Biomarkers for CNS Toxicity—From Bench to Bedside (S07)

Symposium Chair(s)

• Nawshaba Nawreen, PhD

  Advisor
  Eli Lilly and Company, Inc.
  Indianapolis, Indiana, United States

• Xihui Xu, PharmD, PhD

  Director
  Toxicology
  QurAlis Corporation
  Cambridge, Massachusetts, United States

Symposium Speaker(s)

• David Compton, PhD, DABT

  Senior Consultant
  Nonclinical Development
  Akkeri, Inc.
  Annandale, New Jersey, United States

• Ingrid Pardo, DVM, DACVP, FIATP

  Scientific Director
  Pathology, Nonclinical Safety
  Biogen
  Cambridge, Massachusetts, United States

• Serguei Liachenko, MD, PhD

  Director of Bioimaging
  Neurotoxicology, NCTR
  US FDA/NCTR
  Jefferson, Arkansas, United States

• Jeffrey Siegel, MD

  Office Director
  ODES/OND/CDER
  US FDA
  Silver Spring, Maryland, United States

Educational Support Provided by: Roundtable of Toxicology Consultants

There is currently an urgent unmet medical need for newer, safer, and more effective medications to treat CNS disorders, thus emphasizing the need for continued attempts at advancing promising molecules into clinical testing. At present, the dearth of reliable biomarkers for CNS injury is impeding efforts to develop new drugs for neurological diseases. There is a need for more sensitive and specific biomarkers for neuronal or glial cell injury that can help detect and predict neuro-toxicities that are relevant across animal models and translational from nonclinical studies to the clinic. Better biomarkers may also provide improved understanding of on- and off-target effects that will be more informative for clinical development and, ultimately, regulatory approval. Fluid-based biomarkers such as those found in serum, plasma, urine, and cerebrospinal fluid (CSF) are urgently needed since biopsies of CNS tissues are not feasible. Non-invasive neuroimaging techniques, such as magnetic resonance imaging/spectroscopy and positron emission tomography, are also in great demand due to their non-invasiveness, high precision, and suitability for longitudinal analyses. Developing and using biomarkers through development from bench to bedside will require close cooperative interactions between sponsors and regulatory agencies. This symposium will address the current state of the art regarding biomarkers for CNS injury and toxicity. Speakers in this session will provide attendees with an overview of current approaches for detecting and monitoring CNS injury and toxicity with the aim of catalyzing interest and efforts to identify new biomarkers that can better facilitate efforts to bring therapies to patients with neurological diseases.

Presentations:

• 9:00 AM - 9:05 AM CT

  Welcome
  

  Symposium Chair: Nawshaba Nawreen, PhD – Eli Lilly and Company, Inc.

  Symposium Chair: Xihui Xu, PharmD, PhD – QurAlis Corporation
• 9:05 AM - 9:40 AM CT

  Introduction to Neurotoxicity Biomarkers with Special Reference to AAV Gene Therapy
  

  Symposium Speaker: David R. Compton, PhD, DABT – Akkeri, Inc.
• 9:40 AM - 10:15 AM CT

  Fluid-Based Biomarkers of the Nervous System
  

  Symposium Speaker: Ingrid D. Pardo, DVM, DACVP, FIATP – Biogen
• 10:15 AM - 10:45 AM CT

  Break
• 10:45 AM - 11:15 AM CT

  In Vivo Imaging Biomarkers of Neurotoxicity
  

  Symposium Speaker: Serguei M. Liachenko, MD, PhD – US FDA/NCTR
• 11:15 AM - 11:45 AM CT

  Biomarkers of Neurotoxicity: An FDA Perspective
  

  Symposium Speaker: Jeffrey Siegel, MD – US FDA
• 11:45 AM - 12:00 PM CT

  Panel Discussion