Hematopoietic Stem Cell Therapy for Inflammatory MS
Hematopoietic Stem Cell Therapy for Patients with Inflammatory Multiple Sclerosis Failing Alternate Approved Therapy: A Randomized Study
Multiple sclerosis (MS) is, at onset, an immune-mediated demyelinating disease. In most cases, it starts as a relapsing-remitting disease with distinct relapse from which patients may recover partially or completely with no progression between one relapse and another. Over years or decades, most patients transition into a progressive disease in which at least insidious and slow neurologic deterioration occurs with or without acute relapses. Relapsing-remitting disease is often responsive to immune suppressive or modulating therapies, while immune based therapies are generally ineffective in patients with a progressive course. This clinical course, response to immune suppression, as well as evidence form a number of neuropathology and neuroimaging studies, suggest that disease progression is associated with neuro-degeneration and axonal loss. Disability correlates better with measures of axonal atrophy than immune mediated demyelination. Therefore, immune based therapies, in order to be effective, need to be started early in the disease course whilst the illness is predominately an immune-mediated and inflammatory disease. While current immune based therapies delay disability, no intervention has been proven to prevent progressive disability. We propose, as a randomized study, autologous unmanipulated Peripheral Blood Stem Cell Transplant (PBSCT) using a conditioning regimen of cyclophosphamide and rATG versus approved standard of care (i.e. interferon, Copaxone®, Tysabri®, etc) in patients with inflammatory relapsing MS who have failed to respond to an approved fist line therapy.
East Midlands - Leicester Central Research Ethics Committee
Date of REC Opinion
18 Sep 2013