The treatment of relapsing multiple sclerosis (MS) remains a rare beacon of success in the long campaign against neurological disease. The ready availability of a phase II clinical outcome (eg, gadolinium-enhancing lesion) that tracks a relevant phase III clinical outcome (eg, annualized relapse rate) is one of the major reasons for this success. Eight therapies have been approved by the US Food and Drug Administration in less than 20 years, and 3 more are under current consideration at the Food and Drug Administration following successful phase III clinical programs. However, before feeling triumphant, we must recognize that our current therapies are principally effective in the prevention of relapses and new brain lesions detected using magnetic resonance imaging (MRI) and that their therapeutic benefit over the long term remains a matter of controversy.1,2 In fact, it is widely agreed that we lack robust, potent therapies to overcome the fearsome long-term progressive neurological decline that characterizes MS. Furthermore, this progressive neurological decline manifests itself in the later stages of the disease and appears irreversible once it appears—facts that make it difficult to adequately time therapeutic intervention and assess its effects. As a result, progressive MS is an arena in desperate need of compelling biomarkers and predictors of disease course. Precisely for this reason, retinal optical coherence tomography (OCT) has garnered great interest as a potential phase II clinical outcome in neuroprotective trials in MS. Nearly 200 papers have been published on the topic in the last 8 years (as of July 2012 in MEDLINE).
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