Biomarkers for Neurodegenerative Diseases: Immunoassays and LC-MS/MS to Track Disease Progression

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).

Speakers

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.

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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.

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