Study Purpose:This study will evaluate the safety and efficacy of repeated administration of NurOwn® (MSC-NTF cells) therapy, which is based on transplantation of autologous bone marrow derived mesenchymal stromal cells (MSC), which are enriched from the patient's own bone marrow, propagated ex vivo and induced to secrete Neurotrophic factors (NTFs).
The autologous NurOwn® (MSC-NTF cells) are back-transplanted into the patient intrathecally by standard lumbar puncture where neurons and glial cells are expected to take up the neurotrophic factors secreted by the transplanted cells
Amyotrophic Lateral Sclerosis (ALS)
Type of Intervention:
NurOwn® (MSC-NTF cells), Placebo, Bone Marrow aspiration
Study Chair(s)/Principal Investigator(s):
Merit E. Cudkowicz, MD, Massachusetts General Hospital, Robert H. Brown, MD, PhD, UMass Medical School, Anthony J. Windebank, MD, Mayo Clinic, Namita A. Goyal, MD, UC Irvine, Robert G. Miller, MD, California Pacific Medical Center (CPM) Research Institute, Robert Baloh, MD, Ph.D., Cedars-Sinai Medical Center
Coordinating Center Contact Information
Full Study Summary:
Participants meeting the inclusion and exclusion criteria will be randomized and will undergo bone-marrow aspiration. MSC of the participants randomized to the treatment group will be induced into MSC-NTF cells. Participants will undergo a total of three intrathecal (IT) transplantations with NurOwn® (MSC-NTF cells) or matching placebo at three bi-monthly intervals
Estimated Study Start Date:
08 / 28 / 2017
Estimated Study Completion Date:
10 / 30 / 2020
Posting Last Modified Date:
10 / 06 / 2021
Date Study Added to neals.org:
09 / 12 / 2017
Can participants use Riluzole?
- ALS diagnosed as possible, laboratory-supported probable, probable, or definite as defined by revised El Escorial criteria.
- Having onset of ALS disease symptoms, including limb weakness within 24 months at the Screening Visit.
- ALSFRS-R ≥ 25 at the screening Visit.
- Upright slow vital capacity (SVC) measure ≥ 65% of predicted for gender, height, and age at the screening Visit.
- Rapid progressors
- Participants taking a stable dose of Riluzole are permitted in the study
- Citizen or permanent resident of the United States or Canadian citizen able to travel to a US site for all follow-up study visits
- Prior stem cell therapy of any kind
- History of autoimmune or other serious disease (including malignancy and immune deficiency) that may confound study results
- Current use of immunosuppressant medication or anticoagulants (per Investigator discretion)
- Exposure to any other experimental agent or participation in an ALS clinical trial within 30 days prior to Screening Visit
- Use of RADICAVA (edaravone injection) within 30 days of screening or intent to use edaravone at any time during the course of the study including the follow up period
- Use of non-invasive ventilation (BIPAP), diaphragm pacing system or invasive ventilation (tracheostomy)
- Feeding tube
- Pregnant women or women currently breastfeeding
University of California Irvine Alpha Stem Cell Clinic
Cedars-Sinai Medical Center
Los Angeles, California
California Pacific Medical Center
San Francisco, California
Massachusetts General Hospital
University of Massachusetts Medical School