Commencing January 2022, Dr Izanne Roos, University of Melbourne was granted a 3-year Trish Translational Research Project Grant titled, “Personalised therapy to prevent disability in aggressive multiple sclerosis”.
Dr Roos’ diligent, very hard work is enabling excellent progress to be achieved.
In this research project, Dr Roos and her team are aiming to validate statistical models which can predict an individual’s risk of developing aggressive MS at the earliest stages of MS. They will then establish whether early use of the most potent therapies can prevent aggressive disease in those patients at high risk. They aim to achieve this by using data from big MS registries (MSBase, an international registry, and OFSEP, the French National registry), and a cohort of patients with MS who are followed from early after MS onset from Barcelona, Spain. The focus in 2022 was on setting up the necessary collaborations, and obtaining data from all sources, while abiding to the requirements of the European General Data Protection Regulation. All data had been obtained by December 2022, and data mapping and quality control procedures have been applied.
Several key outcomes had been achieved in the first year of the Project:
- Establishment of collaborations with two large MS teams led by eminent leaders in the field of MS and neuroimmunology.
- Full execution of two GDRP compliant agreements and subsequent data transfer.
- Completed data mapping for both Projects 1 and 2.
- Preliminary results from models 1 and 2 of project 1, with resultant further refinement of the included cohorts as agreed by all parties.
- Advancement of the statistical and methodological expertise required for project completion as evidenced by first author publication in Multiple Sclerosis Journal by CIA Roos (Roos et al. Comparative effectiveness in multiple sclerosis: A methodological comparison.)
These outcomes are key to the subsequent analytical steps and have occurred in line with the proposed timelines.
In 2023 Dr Roos and her team are focusing on the completion of project 1, and completion of the first two analyses of project 2. Project 1 involves the validation of prognostic models for the development of aggressive MS. The results of the validation models will be submitted for publication in a peer-reviewed international journal.
Project 2 will focus on optimal treatment approaches for patients at highest risk of aggressive MS. Dr Roos aims to explore this in a series of analyses. Analysis 1 will compare the odds of aggressive MS among high-risk patients treated with high-efficacy vs standard/no treatment, using marginal structural models. It will also compare the risks of relapses and disability worsening. Analysis 2 will use similar methodology to study the interaction of therapy with time from MS onset to start of high-efficacy therapy, with the aim of evaluating the influence of the timing of treatment start. Dr Roos aims to have completed both these analyses by the end of 2023, with planned manuscript submission in early 2024.
If Dr Roos is successful in validating statistical models which can predict an individual’s risk of developing aggressive MS at the earliest stages and then establishing whether early use of the most potent therapies can prevent aggressive disease in those patients at high risk, people living with – or diagnosed with – aggressive MS would benefit enormously. Dr Roos is making great inroads into achieving this.