As a recently completed phase 1 clinical trial shows, stem cells can be safely applied in patients with amyotrophic lateral sclerosis (ALS). The results of the study conducted in South Korea have been published in the journal STEM CELLS Translational Medicine.
The trial, which was conducted by a team of researchers at Hanyang University and Corestem Inc. (Seoul), and Inje University College of Medicine in Busan, had two main goals: to test the safety of stem cell treatment for ALS patients and to figure out whether two stem cell injections bring better therapeutic results than a single injection.
The study involved seven patients with definite or probable ALS. After a lead-in period which lasted for three months, the researchers isolated bone marrow mesenchymal stem cells from each of the patients. The stem cells were isolated twice from each patient, at an interval of 26 days. The collected cells were cultivated at the lab for 28 days prior to being injected back to the patients. The cells were administered intrathecally, i.e. in cerebrospinal fluid space, which did no harm to the patients, in two separate treatments given with an interval of 26 days apart.
In a pilot study that preceded this trial the researchers gave their participants a single injection of mesenchymal stem cells and watched patients for 6 months. In the phase I clinical trial, the researchers wanted to understand whether two stem cells injections would be more beneficial for the patients. They also planned to follow up patients longer – for 12 months instead of 6 months – to deeper investigate the safety issues of the treatment.
As the researchers report, no serious adverse events were observed during this period in any of the patients. Also, the disease did not show progression in any of the trial participants over the 12 months post treatment. Currently, the team is working on a randomized, semi-double blind controlled phase 2 clinical data involving 72 ALS patients.