An early clinical trial investigating a new form of stem cell therapy for multiple sclerosis (MS) has demonstrated neurological improvement and enhanced quality of life for patients. The research, published in JAMA, suggested reversal of some of the debilitating effects of MS.
Researchers at the Northwestern University Feinberg School of Medicine, Chicago, USA, assessed a small group of 145 patients with MS. The novel approach used low-dose chemotherapy combined with an infusion of the patients' own stem cells in order to 'reset' their immune systems.
The study employed a standardised measure of disability associated with neurological impairment, the Expanded Disability Status Scale (EDSS), which showed a significant improvement in 50 percent of patients tested at two years and 64 percent of patients tested at four years.
'This is the first report of significant and sustained improvement in the EDSS score following any treatment for MS,' say the authors.
MS disease progression and severity were also assessed according to the total volume of brain lesions, using magnetic resonance imaging, and a significant decrease in overall lesion volume was evident following stem cell therapy.
MS is an immune-mediated disorder of the central nervous system in which the immune system mistakenly attacks the protective coating surrounding fibers in the brain and spine known as the myelin sheath. Following diagnosis, approximately 50 percent of patients with MS are unable to continue employment ten years after diagnosis or to walk after 25 years.
Despite widespread research efforts, there is currently no commercialised treatment for MS that is effective in reversing neurological impairment or improving patients' quality of life. Available drugs are only able to slow progression of the disease.
The authors note that the current study is limited by the lack of a control group and because effects were only seen among patients with the relapsing-remitting form of the disease and not in those with secondary-progressive MS, which is more severe. Nor were effects seen in those who had been affected by the disease for more than ten years.
Nonetheless the findings should provide a strong basis for initiating a randomised study within an appropriate patient population, the authors conclude.