In a clinical trial, an international research team has achieved a significant milestone in the battle against multiple sclerosis (MS). The study, spearheaded by the University of Cambridge and partners, involved injecting neural stem cells directly into the brains of patients with secondary progressive MS. Remarkably, over a 12-month period, no patient experienced an increase in disability or worsening of symptoms. This trial suggests that the therapy may not only be safe but could also stabilize the otherwise relentless progression of the disease. The findings, published in Cell Stem Cell, indicate that the larger the dose of injected stem cells, the more pronounced the reduction in brain volume loss, hinting at a potential neuroprotective effect.
- tem cell therapy trial stabilizes MS progression, no disability increases over 12 months.
- International team injects neural stem cells into patient brains, sees neuroprotective effects.
- Treatment proven safe, tolerated well, meets primary endpoints on feasibility and safety.
The quest for an effective treatment for multiple sclerosis (MS) has been long and fraught with challenges. The condition, which affects over two million people worldwide, often leads to a secondary progressive phase, characterised by a steady increase in disability. However, a ray of hope has emerged from the laboratories and clinics of several esteemed institutions, where a pioneering early-stage clinical trial has been conducted.
A novel approach to stem cell therapy
At the heart of this trial is the use of allogeneic human neural stem/progenitor cells (hNSCs), derived from the brain tissue of a single miscarried foetal donor. These cells were transplanted via intracerebroventricular injection, a method chosen for its potential to target core drivers of MS progression. The trial’s safety and the cells’ neuroprotective qualities have been the focus, providing a foundation for future explorations into their efficacy in combating the disease.
The trial was a collaborative endeavour, with teams from the University of Cambridge, University of Milan Bicocca, Hospital Casa Sollievo della Sofferenza, S. Maria Terni, Ente Ospedaliero Cantonale and the University of Colorado all contributing to this groundbreaking research. The international effort underscores the global commitment to finding a solution to MS that transcends borders and brings together the brightest minds in the field.
Assessing the impact on patients
All participants in the study showed high levels of disability at the outset, yet remarkably, none demonstrated any increase in disability or a worsening of symptoms during the follow-up period. This stability is a notable achievement, considering the typical progression of the disease. Moreover, the trial’s findings suggest that the larger the dose of injected stem cells, the smaller the reduction in brain volume associated with disease progression.
Perhaps the most critical aspect of the research was the absence of treatment-related deaths or serious adverse events, a reassuring confirmation of the therapy’s safety. The hNSCs were tolerated well by patients, and the trial met its primary endpoints, which focused on the feasibility, safety, and tolerability of the stem cell transplantation.
The study also delved into the metabolomic and lipidomic responses, revealing time- and dose-dependent changes. Interestingly, levels of acyl-carnitines and fatty acids in the cerebrospinal fluid increased, suggesting a metabolic response to the treatment. Moreover, MRI scans corroborated the clinical outcomes, showing no unexpected findings related to the therapy, despite MS-related disease activity.
A step towards future treatments
Professor Stefano Pluchino of the University of Cambridge expressed cautious optimism about the findings. “We desperately need to develop new treatments for secondary progressive MS, and I am cautiously very excited about our findings, which are a step towards developing a cell therapy for treating MS,” he stated. The trial’s success has been a collaborative triumph, with Professor Angelo Vescovi of the University of Milan Bicocca noting the nearly three-decade journey from the discovery of brain stem cells to this promising therapeutic application.
Implications for MS sufferers
The MS Society has also recognised the significance of this study. Caitlin Astbury, Research Communications Manager at the MS Society, commented on the exciting nature of the results and the potential for stabilising disability progression. This sentiment is echoed across the research community as these findings could represent a paradigm shift in how MS, particularly in its secondary progressive phase, is treated.
While the results are indeed promising, it is crucial to acknowledge the study’s limitations. As an early-stage trial with a small cohort, it primarily sought to establish safety and set the stage for larger efficacy studies. The next phase of clinical trials will be critical in ascertaining the long-term benefits and potential for widespread treatment application.
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