Rapidly evolving multiple sclerosis: opening the window of therapeutic opportunity

Multiple Sclerosis (MS) is a demyelinating disease of the nervous system that represents the most common cause of disability in young adults. It is a long-term disease with a significant medical, economic and social impact, largely due to the accumulation of disability. Results of studies and clinical practice show that patients affected by rapidly evolving form of MS may not benefit from the available immunomodulatory drugs and are at high risk to become disabled. We want to establish a group (?cohort?) of patients with very active forms of MS that has not been controlled by the standard treatments. With ethical approval and the consent of each patient who wishes to enter this cohor, we plan to study in great detail the characteristics of disease in each subject by clinical tests, brain scans, blood and spinal fluid tests for indices of inflammation and by mapping some variations of genes that are associated with MS. With this information we will be able to identify within the group the most suitable patients who could benefit from new therapies being studied. The patients participating in the cohort will have the option to participate in the clinical trial most suitable to them. By accelerating the access of candidate patients, the clinical trials will be run more efficiently, speeding up the development of effective cures for MS.

Multiple sclerosis (MS) is a heterogeneous inflammatory and degenerative disease of the central nervous system that presents with a variable clinical course and a broad spectrum of severity. This heterogeneity is likely to reflect at least in part the involvement of different components to the pathogenesis of the disease, as suggested by studies of MS lesion pathology. Clinical and pathological heterogeneity in patient populations represents a difficulty in the evaluation of responses to MS therapies. However, progress in the characterisation of the components of MS pathogenesis and the increasing availability of biological agents targeting specific cell populations or functions now offer great opportunity to develop more individualised and effective therapies.

By establishing a research cohort of patients with rapidly evolving MS in a relatively early stage of disease we intend to maximise the opportunity to develop effective therapies. Our group has expertise in state-of-the-art methodologies for clinical, immunological, genetic and genome-wide transcriptional characterisation of MS phenotypes. Systematic collection of phenotypic data within the research cohort will allow delineating homogenous subgroups of patients who are appropriate candidates to clinical trials of new therapeutics. By developing search algorithms for existing or newly designed academic- or industry-driven clinical trials we will be able to rapidly and objectively identify patient subgroups with specific disease characteristics.

The products that our research cohort programme can deliver are (i) providing to patients with rapidly evolving MS the option to participate in trials of novel therapeutics exploiting the appropriate window of therapeutic opportunity, and (ii) to drive development of novel pharmacological or cell therapies through an innovative model to efficiently recruit specific patient populations.