Intraspinal delivery of bone marrow stromal cell-derived neural stem cells in patients with amyotrophic lateral sclerosis: A safety and feasibility study

• In this study, 11 patients with ALS according to El Escorial criteria were selected.
• Intraspinal injection of bone marrow derived neural stem cells was performed.
• None of the patients had perioperative mortality or major morbidity.
• Patients experienced a temporary stabilization for the first few months post-injection.

BackgroundStem cells have been used in several studies with different methodologies to treat patients with ALS.MethodsIn this safety and feasibility study, 11 patients with definite or probable ALS according to El Escorial criteria were selected. 3 patients were excluded due to inadequate bone marrow or safety measures after acquisition of bone marrow. Bone marrow stromal cell-derived neural stem cells were injected in C7-T1 spinal cord under general anesthesia. Patients were followed for 12 months after injection with manual muscle testing, ALSFRS-R, quality of life changes, pulmonary function test and electromyography.ResultsNone of the patients had perioperative mortality or major morbidity. One patient had temporary deterioration in lower extremities after injection which improved after a few weeks. In the 12 months post-injection, only one patient died due to pulmonary embolism. From the remaining 7 patients, all had a stable course after 4 months and 5 were stable for the first 8 months post-injection and deteriorated afterwards.DiscussionIn this study, intraspinal injection of bone marrow derived neural stem cells appears to be safe. Patients experienced a temporary stabilization for the first few months post-injection and then gradually deteriorated.