Autologous Purified Bone-Marrow-Derived Stem Cell Therapy for Motor Neuron Disease
Overview
- Phase
- Phase 1
- Intervention
- Not specified
- Conditions
- Motor Neuron Disease
- Sponsor
- Stem Cells Arabia
- Enrollment
- 40
- Primary Endpoint
- Changes in Functional Rating Scale - via questionnaire
- Last Updated
- 7 years ago
Overview
Brief Summary
Herein, the investigators study the safety and efficacy of transplanting purified autologous bone marrow-derived stem cells transplanted via the intrathecal route by interventional radiology and the intravenous route.
Detailed Description
A motor neuron disease (MND) is any of several neurological disorders that selectively affect motor neurons, the cells that control voluntary muscles of the body. They include amyotrophic lateral sclerosis (ALS), primary lateral sclerosis (PLS), progressive muscular atrophy (PMA), progressive bulbar palsy (PBP) and pseudobulbar palsy; spinal muscular atrophies are also sometimes included in the group.They are neurodegenerative diseases that cause increasing disability and eventually, death. In spite of intensive research, motor neuron diseases (MNDs) are still incurable. Some of the key factors in this failure to find a cure have been the lack of human cell-based disease models for molecular analysis and drug screening and the difficulty of obtaining homogeneous populations of specific cell types for clinical applications. Stem cell biology has the potential to transform our understanding of disease processes and to revolutionize our approach to develop novel therapies for MNDs. In this study, the investigators suggest a protocol for the treatment of MND by transplantation of purified autologous bone marrow-derived stem cells via the intrathecal and intravenous route.
Investigators
Eligibility Criteria
Inclusion Criteria
- •Patients age 24-20 years old
- •Patients with the diagnosis of definite Motor Neuron Disease
Exclusion Criteria
- •HIV/HBV/HCV
- •Malignancies
- •Pneumonia
- •Renal failure
- •liver dysfunction
- •Severe anemia (hb \< 8)
- •Bone Marrow diseases
- •Pregnancy and lactation
- •Acute medical conditions/infections such as respiratory infections
- •ventilatory support
Outcomes
Primary Outcomes
Changes in Functional Rating Scale - via questionnaire
Time Frame: 6 months
Secondary Outcomes
- Change in SF-36 (The Short Form (36) Health Survey is a 36 Item)(4 months)
- Change in Forced Vital Capacity (FVC)(4 months)
- Change in Appel Scale(4 months)