Safety and Efficacy of Repeated Administrations of NurOwn® in ALS Patients

Conditions:Neurology, Neurology, ALS
Therapuetic Areas:Neurology, Other
Age Range:18 - 60
Start Date:August 28, 2017
End Date:July 30, 2020
Contact:Ralph Z. Kern, MD

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A Phase 3, Randomized Double-Blind, Placebo-Controlled Multicenter Study to Evaluate Efficacy and Safety of Repeated Administration of NurOwn® (Autologous Mesenchymal Stem Cells Secreting Neurotrophic Factors) in Participants With ALS

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

Neurotrophic factors (NTFs) are potent survival factors for embryonic, neonatal, and adult
neurons and are considered potential therapeutic candidates for ALS. Delivery of multiple
NTFs to the immediate environment of afflicted neurons in ALS patients is expected to improve
their survival and thus slow down disease progression and alleviate symptoms. NTF-secreting
mesenchymal stromal cells (MSC-NTF cells) are a novel cell-therapeutic approach aimed at
effectively delivering NTFs directly to the site of damage in ALS patients.

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

Inclusion Criteria:

- 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

Exclusion Criteria:

- 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

- 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
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Principal Investigator: Namita A. Goyal, MD
Phone: 949-824-3990
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