February 20-22, 2018
Boston, USA
Day Two
Thursday, February 22nd, 2018
08.25 Chairs Opening Remarks
Developing Next Generation Directed Therapies for Aβ Tau in Alzheimer’s
08.30 The Amyloid Cascade Hypothesis: The Path Forward in Developing Targeted Therapies for Alzheimer’s Disease
Synopsis
- Highlighting strategic benefits of applying genomic stratification of patients for validation of biomarkers from target discovery to therapeutic development
- Describing discovery of a novel molecular mechanism of action of ALZ-801, which blocks formation of toxic amyloid oligomers associated with development and progression of Alzheimer’s disease
- Reviewing development of ALZ-801, a Phase 3-ready, first-in-class, small molecule oral inhibitor of amyloid aggregation and neurotoxicity
- Emphasizing need for application of precision medicine approach in neurodegeneration, based on individual genetic and biological information, to advance therapies with the greatest impact for patients
08.50 Development of Phospo-Tau Antibodies Targeting Pathologic Forms of Tau
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Jan Torleif Pedersen
Director, TBL Alzheimers Disease and Dementia, Neurodegeneration, H. Lundbeck A/S
Synopsis
- Demonstrating that microtubule binding protein Tau is hyperphosporylated and aggregated during neurodegenerative processes such as Alzheimers disease
- Revealing data showing hyperphosphorylated aggregated forms of Tau as being able to act as an endopathogen in preclinical models of tauopathies
- Showcasing the development of highly specific and selective monoclonal antibodies which will target hyperphosphorylated aggregated forms of Tau. These antibodies will prevent seeding of pathology in preclinical models of tauopathies
09.10 Precision Medicine in a Data-rich Era – Challenges and Successful Bioinformatics Approaches
Synopsis
- Highlighting the promise biomedical data holds as a powerful tool for enabling precision medicine
- Making effective use of big data in R&D processes to establish reliable hypotheses that can be tested with relatively small patient numbers in clinical trials given an incomplete biological understanding of the underlying disease
- Demonstrating real-world biomarker cases from non-oncology projects and presenting typical challenges and successful multi-omics approaches for identifying response biomarkers that correctly identify 85-95 % of the responding patients
09.30 Session Q&A Panel
Synopsis
- Does Amyloid Beta really still remain the holy grail of treatment targets
- Is there a need to apply the combinatorial therapy paradigm to neurodegenerative diseases? If so, how?
- What preclinical and clinical funding strategies are available to maximize resources for drug discovery and development?
10.00 Afternoon Refreshments & Networking
Discovery, Development & Validation of Non-Invasive Signatures as Prognostic & Diagnostic Biomarkers of Disease
11.00 Perspectives on Biomarker Applications Through Drug Development
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Johan Luthman
Vice President, Neuroscience Business Group, Eisai Pharmaceuticals
Synopsis
- Exploring the utility of biomarkers and imaging for early development and in late clinical development
- Divulging where we are with ‘tool box use’ of these markers for various diseases and the current obstacles and limiting factors to be overcome for regulatory adoption
11.20 Amyloid PET Imaging in Aducanumab Ph1b PRIME study
Synopsis
- Showcasing recent data from the PRIME study, highlighting aducanumab as an Aβ- removing and potentially disease-modifying therapy for AD
- Presenting supportive evidence, showcasing how amyloid PET has been effectively utilized as an informative clinical biomarker
11.40 Presentation to Be Confirmed
12.00 Session Q&A Panel
Synopsis
- What remain the analytical and regulatory considerations for incorporating neuroimaging systems to advance biomarker monitoring?
- How can we drive innovation with imaging systems for more accurate disease identification and characterisation?
- Can we effectively combine fMRI and PET imaging to develop better indicators of cognitive function for neurodegenerative diseases?
- How should we approach issues around validation and quantification of diagnostic biomarkers?
Exploring Advances in Emerging CSF-Based Biomarkers
13.30 Multi-Center Biomarker Development for Diagnosis and Prognosis of Amyotrophic Lateral Sclerosis
Synopsis
- US, UK, Asian Cohorts for ALS diagnosis using whole blood samples
- UK cohort for ALS prognosis
- Relevance for other neurodegenerative and motor neuron diseases, like Alzheimer’s Disease, Parkinson’s, Dementia, Multiple Sclerosis, Depression and Schizophrenia
13.50 Utility of Neurofilament Light Chain as a Translational Tool in Neurodegenerative Disorders
Synopsis
- Reviewing of NFL changes in preclinical models
- Highlighting the relationship of NFL with disease progression in neurodegeneration
- Determining the utility of NFL to predict decline in neurodegeneration
14.10 Biomarker Data in Cerebrospinal Fluid in Parkinson’s Disease: The PPMI Study
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Douglas Galasko
Professor, Department of Neurosciences, University of California, San Diego
Synopsis
- Exploring how CSF biomarkers related to key neurodegenerative lesions are altered in early Parkinson’s disease (PD) and how they change over time
- Understanding how CSF biomarkers relate to measures of movement and cognition
- Evaluating how CSF biomarkers can be used to complement clinical trials in PD
14.30 Session Q&A Panel
Synopsis
- Where should we pool our efforts in the development of fluid-based biomarkers and associated strategies for targeting, to measure clinical outcomes and ensure the greatest chance of clinical trial success?
- Where can big data, statistics and in silico efforts drive innovation in identification of biomarkers with legitimate clinical reproducibility?
- How can we ensure confidence of therapeutic efficacy through initial trials in genetically defined and relevant populations?
- Is this all too little too late? Should our efforts focus more towards the integration of target engagement biomarkers in preclinical development?
15.00 Afternoon Refreshments & Networking
Targeting the Spread of Pathology Through Alternative Aetiologies
15.20 Developing Preclinical Biomarkers of Proteopathic Seeds in Neurodegenerative Disease
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Jonathan Levenson
Senior Director, Preclinical Research & Development, Proclara Biosciences
Synopsis
- Reviewing disease association with misfolding and aggregation of a number of different proteins
- Highlighting the pathological role of spread, at least in part, due to transmission of proteopathic seeds from cell-to-cell
- Exploring how next-generation fluid biomarkers for neurodegenerative diseases should track the transmissible form pathological proteins
15.40 The Role of GBA in Lysosomal Function, Neuroprotection and Progression in Parkinson’s Disease
Synopsis
- Highlighting clinical, genetic and experimental evidence underlies the relevance of lysosomal dysfunction in Parkinson’s disease
- Showcasing how stimulation of the lysosomal GBA pathway in the CNS can improve the pathological and behavioral abnormalities in preclinical models of disease
- Revealing the potential impact of modulating this lysosomal pathway to slow the progression of Parkinson’s disease is being studied in a genetically defined population
16.00 Targeting Autophagic Protein Clearance Mechanisms for the Treatment of Neurodegenerative Disease
Synopsis
- Demonstrating the rationale for developing targeted therapeutics for autophagy
- Strategies for tackling the related challenges both preclinicaly and clinically
- Presenting the story of the discovery and validation of data from therapeutics directed at LAMP2a, PI3K and/or TLR 2
16.20 Session Q&A Panel
Synopsis
- Although spread offers a unifying pathophysiological concept of neurodegenerative diseases, are the mechanisms of spread universal?
- How can advanced research showing the propagation of spread in Parkinson’s be applied to AD and other neurodegenerative diseases?
- What importance does the biochemical characteristics of the proteopathic seeds hold? E.g. are oligomers, are more detrimental to cells than are fibrillar forms?
- To what relevance and extent does lysosomal dysfunction, glial disruptions etc have on the transmission of protein pathology in neurodegenerative diseases?
17.20 Afternoon Refreshments & Networking
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