Autologous Bone Marrow Mesenchymal Stem Cells in the Treatment of Patients With Amyotrophic Lateral Sclerosis (UwmBmmscALS)

University of Warmia and Mazury

Status and phase: Unknown; Phase 1

Conditions: Amyotrophic Lateral Sclerosis

Treatments: Other: Biological: Cell-based therapy

Study type: Interventional

Funder types: Other

Identifiers

NCT02881489

UWM/ALS-MSC.2015/002

Details and patient eligibility

About

The goal of this study is to investigate the safety and tolerability of autologous bone marrow-derived mesenchymal stem cells administration in the individuals with diagnosed amyotrophic lateral sclerosis.

Full description

Amyotrophic lateral sclerosis (ALS) is one of the progressive neurodegenerative disorders, affecting upper and lower motor neurons in the cerebral cortex, brainstem and spinal cord. Hence, the signs of damage motor neurons are both at the peripheral (eg. atrophy), and central (eg. spasticity) level. There is no effective treatment for ALS and the majority of patients die within 5 years after diagnosis, usually due to respiratory failure. Numerous studies on murine models revealed that mesenchymal stem cells (MSCs) successfully improve the clinical and pathological features of ALS. The goal of this nonrandomized, open label study is to investigate the safety and tolerability of autologous bone marrow-derived mesenchymal stem cell transplantation into the individuals with diagnosed amyotrophic lateral sclerosis. This clinical trial is conducted to test the therapeutic (neuroprotective and paracrine) effect of autologous bone marrow-derived mesenchymal stem cells (BM-MSCs). All patients enrolled will have a documented history of ALS disease prior to study enrollment. Patients are recruited for a clinical trial not longer than 1 year from disease diagnosis. Then, patients are divided into two groups: Group I - patients receiving intrathecally one application of BM-MSCs and Group II - patients receiving intrathecally three applications (each administration every two months) of BM-MSCs. Subsequently, autologous bone marrow-derived mesenchymal stem cell transplantation to the cerebrospinal fluid at the site of the spinal cord will be performed. Finally, treatment safety, adverse events and exploratory parameters, including electromyographic (EMG) studies, forced vital capacity (FVC) and functional rating scale (FRS) to establish ALS progression rate will be recorded throughout the duration and in the post-treatment follow up period.

Enrollment: 30 estimated patients

Sex: All

Ages: 18 to 65 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• diagnosis of the ALS disease before the cell transplantation (diagnose established following the El Escorial criteria for definite ALS)
• good understanding of the protocol and willingness to consent
• signed informed consent
• disease duration: up to 2 years
• FVC > 50% / pulmonologist certificate about respiratory function of the patient

Exclusion criteria

• cancer,
• autoimmune diseases
• renal failure,
• subject is a respiratory dependent.
• subject unwilling or unable to comply with the requirements of the protocol
• pregnancy, breastfeeding

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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30 participants in 1 patient group

Autologous BM-MSCs injection

Experimental group

Description:

Intervention: Biological: Cell-based therapy of autologous bone marrow-derived mesenchymal stem cells which are transplanted intrathecally (via a standard lumbar puncture) into the ALS subjects.

Treatment:

Other: Biological: Cell-based therapy