“New Therapies for ALS”

Hosted by the Hope Center for Neurological Disorders

Short Bio: Dr. Merit Cudkowicz is the Chief of the Massachusetts General Hospital Neurology Service, Director, Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital, and the Julieanne Dorn Professor of Neurology at Harvard Medical School in Boston. Dr. Cudkowicz is co-founder and former director of the NCRI. She directs the Massachusetts General Hospital ALS Program and the Massachusetts General Hospital Neurological Clinical Research Institute. She is one of the founders and former co-directors of the Northeast ALS Consortium (NEALS), a group of over 100 clinical sites in the United States, Canada, Europe and the Middle East dedicated to performing collaborative academic led clinical trials and research studies in ALS. She is Principal Investigator of the Clinical Coordination Center for the National Institute of Neurological Disorders and Stroke’s Neurology Network of Excellence in Clinical Trials (NeuroNEXT). The NeuroNEXT network is an efficient phase II network to develop innovative and new treatments for people with neurological disorders.

Retreat Program

8:30a      Welcome and Opening Remarks – David Holtzman, MD and Anneliese Schaefer, JD, PhD

8:45a       NeuroRestorative Therapy Group
NeuroRestorative Chair: Hiroko Yano, PhD (WashU Neurosurgery)

• Lu Zhao, PhD (Cui Lab, WashU Biomedical Engineering)
  “A potential sonogenetic actuator for ultrasound-mediated neuromodulation”

9:00a       Neurogenetics & Transcriptomics Group
Neurogenetics Chair: Gabriel Haller, PhD (WashU Neurosurgery)

• Logan Brase (Harari Lab, WashU Psychiatry)
  “Single-nuclei RNA-sequencing of Alzheimer Disease brains reveals altered cell expression state compositions linked to autosomal dominant, TREM2 and rs1582763 variants”
• Chengran Yang (Cruchaga Lab, WashU Psychiatry)
  “Genomic and multi-tissue proteomic integration for understanding the genetic architecture of Alzheimer disease and Parkinson’s disease”

9:30a       Break

9:45a       Protein Aggregation & Neurodegeneration Group
Neurodegeneration Chair: Chris Weihl, MD, PhD ( WashU Neurology)

• Jiang Zhu (Weihl Lab, WashU Neurology)
  “VCP protects neurons from proteopathic seeding”
• Maud Gratuze, PhD (Holtzman Lab, WashU Neurology)
  “Activated microglia mitigate Aβ-associated tau seeding and spreading”
• Lucia Capano (Yoo Lab, WashU Developmental Biology)
  “MicroRNA-mediated neuronal reprogramming generates human neurons that express tau isoform expression comparable to adult brains”
• Zachary Grese (Ayala Lab, SLU Biochemistry Molecular Biology)
  “RNA is a platform for TDP-43 liquid-liquid phase separation and promotes TDP-43 solubility through specific binding”

10:45a       Clocks & Sleep Club
Clocks & Sleep Chair: Erik Herzog, PhD (WashU Biology)

• Patrick Sheehan (Musiek Lab, WashU Neurology)
  “Astrocytic BMAL1 regulates protein aggregation in mouse models of alpha-synuclein and tau pathology”

11:00a       Break

11:15a       Hope Center Lecture
Speaker Introduction: Timothy Miller, MD, PhD (WashU Neurology)

• Merit Cudkowicz, MD, MSc (Harvard Medical School, Massachusetts General Hospital)
  “New Therapies for ALS”

For inquiries contact Jan Konrad or the Hope Center.