Purpose of review: We summarize MRI measures currently available to assess treatment efficacy and safety in multiple sclerosis (MS) clinical trials and discuss novel metrics that could enter the clinical arena in the near future.
Recent findings: In relapsing remitting MS, MRI measures of disease activity (new T2 and gadolinium-enhancing lesions) provide a good surrogacy of treatment effect on relapse rate and disability progression; however, their value in progressive MS remains elusive. For the progressive disease forms, these measures need to be combined with quantities assessing the extent of irreversible tissue loss, which have already been introduced in some clinical trials (e.g., evolution of active lesions into permanent black holes and brain atrophy). Novel measures (e.g., quantification of gray matter and spinal cord atrophy) have demonstrated a great value in explaining patients' clinical outcome, but still need to be fully validated. Despite showing promise, evaluations of cortical lesions, of microscopic tissue abnormalities, and of functional cortical reorganization are still some way off for monitoring of treatment effects.
Summary: Trial outcomes in MS should include measures of inflammation and neurodegeneration, which should be combined according to the disease clinical phenotype, phase of the study, and the supposed mechanism of action of the drug tested.