Neurodegenerative diseases remain one of the major causes of death and constitute one of the leading causes of disability. Although novel drugs and new treatments to reduce the burden have been implemented over the past 25 years, the incidence, mortality and disability rates of neurological disorders remain high, due to population aging and growth.
The implementation of new strategies to predict drug outcomes for more effective drug development and establishing a more personalized medicine approach is needed. Recent advances in both biology and technology platforms have enabled reliable and affordable measurement of novel biomarkers — biological measures of physiological or pathophysiological processes or pharmacological responses — which can have a variety of applications.
Some of these biomarkers can be used in improving diagnostic and prognostic accuracy in disease monitoring and as surrogate endpoints in pre-clinical and clinical studies and clinical trials, as well as to help stratify patients, providing the basis for a more personalized medicine approach. Despite the large numbers of candidate biomarkers which have been proposed and extensively evaluated, very few are currently integrated into routine clinical practice and the quest for novel brain injury markers in still ongoing.
Join this webinar to hear the panelists discuss two strategies to approach neurodegenerative disease monitoring using objective biomarkers and offering new and unique perspectives.
Through a case study in the field of Huntington’s disease, the panelists will show the development of a single molecule counting (SMC) ultra-sensitive assay to detect fM-level huntingtin (HTT) in human cerebrospinal fluid. The relevance of the antibody and the calibrator analyte selection in the light of HTT biology will be discussed, together with the assessment of possible confounding factors while detecting highly diluted analytes in peripheral fluids.
In the second segment of the presentation, mass spectrometry-based methods for robust measurement of novel investigative biomarkers for the identification of a neurodegenerative disorder will be shown. A case study will be used to highlight a liquid chromatography–mass spectrometry (LC-MSMS) approach developed to follow predictive biomarkers in the context of pantothenate kinase-associate neurodegeneration (PKAN).
Dr. Pamela di Pasquale, PhD, Research Investigator, IRBM. S.p.A.
Pamela di Pasquale received her PhD in Chemical Sciences from the University of Naples Federico II, where she studied DNA alkylation by biochemical and proteomics approaches. Pamela joined IRBM as a member of the DMPK group in 2015, where she acquired extensive experience in developing, qualifying and validating mass spectrometry-based methods for quantification of proteins and metabolites in peripheral fluids or in tissues. She leads the Biomarkers and LC-MS platforms team focused on developing new methods for measuring small and large molecules biomarkers to support preclinical research.Message Presenter
Dr. Manuel Daldin, PhD, Research Investigator, IRBM. S.p.A.
Dr. Daldin is leading the group of ligand binding assays at IRBM. S.p.A. During the past eight years he has been involved in assay development, feasibility, qualification and validation. During these years, Dr. Daldin actively collaborated in multiple project settings, including big pharma, CRO, biotech, academic consortia and not-for-profit foundations. He has contributed or led a number of pre-clinical and clinical studies, mostly in the neuroscience area. Dr. Daldin earned his MSc. in “Industrial and Environmental Biotechnology” together with a master in “Industrial Pharmacy” at the University of Rome “La Sapienza”, where he strengthened his skills in the drug development area including registration, production, control and distributions of drugs.Message Presenter
Who Should Attend?
Senior professionals who are:
- In vivo Research Scientists
- PK/PD Scientists
- Neurodegeneration Scientists
What You Will Learn
At the conclusion of this webinar, the attendees will be able to:
- Discuss biomarkers found to be dysregulated in Huntington’s disease and Neurodegeneration (i.e. PKAN)in patients and/or animal models
- Understand the fundamentals for the development of a pharmacodynamic ligand binding assay.
- Describe approaches to assessing problems and improving method specificity and sensitivity for the quantitation of small and large molecule biomarkers
IRBM is an innovative contract research organization working across all aspects of drug discovery and early development for different modalities – small molecule, peptide, RNA and antibodies. We foster collaborations with organizations from the pharmaceutical, biotech and academic sectors to accelerate drug discovery from target validation and hit identification to candidate nomination. Our 200 plus-strong, multidisciplinary team works at a state-of-the-art R&D facility near Rome, Italy where projects are carried out “under one roof” enabling rapid cycle times and close integration of the scientific teams. IRBM’s scientists have discovered several drugs that are on the market, and more than 25 molecules have progressed to clinical testing. The company was founded in 2010, as a spin-off from Merck & Co. Now in our 11th year as an independent research organization, we have laid the foundations to become a global center for excellence in all aspects of drug discovery and early development.