Intraventricular autologous mesenchymal stem cell injection with navigation to treatment of amyotrophic lateral sclerosis: preliminary results of a phase i trial

Amyotrophic lateral sclerosis (ALS) is a devastating progressive paralytic disorder due to the death of motor neurons in the brain and spinal cord. ALS patients typically die within 3–5 years from diagnosis due to respiratory failure. Therapeutic options for ALS are limited to a single medication and supportive care, thus driving the search for innovative approaches to slow disease progression and improve survival.In this study, three patients with recently diagnosed ALS observed for determination of progress of disease for six months. In this period, patients followed clinically with revised ALS Functional Rating Scale (ALSFRS-R) questionnaire and Pulmonary Function Test (PFT). After six months, the patients underwent intraventricular injection of 1*107 autologous bone marrow mesenchymal stem cell with guidance of navigation under intravenous sedation.The average time of follow up for patients are 6 months. Except positional headache that was self-limited, it did not observe any side effects including meningitis, hydrocephalus or intraventricular bleeding. In spite of concerns about respiratory intolerance under sedation in these patients because of respiratory muscle weakness, all patients could tolerate and intubation was not needed. During follow up, the patients did not experience any change in progression speed of disease.These data show that intraventricular autologous mesenchymal stem cell injection at the dose used in this study is safe. Long- term follow up and comparing with control group are needed to assess the efficacy of this way to ALS treatment.

کلیدواژه ها:

Amyotrophic Lateral Sclerosis ، Mesenchymal Stem Cell ، Intra-ventricular injection