While advances in the development of immunomodulatory disease-modifying therapies (DMTs) have had a substantial impact on the severity and frequency of relapses ( Derfuss et al., 2020), within 30 years of diagnosis, two-thirds of RRMS patients will ultimately transition into the debilitating secondary progressive (SP) phase of the disease ( Scalfari et al., 2014). The disease typically manifests in a relapsing-remitting (RR) form marked by sporadic attacks of neurological dysfunction (i.e., clinical relapses) followed by a (full or partial) functional recovery. Multiple sclerosis (MS) is a chronic neuroinflammatory condition that affects over 2 million people worldwide ( Stenager, 2019). Herein, we explore the current state of preclinical and clinical evidence supporting the use of stem cells in treating PMS and we discuss prospective hurdles impeding their translation into revolutionary regenerative medicines. Ostensibly providing neurotrophic support, immunomodulation and cell replacement, stem cell transplantation holds substantial promise in combatting the complex pathology of chronic neuroinflammation. In looking to address these unmet needs, the multifactorial therapeutic benefits of stem cell therapies are particularly compelling. The few medications approved for the treatment of PMS are typically limited in their efficacy to active forms of the disease, have little impact on slowing degeneration, and fail to promote repair. Therapeutic intervention in progressive MS (PMS) suffers from a lack of well-characterized biological targets and, hence, a dearth of successful drugs.
While there are several effective disease-modifying therapies able to address the inflammatory relapses associated with RRMS, most patients will inevitably advance to a progressive disease course marked by a gradual and irreversible accrual of disabilities. MS patients typically present with a relapsing-remitting (RR) disease course, manifesting as sporadic attacks of neurological symptoms including ataxia, fatigue, and sensory impairment. Multiple sclerosis (MS) is a chronic inflammatory disease of the central nervous system characterized by demyelination and axonal degeneration. 2Department of Clinical Neurosciences and National Institute for Health Research (NIHR) Biomedical Research Centre, University of Cambridge, Cambridge, United Kingdom.1Cambridge Innovation Technologies Consulting (CITC) Limited, Cambridge, United Kingdom.Nicaise 2, Rosana-Bristena Ionescu 2, Regan Hamel 2, Luca Peruzzotti-Jametti 2* and Stefano Pluchino 2*
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