By Dr. Peter Chin, Group Medical Director, Neuroscience, U.S. Medical Affairs, Genentech
Over the last 25 years, the scientific community has made significant advancements in treating multiple sclerosis (MS), which have helped transform what it means to live with the disease. A deeper understanding of the biologies and course of MS progression — coupled with scientific, clinical, and technology-driven innovations — has brought patients toward better long-term outcomes.
Additionally, this is an unprecedented era of partnerships between patient advocates, academic scientists and institutions, professional societies, and industry, all working together to address MS. For example, on March 28, the MS community will come together to observe Progressive MS Day, a day to recognize those living with the most debilitating forms of the disease and call for more research to advance care and reduce disability.
Today, people with MS have a far better chance of delaying disability, due to clinical advances in diagnosing MS faster and the availability of more medicines. But the question remains — how can understanding of the disease be improved to move from delaying progression to stopping it, or even potentially one day reversing it?
MS is a chronic disease that often strikes in the prime of life, has no cure, and is the leading cause of non-traumatic disability in young adults. The disease can cause a wide variety of symptoms including fatigue; loss of hand, arm, or leg function; vision problems; weakness and numbness; incontinence; and cognitive issues. A recent study revealed the prevalence of MS in the United States is nearly 1 million people,[i] more than twice the previously reported estimate, shedding new light on the degree of medical need.
The majority of people diagnosed with MS will be diagnosed with, or advance to, Progressive MS within their lifetime. People living with Progressive MS experience more problems with walking, more difficulty remaining in the workforce, and require more assistance with everyday activities than people with relapsing forms of MS.[ii]
There is growing recognition in the scientific community that MS is a progressive disease from the start, regardless of how it manifests.[iii] As the understanding of how MS evolves has improved, researchers have learned there’s a heavy component of acute inflammation in the beginning, caused by the immune system attacking the nervous system. This can cause noticeable relapses, or it can cause silent damage and changes in the brain and spinal cord that can only be detected by MRI. Over time, as the disease continues to progress, a person experiences steadily worsening disability.
A better understanding of MS progression and an improved ability to identify its underlying signs — paired with early treatment — could help tackle progression and preserve people’s day-to-day function over the long-term. This also may help healthcare providers and people living with MS make more informed decisions about managing the disease and the community to develop effective new therapies that address unmet medical needs.
For several years, Genentech has contributed to expanding the understanding of MS progression, innovating a new therapy and improving outcomes measures. The company continues to work closely with the MS research community to invest in moving the science forward by developing modern techniques and tools, designing clinical trials with novel endpoints, and including populations that haven’t historically been studied to provide a more comprehensive view of disease progression.
For example, the CONSONANCE trial, which looks at people with progressive forms of MS, employs traditional measures of clinical disability that have been in use for decades, as well as more modern techniques to better understand the disease. ORATORIO-HAND is a Phase III study that, for the first time, uses hand and arm function as a primary outcome measure. The study includes participants who are older or who have more advanced disability – people who never have been studied in a Progressive MS therapeutic clinical trial before.
Additionally, Genentech continues to innovate how MS is measured. The company is using novel clinical endpoints such as Progression Independent of Relapse Activity (PIRA), a newer concept that allows researchers to think about underlying disease progression that happens irrespective of relapse activity, providing a more holistic view of the disease. Another Genentech study is assessing the use of cerebrospinal fluid (CSF) biomarkers, including neurofilament light chain (NfL), which may be used in the future to more rapidly measure new disease activity and how patients respond to different therapies. Genentech also is developing 21st century tools to better monitor progression, including a new mobile app called FLOODLIGHT that may offer people living with MS the ability to regularly track their own symptoms.
This is an exciting time to be working in MS, especially as the community comes together to recognize Progressive MS Day. By improving the understanding of disease progression, Genentech hopes to help people maintain their independence, improve patients’ lives and, one day, potentially find a way to stop or maybe even reverse this disease.
Learn more about Progressive MS Day and how to show your support here.
[i] Wallin MT, Culpepper WJ, Campbell JD, Nelson LM, Langer-Gould A, et al. The prevalence of MS in the United States: a population-based estimate using health claims data. Neurology. 2019;92(10). https://n.neurology.org/content/92/10/e1029. Accessed March 21, 2019.
[ii] Primary Progressive MS (PPMS). National Multiple Sclerosis Society. www.nationalmssociety.org/What-is-MS/Types-of-MS/Primary-progressive-MS. Accessed March 21, 2019.
[iii] Kutzelnigg A, Lassmann H. Pathology of multiple sclerosis and related inflammatory demyelinating diseases. Handb Clin Neurol. 2014:122:15-58.