Autologous adult human mesenchymal stem cells: a neuroprotective therapy for multiple sclerosis

MS is a disease where nerves and its insulation layer are damaged leading to symptoms. Over time the nerve damage accumulates leading to fixed and progressive disability in the vast majority of patients. Therapies for MS presently are limited only to partially reducing the frequency of individual attacks. There are no effective treatments to protect the nerves. This study is trying to explore whether stem cells taken from the patients bone marrow or skin could through a cell infusion (similar to blood transfusion) protect the nerves of the MS patient.

Multiple sclerosis (MS) is the commonest cause of neurological disability in young adults. It is a cause of significant morbidity and mortality in the industrialised world with prevalence estimates ranging from 110-175 per 100 000 in the UK. It is a multifocal and multiphasic immune mediated disease characterised pathologically by inflammatory demyelination, axonal injury and partial remyelination. Therapeutic treatments of MS thus have two aims; to prevent (disease modifying) and to repair damage that has already occurred. Although some advances in treatment to reduce relapse rate have been made in the last decade, little has been achieved in terms of definitive treatments for relapse, prevention or repair of fixed disability and disease progression. The lack of such therapies represents a substantial gap in the treatment of MS. There is a need to develop treatments that prevent axonal loss, the primary correlate of clinical disability. This first into man proposal builds on our existing expertise on somatic adult stem cells, immune treatment of MS and non-invasive imaging markers of axonal loss and remyelination. A series of studies using animal models of MS have demonstrated neuroprotection following intravenous delivery of precursor cells. We propose to define an initial cohort of patients and using a cross-over trial design will examine the neuroprotective therapeutic potential of intravenously administered autologous adult human mesenchymal stem cells. Our approach is to identify prospectively cohorts with clinically articulate lesions involving the optic nerve in which detailed protocols can be subsequently evaluated for screening the outcome of a more widely distributed neuroprotective cellular therapy, extrapolating the experience of the sentinel lesion to the disease as a whole. The optic nerve is our favoured target given its clinical eloquence and the ready access to neurophysiological and serial quantitative imaging outcomes. This proposal addresses several areas highlighted by the grant call being a proof of concept study to examine the neuroprotective therapeutic potential of intravenously administered autologous adult human mesenchymal stem cells in MS.