Synopsis
Motor neurone disease (MND) occurs in all ages and is a neurodegenerative disorder in which the loss of large nerve cells in the brain and spinal cord produces progressive weakness, significant disability and frequently death. We used spinal cord neural progenitor stem cells in two American FDA approved clinical trials to treat MND. In both the Phase 1 and 2 clinical trials, MND patients received direct intraspinal transplantation of stem cells into either the lumbar, cervical or both lumbar and cervical regions of the spinal cord. Safety of the therapy and MND clinical progression were monitored and results show that the approach is both feasible and well tolerated. The therapeutic signal also warrants another larger Phase 3 FDA trial that will begin in 2017. This marks a new era for stem cell therapy and for our treatment of MND and opens the door for similar approaches in other neurological disorders.
Speaker
Eva L. Feldman MD, PhD is the Russell N. DeJong Professor of Neurology at the University of Michigan Medical School and Director of the A. Alfred Taubman Medical Research Institute. She also directs her own 30-scientist laboratory and is the principal investigator of two first-ever FDA approved human clinical trials of intraspinal stem cell transplantation for Motor Neurone Disease. Dr Feldman has published more than 375 original peer-reviewed articles, 70 book chapters, and 4 books and has had continuous National Institutes of Health funding for 25 years. In her role as President of the American Neurological Association from 2011-2013 and as a member of the National Academy of Medicine, she strongly advocates for stem cell research and stem cell therapy in regulated clinical trials. In recognition of her work, she was named the 2016 National Clinician of the Year by Best Doctors in America.