Therapeutic Treatment of Amyotrophic Lateral Sclerosis

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 the respiratory failure. Numerous studies on murine models revealed that mesenchymal stem cells (MSCs) successfully improve the clinical and pathological features of ALS patients. The goal of this nonrandomized, open label study is to investigate the safety and tolerability of allogeneic Wharton's jelly-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 allogeneic Wharton's jelly-derived mesenchymal stem cells (WJ-MSCs). All patients enrolled will have a documented history of ALS disease prior to enrollment. Patients are recruited for a clinical trial no more than 1 year from the disease diagnosis. Then, patients are divided into two groups: Group I - patients receiving intrathecally one application of WJ-MSCs and Group II - patients receiving intrathecally three applications (each administration every two months) of WJ-MSCs. Subsequently, allogeneic Wharton's jelly-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.