Autologous Bone Marrow-Derived Stem Cell Therapy for Motor Neuron Disease
|Motor Neuron Disease Amyotrophic Lateral Sclerosis Primary Lateral Sclerosis Progressive Muscular Atrophy Progressive Bulbar Palsies||Biological: Stem Cells||Phase 1 Phase 2|
|Study Design:||Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
|Official Title:||Autologous Purified Bone-Marrow-Derived Stem Cell Therapy for Motor Neuron Disease|
- Changes in Functional Rating Scale - via questionnaire [ Time Frame: 6 months ]
- Change in Appel Scale [ Time Frame: 4 months ]
- Change in SF-36 (The Short Form (36) Health Survey is a 36 Item) [ Time Frame: 4 months ]
- Change in Forced Vital Capacity (FVC) [ Time Frame: 4 months ]
|Study Start Date:||September 2016|
|Estimated Study Completion Date:||August 2019|
|Estimated Primary Completion Date:||January 2019 (Final data collection date for primary outcome measure)|
Experimental: Stem Cells
Intravenous and Intrathecal thecal transplantation via interventional radiology of purified autologous bone-marrow derived specific stem cell populations.
Biological: Stem Cells
Autologous bone marrow-derived stem cell populations injected via the intravenous and intrathecal routes.
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.