Hanyang University and Corestem, both in Seoul, South Korea, and Inje University College of Medicine in Busan released positive results in a phase I clinical trial for amyotrophic lateral sclerosis (ALS).

ALS is a rapidly progressive, invariably fatal neurological disease that attacks the motor nerve cells (neurons) responsible for controlling voluntary muscles. The Hanyang University team’s phase I trial had two goals: to test whether stem cells were a safe treatment for ALS, and to learn whether two injections of the cells might prove more beneficial than a single injection.

“In our pilot study we followed a group of ALS patients for six months after giving them a single injection of mesenchymal stromal cells (MSCs) and found the treatment to be both safe and feasible,” said the trial’s co-leader, Seung Hyun Kim, Ph.D., director of Hanyang University Hospital’s Cell Therapy Center and professor in Hanyang University’s department of neurology. “In this next phase, we wanted to see if two injections would prove even more beneficial, and we wanted to follow the participants for a longer period of time to determine if the treatment proved safe for a longer term than in the pilot phase.”

MSCs, which are found in bone marrow, generate bone, cartilage and fat cells that support the formation of blood and fibrous connective tissue. They have emerged as a potentially promising treatment for ALS due to their ability to regenerate lost or damaged cells and for their anti-inflammatory capabilities.

Eight patients with definite or probable ALS were enrolled in the new study, although one died shortly after enrollment. After a three-month lead-in period, MSCs were isolated from each patient’s bone marrow two times, at an interval of 26 days, with each group of MSCs then expanded in the lab for 28 days before being injected into the donor patients. The seven patients received intrathecal (cerebrospinal fluid space) injections of their own MSCs in two separate treatments given 26 days apart.

Kim said, “Intrathecal injections have the advantages of not only avoiding invasive surgical technique that had been done in the previous other group’s study, but also it’s easy to do repeated procedures without harm to the patients.”

Their ALS functional status and safety was then evaluated for 12 months after the first injection.

“No serious adverse events were observed during this period,” said co-leader Ki-Wook Oh, M.D., also of Hanyang University’s neurology department. “Additionally, there was no advancement in ALS symptoms in any of the patients during the 12-month period.”

Kim said, “This study shows that stem cells as a therapeutic approach for ALS are feasible and well-tolerated at least for 12 months, supporting the need for a late-stage clinical trial to examine their in-depth safety, biological effects and efficacy. Randomized, semi-doubleblind, controlled phase II clinical data on 72 ALS patients, which recently was submitted to the Korean FDA, will be released in the near future.”