Autologous Mesenchymal Bone Marrow Stromal Cells Secreting Neurotrophic Factors (MSC-NTF), in Patients With Amyotrophic Lateral Sclerosis (ALS)
Condition: Amyotrophic Lateral Sclerosis
MeSH terms: Motor Neuron Disease; Amyotrophic Lateral Sclerosis; Sclerosis
Study type: Interventional
Phase: Phase 2

Summary: 
      The study will evaluate the safety, tolerability and therapeutic effects of transplantation
      of escalating doses of autologous cultured mesenchymal bone marrow stromal cells secreting
      neurotrophic factors (MSC-NTF), in patients with amyotrophic lateral sclerosis (ALS).
    

Detailed description: 
      This is a phase IIa prospective, open label, dose-escalating, three patient-group clinical
      study, to evaluate the safety, tolerability and preliminary efficacy of autologous cultured
      mesenchymal bone marrow stromal cells secreting neurotrophic factors (MSC-NTF), as a potent
      treatment for patients with Amyotrophic Lateral Sclerosis (ALS) at the early disease stages.
      This study is a single center trial. It is anticipated that the study will be conducted at
      the Department of Neurology & Laboratory of Neuroimmunology, at the Hadassah Hebrew
      University Medical Center, Jerusalem in Israel. In addition, ALS patients could also be
      referred to the clinical site above by other medical centers.

      All patients enrolled will have a documented history of ALS disease prior to study
      enrollment. Patients diagnosed as early stage ALS disease with duration of less than 2 years.
      ALS patients identified as "predisposed" will be approached and requested to sign an Informed
      Consent Form (ICF). Overall, 14 patients will be recruited.

      Treatment will start with the lowest dose (94x106 cells) and the dose will be increased to
      the next medium and high dose (141x106 and 188x106 respectively), for the next patients group
      only following safety analysis.

      The expected duration of patient screening period prior to enrollment into this study is
      in-between two weeks up to 2 days prior to the study enrollment day during visit 2
      (verification of compliance with inclusion/exclusion criteria including clinical laboratory
      results). Eligible patients will be enrolled into the study and will be observed for every
      month during a "run in period" of 3 months for determination of the progression rate of the
      disease (allowing a time window of ± 5 days for all visits). During the "run in period" after
      about 6 weeks following enrollment, patients of both study groups will undergo a Bone Marrow
      Aspiration (BMA) procedure and MSC-NTF cells will be produced from the bone marrow aspirate
      based on Brainstorm Cell Therapeutics Ltd proprietary method. On the last "run in period"
      visit, patients will undergo the treatment and MSC-NTF will be transplanted by IM+IT to the
      early ALS patients.

      After the MSC-NTF transplantation patients will be observed on a monthly basis for a post
      treatment follow up period of 6 months (allowing a time window of ± 5 days for all visits).
      Treatment safety, adverse events and exploratory parameters, to establish ALS progression
      rate assessment of the disease will be recorded throughout the duration of the "run in
      period" and the post treatment follow up period.
    

Intervention name(s): MSC_NTF cells transplantation by multiple intramuscular injections at 24 separate sites, in addition to a single intrathechal injection into the CSF
Intervention type(s): Biological
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: Mesenchymal Strmal Cells (MSC); Amyotrophic Lateral Sclerosis (ALS); Neuroprotection; Neurodegeneration; Stem cells