ALS Stem Cell Treatment Deemed Safe in Phase 2 Trial

Findings from a phase 2 clinical trial for amyotrophic lateral sclerosis (ALS) suggests that transplanting human stem cells into the spinal cord may be done safely.

A study published in Neurology did not seek to determine whether or not the treatment was effective, but researchers noted it did not slow down disease progression.

“Though there are 2 serious complications related to the treatment, the level of acceptable risk for treating patients with ALS, where the prognosis is poor and treatments are limited, is arguable higher than that for many benign disorders,” said researcher Jonathan D. Glass, MD.

The open-label study enrolled 15 ALS patients at 3 university hospitals, with every patient having shown their first symptoms within 2 years at the beginning of the study. Participants were divided into 5 treatment groups to receive increasing doses of stem cells by increasing the amount of injections.

Every study participant was given bilateral injections in the cervical spinal cord between the C3 and C5 regions. The final group received injections into both the lumbar (L2-L4) and cervical cord through 2 separate surgical procedures.

Participants received between 10 and 40 injections, with the number of cells injected ranging from 2 million to 16 million. Researchers recorded information on side effects, and measured disease progression using the ALS functional rating scale during 9 months of follow-up.

The results of the trial showed that a majority of the side effects were related to temporary pain associated with medications that suppress the immune system and surgery. There were 2 participants who had serious complications related to treatment, and 1 person who had spinal cord swelling that caused pain, partial paralysis, and sensory loss. One participant developed central pain syndrome.

When the patients’ functioning was compared with 3 historical control groups, researchers found no difference in how rapidly the disease progressed between participants who received stem cells and those who did not. Since the study was small, authors noted that a conclusion on the efficacy of the treatment should not yet be made.

“This study was not designed, nor was it large enough, to determine the effectiveness of slowing or stopping the progression of ALS,” Glass said. “The importance of this study is that it will allow us to move forward to a larger trial specifically designed to test whether transplantation of human stem cells into the spinal cord will be positive treatment for patients with ALS.”