Stem Cells for Research in Motor Neuron Disease
The purpose of this study is to obtain skin biopsies from subjects with MND or pre-symptomatic familial ALS (fALS) and reprogram the fibroblasts from these samples into induced pluripotent stem (iPS) cells. The properties of the derived neurons will be compared to gain insights into potential causes of MND.
Disease:Amyotrophic Lateral Sclerosis (ALS), Familial ALS, Sporadic ALS, Primary Lateral Sclerosis (PLS), Hereditary Spastic Paraplegia (HSP), Healthy Volunteer, Healthy Volunteer with a Family History of ALS
Study Type:Observational Study
Study Chair(s)/Principal Investigator(s):
James D. Berry, MD, MPH (Massachusetts General Hospital)
Clinicaltrials.gov ID (11 digit #):
Coordinating Center Contact InformationMassachusetts General Hospital
Boston, Massachusetts 02114 United States
Full Study Summary:
Neurodegenerative diseases are diseases in which nerve cells of the brain and spinal cord die. There is a need to understand the cause of these diseases and to develop treatments. While we do have animal models of these diseases, they are very limited. We are in need of models that more closely match what happens in humans. Luckily, recent advancements in stem cell technology have allowed us to create a person™s own nerve cells by taking a skin biopsy or blood sample. This study wants to use this new technology to make models for neurodegenerative diseases. We hope this will give us a better understanding of the diseases, enable us to use the cells for drug screening, and in the future, develop treatments.
Study Sponsor:Department of Neurology Funds
This is a one time study visit at MGH that lasts about 1 to 3 hours.
Estimated Study Start Date:02/28/2011
Estimated Study Completion Date:12/28/2020
Posting Last Modified Date:08/05/2020
Date Study Added to alsconsortium.org:11/02/2016
Parking will be covered for study participants. No other compensation is available.
Time since Symptom Onset:N/A
Time since Diagnosis:N/A
Can participants use Riluzole?Yes
If you are taking anti-coagulant or anti-platelet medications (blood thinners), with the exception of aspirin, or have a history of keloid formation, you will not be able to take part in this study. To minimize the likelihood of an allergic reaction to the anesthetic, you cannot take part in this study if you are allergic to Lidocaine. Subjects undergoing the voluntary lumbar puncture (LP) must be medically able to undergo a LP as determined by a Study Investigator (ie: no bleeding disorder, allergy to local anesthetics, a skin infection at or near the LP site, or evidence of high intracranial pressure).
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