Michael Benatar is a professor of neurology; the Walter Bradley Chair in ALS Research; Executive Director of the ALS Center; Chief of Neuromuscular Division; and Vice Chair for Clinical & Translational Research in the Department of Neurology at the University of Miami. His clinical/translational research program is focused on biomarker and therapy development for ALS, with a particular focus on pre-symptomatic ALS and disease prevention. He leads the Pre-Symptomatic Familial ALS (Pre-fALS) study, which he initiated in 2007, and the CReATe Consortium, a ~35-center network focused on therapy development for ALS and related disorders. Dr. Benatar is best known for his pioneering work in defining the field of pre-symptomatic ALS, including discovery of the first biomarker of pre-symptomatic disease that has been critical to the design and initiation of the first pre-symptomatic ALS trial. He has also been a thought-leader in challenging existing paradigms for pre-clinical therapeutic studies; shaping how we conceptualize and use biomarkers for therapy development; and championing the use of enrichment strategies in ALS trial design.
Dr. Benatar will contribute expertise and expertise from ~15 years of studying pre-symptomatic ALS, and also provide biological samples and accompanying deep phenotypic data from the Pre-fALS study to advance the goals of identifying a clinico-molecular fingerprint of early ALS.
Philippe Corcia is Professor of Neurology and has conducted the ALS Center of Tours since 2003. This Center takes in charge more than 250 patients and diagnoses each year between 80-100 new ALS cases. He also chairs the French Study Group on Motoneuron Diseases (GFEMM) and is member of the ENCALS Group and of the governance of the TRICALS group which aim is to care ALS. He also chairs the project Mine. His research focuses on two main topics: identification of susceptibility genetic factors of ALS and of diagnostic and prognostic biomarkers from omics approach within the Inserm Unit U1253 iBrain.
Patients and controls recruitment
In the Cracow lab of Wojciech Kuban, PhD, researchers apply bioengineering principles, complex molecular analysis techniques, and high-end technology to drug development using pharmacogenomics and quantitative systems pharmacology. Research is focused on methodological advancement and application of molecular systems and models to optimization problems of drug development and therapy for neurodegenerative diseases.
Proteomics analysis of blood samples (DIA-MS)
Our group at the University Medical Center Göttingen (UMG) focuses on the development and application of state-of-the-art mass spectrometry methods for proteome analysis in preclinical and clinical research. Our research ranges from methods for the in-depth analysis of cellular and animal research models, to proteome profiling of patient material from tissues and bodily fluids. Taking a strongly collaborative approach and using the UMG’s Core Facility Proteomics infrastructure , we apply our method portfolio to help researchers address a wide range of pressing biomedical questions.
- Development of data-independent acquisition mass spectrometry (DIA) methods for protein profiling in bodily fluids
- DIA-MS analysis of tear fluid samples for the discovery of candidate protein biomarkers
- Analytical consultancy to our premodiALS project partners
Hadassah Ein Karem university hospital, Jerusalem, is a hospital that prioritizes specialization in severe diseases and in clinical research, as well as basic biological research in cooperation with the adjacent Hebrew University school of medicine.
Our ALS clinic is serving patients from the Jerusalem metropolitan area and from other parts of Israel. Our aim is to provide patients with the fullest extent of possibilities for slowing progression of the disease, in a disease with limited possibilities. That includes providing access for drugs that have shown evidence of efficacy in phase II/III trials but not yet available, expanded access programs and clinical trials participation on studies in all clinical phases. The recent work of our team includes observational study of microbiome in ALS patients, phase I clinical trials in Astrocyte transplantation (AstroRx®) and in IPL-344, therapeutic peptide, AKT pathway agonist.
Our center is one of the participants recruiting centers in the study, performing clinical assessments of the subjects and sample collection.
The mission of the Menden Lab is to develop biostatistical and machine learning frameworks applied to biomedical data, to retrieve insights in the aetiology of complex diseases and identify novel intervention strategies. For this, we explore deep molecular characterised biomedical datasets, environmental factors, and tailor our models depending on disease specific knowledge, literature and data driven analyses, thus empowering the next generation of drug target identification, drug repositioning and precision medicine.
- Biostatistical analysis of deep molecular and clinically characterised patients.
- Predict ALS patient outcome with artificial intelligence and machine learning.
- Empower patient stratification by deriving clinico-molecular fingerprint.
Our group at the MND & Neurotrophic Research Laboratory, Flinders Health & Medical Research Institute, Flinders University, Australia has been leading the world, in describing urinary biomarkers for motor neuron disease. We hypothesise urinary biomarkers, which are easily accessible for sample collection purposes, could be used to determine and predict disease prognosis and facilitate improved clinical trial design to establish efficacy of new therapeutics in motor neuron disease. Thus far, we have characterised 2 urinary biomarkers, one of which (p75ECD) has being proposed as a pharmacodynamic marker.
- As an external partner: we will be focus on measuring urinary biomarkers in the preModi samples
Akdeniz University Hospital, Korkut Yaltkaya Clinical Neurophysiology Laboratory is one of the important and main clinical neurophysiology laboratories of the South of Turkey. Most of the ALS patients' EMG of this area are made in our centre and further studies such as axonal excitability, motor evoked potentials are able to hold in this laboratory. Our neuromuscular outpatient clinic supports our laboratory with patients and annually nearly 1250 patients are applying.
As a part of the PremodiALS Project, we will follow familial ALS patients and their family members continuously by their genetically, electrophysiological progress and clinical features. We will get samples of their CSF, serum, tears and we will register their detailed pieces of information.
In collaboration with the ETH Zürich the first cannabinoid typ 2 radioligand has been developed, which is now available for clinical use.
- Protocol development
- Recruiting of patients and healthy controls
- Clinical Assessments
- Biomaterial collection
Norbert Zilka is the director of Institute of Neuroimmunology, SAS and CSO of the biotech company Axon Neuroscience. He is the founder of the first Slovak brain bank. Norbert holds a PhD in Immunology from the Slovak Academy of Sciences (SAS). His main scientific focus is on human neurodegenerative disorders such as Alzheimer’s disease, ALS and FTD. He was actively involved in the preclinical and clinical development of the first-in-man, first-in-kind tau vaccine against Alzheimer’s disease and tau diagnostic assay for AD. He took part of several JPND projects such as BIOMARKAPD (2012-2015), REfrAME (2016-2019) and
- recruitment of participants
- metabolomic analysis
The Lingor lab is located at the Klinikum rechts der Isar of the Technical University of Munich. In a truly translational approach, we are interested in bringing scientific results to the patient. Two major neurodegenerative disorders - Amyotrophic lateral sclerosis and Parkinson's disease - are in the focus of our work. We studying biofluids to identify novel biomarkers for these disorders and we develop novel therapeutic approaches that are translated from the dish and animal experiment up to clinical trials.
In the premodiALS project, one of the main tasks will be the coordination of all work packages between the different partners of this multinational consortium. Furthermore, the clinical site will recruit subjects to the project and participate in biosample processing and redistribution.
Peter Munch Andersen is professor of neurology and Wallenberg Clinical Scholar at the university hospital in Umeå, Sweden. He performed clinical, genetic and molecular-biology research into the causes of various types of ALS disease since 1992. He established the Swedish registry for familial ALS and ALS-FTD and collects longitudinal data and blood samples from patients and their unaffected relatives for prospective medical research. The ALS neurogenetic lab in Umeå participated in finding many of the genes causing ALS and FTD, in particular C9orf72 and TBK1. A major focus of the laboratory has been studying the SOD1 gene. The laboratory is also involved in the development and validation of biomarkers including the first analysis for neurofilaments in the 1990’ties.
Based on our many experiences with genotyping and phenotyping families with different types of ALS and ALS-FTD, in 2005 we proposed the first international guidelines for the use of DNA testing in ALS patients and for genetic counselling and predictive DNA testing of relatives.
The primary contribution of the Umeå lab to the premodiALS project will be to perform state-of-the art genetic analysis.