Autologous Bone Marrow-Derived Stem Cell Therapy for Motor Neuron Disease

Stem Cells Arabia

Status and phase: Unknown; Phase 2; Phase 1

Conditions: Motor Neuron Disease; Primary Lateral Sclerosis; Progressive Bulbar Palsies; Amyotrophic Lateral Sclerosis; Progressive Muscular Atrophy

Treatments: Biological: Stem Cells

Study type: Interventional

Funder types: Other

Identifiers

NCT03067857

SCA-MND1

Details and patient eligibility

About

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.

Full 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.

Enrollment: 40 estimated patients

Sex: All

Ages: 24 to 70 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Patients age 24-20 years old
• Patients with the diagnosis of definite Motor Neuron Disease

Exclusion criteria

• HIV/HBV/HCV
• Malignancies
• Bleeding
• 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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

---

40 participants in 1 patient group

Stem Cells

Experimental group

Description:

Intravenous and Intrathecal thecal transplantation via interventional radiology of purified autologous bone-marrow derived specific stem cell populations.

Treatment:

Biological: Stem Cells