Neuromuscular Diseases and ALS Program

In ALS, we have established the University of Michigan ALS Patient Repository (UMAPR) for blood, skin, fibroblasts, lymphocytes, genomic DNA, clinical data, lifestyle exposure information, and postmortem tissue for ALS patients and control individuals, that are being utilized in a CDC/ATSDR-funded study to identify potential ALS risk factors associated with environmental exposures and to characterize how epigenetic modifications impact gene expression and contribute to the development of ALS. We also examine the therapeutic potential of newly developed stem cell lines in the treatment of ALS and examining the efficacy of intracranial cellular transplantation in an animal model of Alzheimer’s disease, as a demonstration of the translational potential of our ALS cellular therapy strategies to other neurological disorders. Finally, we are developing advanced neuroimaging approaches as a potential diagnostic and prognostic tool to understand and assess ALS in human subjects.

Clinical Translational Trials

Clinical translational trials include observational studies involving patients from the U-M Metabolic Disease Clinics and Bariatric Surgery Programs, collaborative studies with groups from India and China to determine if the metabolic drivers of diabetic neuropathy are different in these diverse populations, health services research studies to evaluate the current clinical practice related to the diagnostic evaluation of diabetic neuropathy, and an investigator-initiated phase 2 clinical trial of stem cell therapy to treat ALS.