As a person living with secondary progressive multiple sclerosis (TLG) and leaders in the multiple sclerosis patient advocacy community (TC and PZ), we are familiar with the hopes and frustrations of people living with progressive multiple sclerosis. All too often they express justified frustration at the paucity of treatments available. For example, a person living with progressive multiple sclerosis in the USA noted in a blog post, “Hearing I have a form of multiple sclerosis that is chronically progressive and has no therapies felt like being told to ‘go home and make the best of it’ because they had nothing to offer me”.
A major concern is the effect that progressive multiple sclerosis has on the quality of life of patients and their families and carers. Investigators of a recent study in Italy reported that people with progressive multiple sclerosis expressed fears associated with increasing disability and identified strong needs for qualified personnel to provide care coordination and psychosocial support. Individuals also expressed a need for supportive social networks and an ability to preserve their roles in their families and the community.
Despite the absence of disease-modifying treatments and the challenges of living with progressive multiple sclerosis, there is cause for patients, families, and clinicians to be optimistic. The research community is developing a deeper understanding of the pathophysiology of the disease and is taking steps to address the challenges of doing clinical trials of treatments for patients with progressive forms of multiple sclerosis.
Sustaining and further propelling this new momentum demands the concerted effort of everyone interested in finding safe and effective disease-modifying treatments for people with progressive multiple sclerosis. In our estimation, increased focus is needed in three areas: research collaboration, regulatory innovation, and funding.
We regard continued and expanded collaboration by the research community to be essential in solving the biological and clinical challenges associated with the treatment of progressive multiple sclerosis. Although research funders are able to facilitate some collaboration through funding announcements, the research community must not only continue to embrace existing conventions, but also create new models of research collaboration. Our collective experience is that patients expect scientists to collaborate to overcome barriers that have hampered the discovery of treatments for progressive multiple sclerosis.
Innovations by regulatory authorities are crucial in ensuring that new treatments are developed and approved in a timely way. Although regulatory authorities have substantial experience with the review and approval of treatments for RRMS, the same cannot be said for the progressive forms of the disease. Clinical trials of treatments for RRMS are already time-consuming and expensive. We are concerned that regulatory expectations of treatment trials for progressive multiple sclerosis could become an onerous bottleneck to progress. Innovation and adaptation by regulators will probably be necessary to overcome this issue. Such innovations could take several forms, including consideration of new outcome measures, adaptive clinical trial designs, and consideration of surrogate markers. Moreover, enhanced engagement of patients with progressive multiple sclerosis by regulatory authorities could ensure that their views are fully incorporated into the regulatory processes.
Finally, sustained and increased funding for research into progressive multiple sclerosis by patient organisations, foundations, governments, and industry will be necessary to achieve the same success that we have seen for RRMS. The launch of the International Progressive MS Alliance bodes well for the global commitment by patient organisations to this effort, but more is needed. We argue that sustained funding has led to the array of treatment options available for RRMS, and that it is reasonable to demand the same investment and step change in treatment for the more than 1 million people living with progressive multiple sclerosis worldwide.
An urgent need exists to solve the challenges of progressive multiple sclerosis. Only the collective engagement of people living with the disease, researchers, patient advocates, medical professionals, funders, governments, and industry can address the collective frustration felt by all stakeholders and offer the confidence that we will soon see a transformation in the treatment and care of people with progressive multiple sclerosis.