Stem Cells for Research in Motor Neuron Disease
Study Purpose:
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 ALSStudy Type:
Observational StudyStudy Category:
Biomarkers/ImagingStudy Status:
EnrollingPhase:
Not ApplicableStudy Chair(s)/Principal Investigator(s):
James D. Berry, MD, MPH (Massachusetts General Hospital)
Clinicaltrials.gov ID (11 digit #):
Neals Affiliated?
YesCoordinating Center Contact Information
Massachusetts General HospitalAlison Clark / .(JavaScript must be enabled to view this email address) / 617-726-4284
.(JavaScript must be enabled to view this email address) 165 Cambridge Street
Suite 600
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 FundsParticipant Duration:
This is a one time study visit at MGH that lasts about 1 to 3 hours.