Multiple sclerosis (MS) is a debilitating disease with few treatment options. But in a new clinical trial, scientists have tested a promising new therapy involving injections of stem cells, which seems to slow progression of the disease.
In patients with MS, the immune system begins mistakenly attacking myelin, the tissue that encases and protects nerve fibers. As this sheath sustains more and more damage, it disrupts electrical signals traveling through nerves, resulting in reduced mobility, balance, sensation and muscle strength.
The disease usually starts with a form called relapse-remitting MS, which involves alternating phases of symptoms and remission. After a while, however, the disease can sometimes progress to a more severe stage called secondary progressive MS, where symptoms steadily get worse with no periods of remission to break it up.
Recent studies in mice and rats have suggested that stem cell treatments may be able to help slow or stop the progression to the later form of MS. So now, researchers have conducted a clinical trial in humans to determine if such a therapy is safe and effective.
Neural stem cells were taken from the brain tissue of a miscarried fetal donor, cultivated and injected directly into the brains of 15 patients with secondary MS. Doses varied from 5, 10, 16 or 24 million stem cells, and the patients were then followed up monthly for a year.
At the beginning of the trial, all patients had high levels of disability, with many needing a wheelchair, but during the trial period none of them experienced worsening physical or cognitive symptoms. The researchers say that this suggests that the treatment stabilized the disease progression.
In a subset of patients, the team examined changes in brain volume over time. Patients that received higher doses of stem cells showed smaller reductions in brain volume, which the scientists hypothesize is because the treatment dampened inflammation. Analysis of the metabolites and lipids in the patients’ cerebrospinal fluid suggested that the stem cells had a protective effect on the nerves.
Importantly, there were no deaths or serious adverse effects in the study cohort. The minor side effects that were seen all proved to be temporary or reversible.
While the results seem to be promising, the team acknowledges that the study has its limitations. For one, it only involved a small group, and confirming the protective effects was hard when starting with an advanced level of disability in patients. On the plus side, it was shown to be safe, so it can proceed to the next stage of clinical trials.
The research was published in the journal Cell Stem Cell.
Source: University of Colorado Anschutz
