Current Research Projects
Growing Stem Cells from Skin
Novel stem cells studies. Studying rat and mouse models of ALS has not predicted which drugs will be successful in humans. With Drs. Danielle Carlisle and Yuanyuan Jiao, we are growing HUMAN motor neurons from individuals with ALS by taking skin cells (called pleuripotent stem cells) obtained by simple skin biopsy and then turning these skin cells into motor neurons and supportive glial cells that are similar to those found in human spinal cord. This process takes months. The biology of the cells should tell us about disease mechanisms and why there are so many differences in disease characteristics among individuals. They will also provide a foundation for testing drugs to predict which ones should move forward and be tested in patients. We are currently enrolling subjects with ALS. Mr. Neil Alexander is pictured having a skin biopsy by Dr. Lacomis. Dr. Jiao received the specimen and the skin cells have been subsequently converted to motor neurons
High Field MRI
High definition fiber tracking studies. We do not fully understand the progression of the loss of motor neurons and their tracks (pathways) in brain and spinal cord areas in ALS. Greater knowledge of these spread patterns will allow us to predict events and provide better clinical care for ALS patients and potentially to help slow the disease progression. Using high field magnetic resonance imaging (MRI), we can track the fibers in these pathways in ALS patients over time and understand what areas are affected earliest and how the disease spreads. We are currently enrolling patients in this study directed by Drs Juan Fernandez-Miranda, Kumar Abhinav, and Friedlander from the Department of Neurosurgery.
Discovery and Validation of Biomarkers
Subjects are being recruited to provide serial samples of blood and spinal fluid from patients with ALS. We are sharing samples with investigators from 5 other Centers. The researchers hope to learn more about the underlying cause of ALS, as well as find unique biological markers, which could be used to develop new therapies.
Drug Trial of Tirasemtiv in ALS
We are participating in a Phase IIb research study of the drug tirasemtiv, that aids in muscle contraction, in patients with ALS. See