Measuring what matters for people with MS and NMOSD

This week, as I join my neurology colleagues all over the world assembling in front of our computers for MS Virtual 2020 (joint ACTRIMS/ECTRIMS conference), I’ve been thinking about what I value most about attending medical meetings. What I think it comes down to is the unique blend of diverse minds and experiences, and hearing from colleagues and friends who are collectively working to progress the science of neuroscience to make life-changing advances for complex conditions that have eluded scientists for so long.

This year, I’m particularly interested in research presentations that grow our understanding of the clinical outcomes that matter most to people who live with multiple sclerosis (MS) and neuromyelitis optica spectrum disorder (NMOSD), a rare and lifelong autoimmune disease of the central nervous system. In several ways, MS and NMOSD have striking similarities – both are demyelinating conditions of the central nervous system that present in the clinic with acute episodes of neurological worsening – so much so that up to 30% of people living with NMOSD are misdiagnosed with MS initially.

Importantly, however, MS and NMOSD differ in the impact that episodes have on neurological function and the sequelae they leave behind, so it’s crucial that we understand – preferably from patients themselves – how best to measure the effectiveness of treatment. Recent data suggest that independently of relapses, MS is a progressive disease from the start, regardless of which MS type a person is diagnosed with initially, and that it ultimately results in the accumulation of noticeable (clinically evident) disability. Put simply, disease progression can occur without noticeable symptoms or acute disability worsening. In NMOSD, the opposite is true; relapses are the main determinant of long-term outcome because they are usually severe and – if untreated – carry the potential to cause irreversible, neurological damage, disability or even death.

Clinical trials in neuroscience have always been challenging – often we’re limited both by what a patient can report, and what a neurologist can assess. For MS, symptoms vary from person to person and progression can be underlying and subtle from the start. In NMOSD, key challenges lie in the very rarity of the disease, the severity and lack of reversibility of the relapses, and unfortunately, the early morbidity and mortality in untreated patients. It is very encouraging and a sign of hope for patients that we now have several therapeutic options available for people with MS, and just in the last two years, three new approved medicines for NMOSD. But there is still a need to optimise how they are used, how early, and for which patients.

At Roche we have long prioritised learning from the communities who have first-hand knowledge of the unique challenges of living with a particular neurological condition. As we work to achieve clinical advancements in both MS and NMOSD, we recognise that to be successful a truly individualised approach is critical. That’s the world of personalised healthcare that we need to create to ensure the best health outcomes for all.

This is why our research efforts in MS are focused on slowing progression earlier in the disease course, and in achieving higher levels of suppression for progression; either by optimising the use of existing medicines or by exploring new mechanisms of action, in order to bring clinically meaningful outcomes to more people living with this disease. We are also at the forefront of better understanding how MS impacts different groups of people by ensuring our clinical trials are as representative as possible of the diverse people who develop the condition. We’ve recently initiated a study in collaboration with healthcare professionals, patient advocates and others designed to provide focused disease insights and better tailored care to African-American and Hispanic- and Latinx-American people with relapsing MS.

At the same time, our focus in NMOSD has been on reducing the risk of relapses, any one of which can severely reduce an individual patient’s quality of life; relapses lead to half of patients requiring a wheelchair, and over half becoming functionally blind. Until recently, people living with NMOSD did not have medicines specifically tested and designed to treat this condition, with many people remaining misdiagnosed and untreated. Our recent approval is supported by data from one of the largest clinical trial programmes for this condition, including a broader age range, treatment background and biological diversity. We want to make sure that our medicines are studied in the real-world population of patients who might benefit from them, so that therapeutic decisions can be well informed.

I’m very proud of the great strides we’ve made as a healthcare community in this area, but we’re far from done. Our mission is to push the boundaries of scientific understanding and create a tomorrow where neurological disorders, such as MS and NMOSD, no longer limit human potential.

So, I’ll leave you with a question – as a scientific and clinical community, what more can we be doing to measure what matters most to the patients we serve?