Mesenchymal Stem Cells for Treatment of Amyotrophic Lateral Sclerosis (ALS)

Study Overview
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Brief Summary
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The purpose of this study is to determine the safety of injecting mesenchymal stem cells through intraspinal delivery for the treatment of ALS.

Detailed Description
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A single patient safety study for harvesting, expanding ex vivo and injecting autologous mesenchymal stem cells (MSC's) into the subarachnoid space of a patient with amyotrophic lateral sclerosis (ALS). Cells will be isolated from adipose tissue by subcutaneous biopsy and expanded using an FDA-approved protocol. They will then be injected by lumbar puncture into the cerebrospinal fluid. Injection will be completed in the in- patient clinical research unit (CRU). the patient will be followed for two years.

Official Title
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A Single Patient Treatment Protocol for Autologous Mesenchymal Stem Cell Intraspinal Therapy in Amyotrophic Lateral Sclerosis (ALS)

Conditions
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Amyotrophic Lateral Sclerosis

Intervention / Treatment
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* Biological: autologous mesenchymal stem cells


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: age greater than 18 years, if female, must be menopausal or had hysterectomy resident and citizen of the United States history of a chronic onset of a progressive motor weakness able to comply with protocol requirements can provide written consent Exclusion Criteria: does not have renal disease (Creatine > 2.0) does not have active systemic disease does not have any clinically significant abnormalities on prestudy laboratory evaluation does not have any clinically significant medical condition (e.g.,within 6 months of baseline, had a myocardial infarct, angina pectoris, and/or congestive heart failure), that in the opinion of the investigator, would compromise the safety of the patient does not have a history of cancer including melanoma with the exception of localized skin cancers (with no evidence of metastasis, significant invasion,or re-occurrence within 3 years of baseline). has not used an investigational drug within 30 days of baseline visit does not have a tracheostomy does not have a Beck's Depression Inventory score >16

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Intervention/Treatment |
| --- |
|Biological: autologous mesenchymal stem cells|single intrathecal dose of 10x6 cells by lumbar puncture.|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Safety | Clinical monitoring of possible reaction to intrathecal MSC delivery including blood pressure, temperature and pain score. | 2 year follow-up | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Neurologic disability score | Quantitative, summated, manual muscle testing | Two year follow-up |