A recent publication in Stem Cells Translational Medicine described an open-label phase I clinical trial that was designed to assess the safety of a stem cell therapy for amyotrophic lateral sclerosis (ALS). The specific procedure involved two repeated injections of autologous bone marrow derived mesenchymal stem cells into the spinal canal of patients with ALS. The researchers found that the injections were safe during their implementation and that they did not have any unsafe effects during a follow-up period of one year.
The phase I clinical trial included 8 patients whose ALS was either definite or probable. The patients underwent a procedure to have mesenchymal stem cells isolated from their bone marrow, and the stem cells were expanded outside the patients’ bodies for 28 days. Unfortunately, a patient died before treatment, so only 7 of the patients then had the spinal canal injections of the stem cells. The two injections were given 26 days apart.
Conventional tests to evaluate the status of ALS in patients were used to assess the impact of the stem cells on the patients’ disease. In the 6 months following the injections, the disease progression did not accelerate, according to the ALS Functional Rating Scale-Revised (ALSFRS-R) score. There were also no serious adverse events observed over a 12 month follow up. Some adverse side effects occurred but subsided with or without treatment within a few days.
The significance of this study is the observation that this particular stem cell therapy procedure appears safe for ALS patients. Further research will help likely determine how stem cell therapy for amyotorophic lateral sclerosis can be used to slow the disease progression, while maintaining high degrees of safety.
Learn more about stem cell therapy for ALS here.