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University of Pittsburgh

Research

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.

http://www.alsconsortium.org/trial.php?id=68

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

http://clinicaltrials.gov/ct2/results?term=tirasemtiv&Search=Search

Diaphragm Pacing Study

We are seeking individuals with ALS and breathing muscle weakness to participate in a research study enrolling under the direction of David Lacomis, MD.

The purpose of the research study is to determine whether the NeuRx® Diaphragm Pacing System™ (DPS) is associated with improved survival or diaphragm function for people with Amyotrophic Lateral Sclerosis (ALS). While participating in this study, participants will undergo many breathing tests and will be asked to complete several questionnaires. The DPS device is surgically implanted during a brief hospital stay. Eligible participants will have a 2/3 chance of being randomized to receive DPS. Those participants who are not randomized to receive DPS will still undergo the same intensive testing of breathing muscle function, which may result in earlier detection of breathing symptoms. From this study, the researchers hope to learn more about ALS and its treatment.

Participants must be at least 21 years of age. Participants should be located close to the study site and be able to follow study tasks. Participants in the study will have approximately 6 study visits and will receive monthly phone calls. The average duration of study participation is estimated to be 18 months. The phone calls will last indefinitely. If interested, please contact one of our coordinator at 412-864-2873 or 412-647-1706 or e-mail us at doerdx@upmc.edu. You may also contact the Coordination Center via the Northeast ALS Consortium (NEALS) website for more information about this study at www.nealsconsortium.org.