Researchers investigated the impact of mesenchymal stem cell injections on cognition, function, and biomarkers for neuroinflammation and neurodegeneration.
Late-breaking research announced at ACTRIMS Forum 2024 revealed an association between repeated stem cell transplants and cognitive and functional benefits for patients with multiple sclerosis (MS).
“Intrathecal injection of autologous, bone marrow-derived mesenchymal stem cells (MSC) showed robust clinical and radiological effects in a previous double-blind randomized study from our center,” wrote Dimitrios Karussis, MD, PhD, and coinvestigators.
To build on those findings, Dr. Karussis and his team investigated the safety and efficacy of repeated injections in patients with progressive MS who participated in the previous study.
The interim analysis involved 23 participants, including 14 male and 9 female patients (mean Kurtzke Expanded Disability Status Scale [EDSS] score: 5.9+0.8). Follow-up ranged from 12 to 18 months.
Dr. Karussis and colleagues assessed several measures, including the EDSS, Functional Systems Scores, Timed 25-Foot Walk test, Symbol Digit Modalities Test (SDMT), and serum neurofilament light chain (NFL) and glial fibrillary acidic protein (GFAP) levels. Study participants received at least 2 MSC injections every 3-6 months.
Cognitive & Functional Improvements
The study team recorded multiple Timed 25-Foot Walk tests for 16 patients. According to the findings, their scores improved after the first injection (median 17%) and at one year of follow-up (median 4.5%; mean reduction by 1 second at 12 months, P=0.049).
By the end of the follow-up period, the sum of all Functional Systems Scores decreased from 10.86±3.14 to 9.98±3.04 (P=0.003), indicating reduced disability. Dr. Karussis and colleagues also reported increased cognition in their study participants, with SDMT scores improving by a mean of approximately 2 degrees (from 42.9±11.9 to 45.0±12.3, P=0.01).
In addition, repeated MSC injections were associated with a 12%-35% improvement in patient-reported outcomes.
Neuroinflammation & Neurodegeneration
MSC injections were also linked to decreased biomarkers of neuroinflammation and neurodegeneration.
“Serum levels of NFL and GFAP showed a gradual and consistent reduction after the intrathecal treatments with MSC,” Dr. Karussis and colleagues wrote, noting a mean reduction of approximately 20% across multiple measurements. “The mean reduction at last observation after one year was 35.8% (P<0.002, Wilcoxon paired test).”
The researchers found reduced GFAP levels in all 20 patients who were tested (mean at baseline: 165.7+70.4 and at last observation: 129.4+54, P<0.001, Wilcoxon paired test).
The researchers reported no serious adverse events in their analysis.
Dr. Karussis and colleagues concluded that their “interim analysis of this extension trial shows indications of significant beneficial effects on cognition, neurological functional tests, quality of life and objective biomarkers of neurodegeneration and neuroinflammation” in patients with progressive MS who received repeated MSC injections.
