the development of biomarkers studying disease progression.
Department
Medizinische Genetik Tübingen
Institut für Humangenetik
Role
WP5 leader (Olaf Riess)
Johann Wolfgang Goethe Universität Frankfurt am Main
The Institute of Pharmacology from the Johann Wolfgang Goethe-University of Frankfurt (GUF). provides excellent research facilities for the qualitative and quantitative analysis of drug effects on parameters related to its key expertise such as mitochondrial function, oxidative stress, membrane biology, cholesterol homeostasis, apoptosis, neuronal restoration and genetic basis for neurodegenerative diseases. Several primary and transgenic cell lines and laboratory animals are used as model organisms of neurodegenerative diseases. The Institute of Pharmacology is member of the Center for Drug Research, Development and Safety (ZAFES; www.zafes.com), GUF, and thus, has access to a multitude of first-class research
Department
Pharmakologisches Institut für Naturwissenschaftler
Role
WP2 leader (Walter Muller)
Assistance Publique – Hôpitaux de Paris La Pitié Salpêtrière
Professor Vincent Meininger is Professor of neurology and one of the most recognized specialists of ALS in the world; he is also the leader of the European network of clinicians involved in the MitoTarget project. Professor Meininger is head of the ALS Reference Centre at La-Pitié Salpêtrière Hospital in Paris. This centre leads and coordinates the 17 reference centres for the treatment of ALS in France. With a team composed of hospital practitioners, neurologists and pneumologists, and of a large trained paramedical staff (nurses, dieticians, occupational therapist, kinesitherapist, speech-language pathologists, neuropsychologist), the ALS reference centre at La-Pitié Salpêtrière provides caring and follow-up to more than 900 patients per year, making of it the largest ALS care centre in France and in Europe. Through a close and constant cooperation with the clinical pharmacology department along with cooperation with other European sites participating to the MitoTarget project, La-Pitie Salpêtrière ALS Centre allowed the development of the only approved drug for the treatment of ALS (Riluzole) and contributed to more than two third of the international and European clinical trials recently carried out
Department
Fédération des Maladies du Système Nerveux
Role
Head of CRSC
Clinical trial’s principal investigator in Paris
Centre Hospitalier Universitaire de Nice
Professor Claude Desnuelle is Head of the Physical Medicine and Rehabilitation Department at the University Hospital Centre of Nice. Prof. Desnuelle and his team are one of the French reference centres for the treatment of Amyotrophic Lateral Sclerosis.
Department
Hôpital l’Archet 1
Pôle NeuroSciences : Médecine Physique et de Réadaptation
Role
Clinical trial’s principal investigator in Nice
Charité Universtitätsmedizin Berlin
Professor Thomas Meyer is Head of the Motor Neuron Diseases clinical research at the Charité Medical School. The main interest of the group is in the pathogenesis and genetics of amyotrophic lateral sclerosis (ALS). Clinical trials on novel treatment options in ALS are an integrated part of the ALS out-patient clinic, in which more than 100 patients are treated. The ALS out-patient service represents a clinical trial center of three phase II-trials for the treatment of ALS. The clinic participated to several other trials including the placebo-controlled trial of the efficacy of pentoxifyllin in combination with riluzole (protocol EHT201), a second phase II-study of the efficacy of a novel substance showing modulating properties on glial cells using a placebo-controlled and double-blind design (Protocol ONO2506) and placebo-controlled, double-blind and multi-center clinical trial for the investigation of the novel compound coded TCH346.
Department
Ambulanz für ALS and andere Motoneuronenerkrankungen
Role
Clinical trial’s principal investigator in Berlin
Medizinische Hochschule Hannover
Professor Dengler is head of the Neurology department at the Centre for Neurological Medicine and an expert in ALS and Clinical Neurophysiology. The ALS/MND consultation at MHH is recognized as a centre of excellence in Germany, and the neurology clinic covers most of the northern Germany. The Neurology department participated to a number of clinical studies in collaboration with other renowned centres in Germany and Europe. The latest of those studies is the placebo-controlled and double-blind design (Protocol ONO2506).
Department
Klinik für Neurologie
Role
Clinical trial’s principal investigator in Hannover
King’s College London Institute of Psychiatry
The Institute of Psychiatry at King’s College London (KCL) is part of the University of London. The research described within MitoTarget will take place within the Medical Research Council's (MRC) Centre for Neurodegeneration Research (MRC-CNR) at the IoP. The MRC-CNR laboratories are newly equipped following major funding from government, MRC (including MRC Centre status grant) and Wellcome Trust awards. Research in Chris Miller’s lab focuses on defective axonal transport in neurodegenerative diseases, particularly ALS and Alzheimer’s disease. Our approaches are to monitor the effects of ALS- and Alzheimer’s disease-associated insults on axonal transport of defined cargoes, and then to understand the molecular mechanisms underlying any defects in transport. Resources include laboratories for cell and molecular neuroscience including advanced microscopy, and advanced mass spectrometry for monitoring changes in post-translational modification of proteins
The Department of Clinical Neuroscience hosts the King's Motor Neurone Disease (MND/ALS) Care and Research Centre, with >200 new MND/ALS referrals each year. The MND Research Group group makes a major contribution MRC-CNR with Prof Leigh heading the clinical research group. Prof Leigh’s team has participated in/taken leadership roles (e.g., member of steering committees) in all the major European clinical trials in ALS/MND since 1993 and was PI for an EU-funded international trial of riluzole in parkinson plus syndromes ('NNIPPS'). He chairs the Trial Steering Committee for the MRC CUPID study in MS, and is member of the Independent Data and Safety Monitoring and Board for the NINDS-funded trial of ceftriaxone in ALS.
Department
MRC Centre for Neurodegeneration Research
Role
Katholieke Universiteit Leuven
Professor Robberecht is Professor of Neurology and Head of the Division of Experimental Neurology at the Katholieke Universiteit Leuven (Belgium). Prof Robberecht’s department is one of the largest departments in Europe combining both basic and clinical research activities. Clinical research is led in the Neuromuscular disease Reference Center (NMRC) at Gasthuisberg University Hospital. Regularly the neuromuscular diseases care programme participates in clinical trials in search for more effective treatments for neuromuscular diseases, e.g. for amyotrophic lateral sclerosis.
Department
Vesalius research center
Role
Clinical trial’s principal investigator in Leuven
Martin-Luther-Universität Halle-Wittenberg
Professor Zierz is Head of the Neurology Clinic of the University of Halle-Wittenberg. This clinic includes two specialised institutes the Muscle Center and the Muscle Lab. These two complementary institutes were opened in 1995 with the support of the German Association for Muscular Disorders (Deutsch Gesellschaft für Muskellkranke). The Muscle Center receives patients and allows them to benefit from the coordinated action of a multidisciplinary team providing high standard care. The Muscle Lab provides diagnostic support to the Muscle Center practitioners through molecular biology, histology, biochemical and functional analysis. More specifically and in relationship with MitoTarget, the Muscle Lab has in hands all the standard techniques required to assess the bioenergetic state of a patient.
Department
Klinik und Polykilinik für Neurologie
Role
Clinical trial’s principal investigator in Halle (Saale)
Centre Hospitalier Régional Universitaire de Montpellier
Professor Camu’s department is a reference center on ALS since 1988 and labeled as such by the French ministry of health since 2003. All key evaluations of ALS patients are done on site (spirometry, muscle testing, biology, oxymetry, ECG etc.). The group participated, since 1992 to all the multicentre placebo-controlled double blind studies in ALS, Phases II, III and IV, done in Europe. Equipment, on site, for therapeutic trials, is complete with independent ADSL access, freezers, centrifugers and temperature monitored rooms for treatment storing.
Prof Camu is member of the ALS collaborative group in France. All the member centres are using the same standard procedures for diagnosis and are trained for using also standard modes of evaluation
As reference centre in ALS, participating to any research strategy for treating patients is one of our main goal. As the largest center in France (at the exception of Paris), the CHRU de Montpellier have a large number of patients who are willing to participate in such trials.
Department
CHU Gui de Chauliac
Service Explorations Neurologiques
Role
Clinical trial’s principal investigator in Montpellier
Centre Hospitalier Régional Universitaire de Lille
The reference centre for the Motor-Neurone Diseases of Lille was recognised as regional reference centre for Amyotrophic Lateral Sclerosis (ALS), by a DHOS letter of 24/12/2002. It is integrated to the neurology and movement pathologies department of Pr. Destée, within the neurology section of the hospital “Roger Salengro”, at the CHRU de Lille. It groups physicians (neurologist, palliative care physician, pneumologist, physical medicine physician and nutritional physician), a psychologist, an ergotherapist, a medical secretary and physiotherapist. All in relation with the other departments of the neurology section, and the section of respiratory diseases, the nutrition and home-nutritional support department, the polyvalent intensive care department, and the mobile team of palliative care of the CHU de Lille. In 2007, the number of patients followed was 269, and new cases referred to the centre were 91. Facilities for the project consist of the organisation of the consultation section on Tuesday morning to permit medical consultation and clinical evaluation (including ALSFRS-R), testing by the physiotherapist (MMT), spirometry (SVC), biological samples and ECG.
Department
Hôpital Roger Salengro
Clinique Neurologique, Neurologie A
Role
Clinical trial’s principal investigator in Lille
Universität Ulm
Professor Albert Ludolph is the head doctor of the neurology clinic and also professor of Neurology at the University of Ulm. The neurology university clinic develops improvements of the comprehension of disease mechanisms and of the treatment of diseases in their research. This research includes large fields of basic research in neurosciences, as well as studies of new medications. MitoTarget is an expression of the long-term interest in clinical ALS research of Prof Ludolph’s department. This interest is based on the fact that Ulm is one of the largest ALS centres in Germany and Europe with more than 500 patient visits/year. It also reflects the interest in translational research (drug development), focussed on in vitro and in vivo studies in orphan neurological diseases, in particular ALS, Huntington´s disease, MSA and PSP. The expected output (development of a relevant clinical therapy for ALS, information about the drug target in humans) is complementary to the goals of the Department.
Department
RKU - Universitäts- und Rehabilitationskliniken Ulm
Role
Clinical trial’s principal investigator in Ulm
Centre Hospitalier Universitaire de Limoges
The ALS reference centre of Limoges is located in the department of Neurology of the University Hospital Dupuytren. It depends on the French coordination of the 17 ALS centres. The active file of ALS patients was 192 in 2006, with 50 incident cases per year. Our ALS center gathers 6 departments of Center West of France [Creuse, Corrèze, Haute Vienne, Charente, Indre, Deux-Sèvres] and covers about 1,2 millions of inhabitants. Last year, 500 multidisciplinary consultations have been performed, including every 3 months the evaluation of all the medical and social aspects involved in ALS (nutrition, respiratory function, psychology, physiotherapy, social evaluation and palliative care). Our ALS center is also involved in clinical research. We participated in multicentric and international trials since 1992 (Riluzole : AG 216, RL301, RL 302; xaliproden, pentoxifylline, ONO). We developed research on 2 domains: epidemiology and nutrition. Concerning the neuroepidemiology, our center belongs to the EURALS consortium. Two PhD students are working on different aspects: compared epidemiology between Limousin and Uruguay, capture-recapture method to evaluate the real incidence of ALS at a European level, research of prognostic factors. Concerning the nutritional aspects, we are interested in elucidating the cause of the hypermetabolism found in sporadic and familial ALS cases. These 2 aspects of our research are integrated in the program of 2 University teams: EA3174 (neuropeidemiology) and EA4021 (Biochemical and molecular biology). Our results have been published in international journals since 1992. We actively participated and communicated in national and international meetings on ALS and other motor neuron disorders
Department
Service de neurologie
Role
Clinical trial’s principal investigator in Limoges
Hospices Civils de Lyon
The ALS Center of Lyon is located in the department of Neurology of the University Hospital Pierre Wertheimer. It depends on the French coordination of the 17 ALS centers. The active file of ALS patients was 277 in 2008, with 111 incident cases per year. Our ALS center gathers 9 departments of East of France [Rhône, Savoie, Haute-Savoie, Ain, Drôme, Ardèche, Saône et Loire, Isère, Jura] and covers about 4 millions of inhabitants. Last year, 647 multidisciplinary consultations have been performed, including every 3 months the evaluation of all the medical and social aspects involved in ALS (nutrition, respiratory function, psychology, physiotherapy, social evaluation and palliative care) and 299 neurological consultations (second device, complementary electroneuomyography). Our ALS center is also involved in clinical research. We participated in the multicentric and international Rilutek trial. We developed research on 2 domains: respiratory function and virological hypothesis. We actively participated and communicated in national and international meetings on ALS and other motorneuron disorders.
Department
ALS Center of Lyon, Department of Neurology, University Hospital Pierre Wertheimer
Role
Clinical trial’s principal investigator in Lyon
