Cell and Gene Therapies for Neurologic Diseases

Howard Federoff, M.D., Ph.D., has a dual role of Vice Chancellor for Health Affairs and the CEO of UCI Health System. As Vice Chancellor for Health Affairs, Dr. Federoff has responsibility for the clinical, medical educational, and research missions. As CEO of UC Irvine Health System, Dr. Federoff oversees the administration of clinical locations, UC Irvine Medical Center, the Chao Family Comprehensive Cancer Center, and UC Irvine Health’s primary and specialty care outpatient centers. A nationally renowned clinical and research leader and a groundbreaking investigator for neurological disorders, Dr. Federoff’s research led to a new gene therapeutic trial in Parkinson’s disease, in predicting the onset of Alzheimer’s disease. Dr. Federoff has an academic appointment in the department of Neurology. He has published 250+ articles, serves as a reviewer for many journals, and serves on the editorial boards of five journals. Dr. Federoff served as Chair of the NIH Recombinant DNA Advisory Committee from 2007-2010. He Chaired the Gene Therapy Resource Program for NHLBI (2010-2016), was President for the American Society for Neural Therapy and Repair (2012-2013) and President of the American Society for Experimental Neurotherapeutics (2014-2016). He is chair-elect of the Association of Academic Health Centers (AAHC) Board. Dr. Federoff is an elected Fellow of the American Association for the Advancement of Science and the National Academy of Inventors. Roger Barker is the Professor of Clinical Neuroscience and Honorary Consultant in Neurology at the University of Cambridge and at Addenbrooke's Hospital.  He trained at Oxford and London and has been in his current position since 2000, after completing an MRC Clinician Scientist Fellowship. Dr. Barker combines basic research looking at novel therapies to treat chronic neurodegenerative disorders of the brain with clinically-based work aimed at better defining such disorders. He is the co-ordinator of the TRANSEURO project looking at fetal cell grafting in patients with early Parkinson's Disease and is part of the GFORCE PD initiative as well as Director of the ISSCR.

Section 1. Cellular Therapies
1. Introduction to stem cell biology and its role in treating neurologic disorders
2. History of cellular grafting for CNS repair - A clinical perspective
3. Autologous vs. heterologous cell replacement strategies for Parkinson disease and other neurologic diseases

Section 2. Gene therapies
4. Viral vectors and gene delivery to the central nervous system
5. Viral versus Non-viral approaches

Section 3. General Considerations (for cell and gene therapies)
6. Clinical/surgical considerations
7. Ethics of gene and cell therapy development for neurological disorders
8. Clinical trial designs and endpoints
9. Immunology of such therapies
10. Scaling of cell and gene therapies to population
11. The Health economics of cell and gene therapies

Section 4. Disease specific interventions using cells and genes
12. Disease specific interventions: The use of cell and gene therapies for Parkinson disease
13. Using gene or cell therapies to treat Huntington’s disease
14. Disorders of the motorneuron including ALS and SMA
15. Gene and cell-based therapies for retinal and optic nerve disease
16. Disease specific interventions using cell therapies for spinal cord disease/injury
17. Stem and progenitor cell-based therapy of myelin disorders
18. Cell and gene therapy in neuro-oncology