Autologous Cultured Mesenchymal Bone Marrow Stromal Cells Secreting Neurotrophic Factors (MSC-NTF), in ALS Patients.
Condition: Amyotrophic Lateral Sclerosis
MeSH terms: Motor Neuron Disease; Amyotrophic Lateral Sclerosis; Sclerosis
Study type: Interventional
Phase: Phase 1/Phase 2

Summary: 
      The study will evaluate the safety, tolerability and therapeutic effects (preliminary
      efficacy) of injection of autologous cultured mesenchymal bone marrow stromal cells secreting
      neurotrophic factors (MSC-NTF), as a possible treatment for patients with Amyotrophic Lateral
      Sclerosis (ALS) at the early and progressive disease stages.
    

Detailed description: 
      This study is a single center trial to be conducted at the Department of Neurology &
      Laboratory of Neuroimmunology, at the Hadassah Hebrew University Medical Center, Jerusalem in
      Israel. All patients enrolled will have a documented history of ALS disease prior to study
      enrolment. Patients diagnosed as early stage ALS disease with duration of less than 6 months
      and patients diagnosed with progressive stage ALS disease with duration of 6-12 months.
      Overall, 24 patients will be recruited and allocated based on their ALS disease severity to 2
      treatment groups: Group A - 12 patients of early ALS disease stage and Group B - 12 patients
      of progressive ALS disease.

      Eligible patients will be enrolled into the study and will be observed for every 2 weeks
      during a "run in period" of 3 months for determination of the progression rate of the
      disease. During the "run in period" after about 6 weeks following enrollment, patients of
      both study groups will undergo a Bone Marrow Aspiration 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 of both study groups will undergo the
      treatment and MSC-NTF will be transplanted by IM or IT injection to the early and progressive
      ALS patients respectively.

      After the MSC-NTF transplantation patients will be observed on a monthly basis for a post
      treatment follow up period of 6 months.
    

Intervention name(s): MSC-NTF cells transplantation (IM); MSC-NTF cells transplantation (IT)
Intervention type(s): Biological; Biological
Intervention description(s): In early ALS subjects: Autologous MSC-NTF cells will be transplanted under mild anesthesia, intramuscularly into patients' clinically unaffected (or only mildly affected) upper arm biceps and triceps muscles according to a pre-designed grid. Intramuscular injections will be by a 26 gauge needle to a 1.5cm depth (ensuring that injection is into muscle and not adipose tissue). The patients will be injected at 24 sites with a total of 24 million cells; In progressive ALS subjects: Autologous MSC-NTF cells will be transplanted under mild anesthesia intrathecally(via a standard lumbar puncture)with a total of 60 million cells.
Allocation: Non-Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: Mesenchymal Stromal Cells (MSC); Amyotrophic Lateral Sclerosis (ALS); Neuroprotection; Neurodegeneration; Stem cells; ALS