Over 2 million individuals worldwide grapple with the challenges of multiple sclerosis (MS), a debilitating disease affecting the nervous system. Characterized by disruptions in movement, vision, and cognitive function, MS can take a severe turn for some individuals, progressing into secondary progressive MS, for which treatment options remain limited. However, a groundbreaking study has unveiled encouraging results from a pioneering, early-stage clinical trial using stem cells – the body’s “master cells.”
The trial involved the direct injection of brain stem cells into the brains of 15 patients diagnosed with secondary progressive MS. These stem cells, sourced from a meticulously screened single miscarried foetus, possess the unique ability to transform into various types of brain cells, offering the potential to repair damage inflicted by MS.
Before undergoing the groundbreaking procedure, participants underwent a thorough assessment of their disability levels and disease activity spanning three months. Remarkably, most treated patients exhibited high levels of disability, including dependence on wheelchairs. To mitigate the risk of graft rejection, the stem cell injections were administered alongside immune-suppressing drugs. Notably, the 12-month post-treatment period revealed no severe adverse events, though transient side effects such as flu-like symptoms and respiratory infections were reported.
Critical to the success of the trial was the absence of relapse-like symptoms or substantial deterioration in movement and cognitive function – outcomes typically anticipated in the absence of treatment. Advanced magnetic imaging of a subgroup of participants further revealed a correlation between higher stem cell doses and a reduction in brain volume, akin to the effects observed with potent drugs used in early-stage MS treatment. This suggests a potential role for these stem cells in preventing brain inflammation and swelling.
To gauge the efficacy of the brain stem cell treatment, researchers delved into understanding its impact on the intricate processes of the brain’s energy production. In MS, the immune system targets and damages myelin, the protective coating around nerve fibers. Central to this process are macrophages, immune cells crucial for eliminating unwanted intruders, particularly microglial cells found throughout the brain and spinal cord.
Previous research in mice indicated that reprogrammed skin cells into brain stem cells could potentially reduce inflammation and repair MS-induced damage when transplanted into the central nervous system. Moreover, these stem cells could modify the metabolism and reprogram microglia, shifting them from a detrimental to a beneficial state.
In this human trial, the focus turned to the impact of brain stem cell treatment on the brain’s energy production processes. Monitoring changes in the fluid surrounding the brain and in the blood over time, researchers discovered persistent alterations induced by the transplant. Notably, a class of molecules called acylcarnitines, crucial for maintaining cellular energy metabolism, exhibited increased levels in patients receiving higher stem cell doses.
While these findings are undeniably exciting, caution is warranted given the small size of the patient group and the concurrent use of immune-suppressing drugs. Nevertheless, this study represents the first compelling evidence that a single brain stem cell transplant directly into the brain is safe and capable of inducing long-lasting effects in individuals with secondary progressive MS.
The road ahead involves further studies to validate and build upon these promising findings. If successful, this groundbreaking approach could emerge as a valuable treatment option for addressing the advanced stages of MS, offering hope to millions globally who contend with the challenges of this complex neurological condition.
