Autologous Purified Bone-Marrow-Derived Stem Cell Therapy for Motor Neuron Disease
试验速览
- 阶段
- 1 期
- 入组人数
- 40
- 主要终点
- Changes in Functional Rating Scale - via questionnaire
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 24 Years 至 70 Years(Adult, Older Adult)
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients age 24-20 years old
- •Patients with the diagnosis of definite Motor Neuron Disease
排除标准
- •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
研究组 & 干预措施
Stem Cells
Intravenous and Intrathecal thecal transplantation via interventional radiology of purified autologous bone-marrow derived specific stem cell populations.
干预措施: Stem Cells (Biological)
结局指标
主要结局
Changes in Functional Rating Scale - via questionnaire
时间窗: 6 months
次要结局
- 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)
