Stem cell therapy may slow ALS functional decline, researchers say
Treatments generally safe, but more studies needed to confirm benefits
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- Stem cell therapies are generally safe, well tolerated, and show preliminary potential in slowing functional decline for patients with ALS.
- Controlled trials indicate that treated patients experience significantly less disease progression, though findings remain exploratory due to variable study designs.
- Future research requires large-scale, multicenter randomized clinical trials with standardized protocols to confirm definitive clinical effectiveness for ALS.
Stem cell therapies are generally safe and well tolerated in people with amyotrophic lateral sclerosis (ALS), and preliminary evidence from clinical trials suggests they may also help slow functional decline, according to a systematic review and meta-analysis of several published studies.
Researchers found that people given stem cell therapies showed significantly less disease progression than those in control groups. However, studies comparing patients’ disease progression before and after treatment did not provide similarly consistent evidence of benefit.
The findings provide “preliminary evidence suggesting potential therapeutic benefit in slowing disease progression in selected patients,” the researchers wrote. “Nevertheless, the existing evidence base remains exploratory, and definitive conclusions regarding clinical effectiveness cannot yet be drawn.”
“Future research should prioritize large-scale, multicenter [randomized clinical trials] with standardized cell manufacturing protocols and longer follow-up periods,” they said.
The study, “The Effectiveness Based on Optimal Dose and Administration Route, and Safety Profiles of Stem Cells and Derived Products in the Treatment of Patients With Amyotrophic Lateral Sclerosis: A Systematic Review and Meta-Analysis,” was published in Stem Cells International.
Studies show mixed results
Stem cell therapies have attracted growing interest as a potential treatment for ALS. Stem cells have the potential to replicate rapidly and develop into many different cell types in the body. Beyond their ability to replace damaged nerve cells, they may also release molecules that help support and protect the remaining nerve cells.
Several types of stem cells have been tested in ALS clinical trials, with different doses and routes of administration. Results, however, have been mixed. Some studies have suggested that treatment may temporarily slow functional decline, while others have found little or no benefit. It also remains unclear whether cell dose or delivery method affects treatment effectiveness or safety.
To learn more, researchers in Iran conducted a systematic review and meta-analysis of the available clinical evidence to assess the effectiveness and safety of stem cell therapies in ALS, with a particular focus on cell dose and route of administration.
The researchers identified 31 studies published from 2009 to 2023, involving 830 people with ALS. Seven were controlled trials, involving a total of 370 participants, in which people receiving stem cell therapy were compared with a separate control group that did not receive the stem cell intervention. The remaining 24 studies, involving 460 participants, were single-arm studies that assessed patients’ outcomes before and after treatment.
The studies varied widely in the stem cells used, doses, and delivery methods. Bone marrow-derived mesenchymal stem cells were the most common, although several other cell types were tested. Doses ranged from fewer than 1 million to hundreds of millions of cells and could be given once or repeatedly. Intrathecal injection, which delivers cells into the fluid surrounding the brain and spinal cord, was the most common route, used in 18 studies. Cells were also delivered into a vein, muscle, or via combined methods of administration.
Across the studies, stem cell therapies were generally well tolerated. Most side effects were mild or temporary, including headache, fever, back pain, nausea, fatigue, and injection-site pain. Serious adverse events were uncommon and generally attributed to ALS progression, other health problems, or treatment procedures rather than the stem cell products.
Findings on effectiveness, however, were mixed. Some studies suggested that stem cell therapy could temporarily slow functional decline, as measured by the ALS Functional Rating Scale (ALSFRS), while others found little or no benefit.
When researchers pooled comparable data from three controlled trials, a clearer benefit emerged. The analysis covered 52 people who received stem cell therapy and 52 controls. Stem cell therapy was associated with significantly less functional decline, with benefits observed at both high and low doses.
A separate pooled analysis of data from the seven single-arm studies produced less consistent findings. Compared with before treatment, functional decline did not differ significantly at three, six, or nine months, although a significant difference was observed at 12 months. The route of administration did not significantly affect ALSFRS outcomes.
“Stem cell therapy for ALS appears to be safe, with preliminary evidence suggesting that stem cell therapy may attenuate the progression of ALS, as shown by improved ALSFRS scores in controlled trials,” the researchers wrote.
However, because no significant differences were observed in the single-arm studies, they said, definitive conclusions cannot be drawn from existing evidence.
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