Eleanor and Lou Gehrig MDA/ALS Research Center, Columbia University Projects Funded by MDA's Wings Over Wall Street 2002: Research Accomplishment  Investigators  Project Major Accomplishment Total Funding Paul Gordon, M.D. Clinical trial of Glatiramer (Copaxone) in ALS *Translation from animal study to human project * All planning completed with grant submitted to MDA * Institutional approval and drug preparation * The cost of drugs carried over to this year $167,199 Hiroshi Mitsumoto, M.D. ALS Marker Study * Novel technology, MR spectroscopy, and transcranial magnetic stimulation can distinguish ALS from other motor neuron diseases * Absolute quantification of NAA has been developed * Several presentations have been made at national and international meetings $92,199 Hiroshi Mitsumoto, M.D. Lou Gehrig Centennial Conference on ALS * A successful international meeting that focused on clinical trials in ALS * Edited 30 manuscripts and prepared for publication (June 2004) $25,000 Hiroshi Mitsumoto, M.D. and Ruth Ottman, Ph.D. Genetic-Environmental Epidemiology * Detailed planning, project development, and grant submission to MDA * A consensus conference on this topic was held in March with 11 national and international collaborators and 10 members of the Columbia faculty. * Study Questionnaire developed. $50,000 Thomas Jessell, Ph.D. Characterization of Motor Neuron Progenitor Cells * Demonstration that retinoids function in an essential manner to promote motor neuron progenitor identity (Novitch et al. Neuron 2003) * Demonstration that FACS sorted motor neuron progenitors can differentiate into motor neurons in vitro (Agalliu et al. In preparation) $125,000 Thomas Jessell, Ph.D. Directed Differentiation of Embryonic Stem Cells into Motor Neurons * Demonstration that ES cells can differentiate into motor neurons (Wichterle et al. Cell, 2002) * Demonstration that ES cell derived motor neurons have appropriate biophysical properties (Miles et al, J. Neuroscience) * Demonstration that ES cell derived motor neurons acquire columnar and pool identities (Wichterle et al. In Preparation) * Demonstration that human ES cells can give rise to motor neurons (Wichterle, Rajagopal, Jessell, Melton; In Preparation) $200,000 Serge Przedborski, M.D., Ph.D. Role of Endoplasmic Reticulum Stress in ALS Neurodegeneration * Most of the key pathways of the ER stressed have been mapped in both ALS rats and post-mortem tissue. * Mutant animals with ALS mutations but lacking key factors of the ER stress are currently evaluated $150,000 Serge Przedborski, M.D., Ph.D. Behavior of Motor neurons Derived From Embryonic Stem Cells in Animal Models of ALS * All of the technical aspects related to the protocol of stem cells preparation and grafting technical have been completed. * Young and old ALS rats are currently evaluated after implantation of stem cells Included in the above budget William Dauer, M.D. Generating and Characterizing an Inducible Model of ALS * Generating a tetracycline inducible line of SOD G93A mice * Performing behavioral and pathologic studies of these mice to determine whether SOD-related neurodegeneration is reversible. $180,000 TOTAL $989,398