In light of World MS Day, and in celebration of HRW’s extensive history researching MS through the eyes of patients and healthcare professionals alike, we took the time to check in with a member of our Expert Panel (our selection of friendly, thought-leader HCPs) to conduct a “temperature check” on MS- where are we today, and what does the future hold? Vincent Huart (Senior Research Manager and member of our neuroscience expert team, Synapse) enjoyed a chat with a key opinion leader within MS. Read on below to learn how interferons are being challenged in the light of novel treatments becoming available for MS patients, and how holistic patient care and assessment is increasingly paramount. As a market researcher with a special interest in neuroscience, I have a passion for working and exploring developments in MS and thoroughly enjoyed chatting to an expert physician on the frontline treating and managing patients with this condition. With their level of in-depth experience as an expert in the field I felt that I was in the safe hands to learn and explore more about current and future treatments in the landscape. I have collated a few key learnings from our conversation detailed below. If you are interested in learning more about our experience in MS, please get in touch firstname.lastname@example.org. Context and the history of MS treatments Disease modifying treatments (DMTs) have come a long way since the release of interferon beta -1a/1b treatments in the late 1990s, which work by balancing the level of pro and anti-inflammatory agents in the CNS, reducing neuro inflammation and degeneration. DMTs have since evolved towards more targeted approaches, focusing on proteins expressed on T-cells and B-cells via injectable monoclonal antibodies to reduce activation of these cells and the overall inflammatory response. Oral therapies have also recently been introduced- also targeting T-cell and B-cell release in the peripheral and central nervous system. These exploit the sphingosine 1-phospate pathway, targeting specific protein sub-types expressed on T-cells and B-cells thus modulating their egress from lymphoid tissue. But the question is where do we go from here? We’ll consider some new developments in MS shortly, but first let’s “zoom out”. MS treatment must consider the entire patient and their world- eliciting many challenges in effectively managing and supporting them through their journey. Challenges: Cost, QoL, and patient monitoring and management Although treatments have advanced in MS, cost constraints can cause patients to still be prescribed more cost-effective injectable treatments requiring patients to visit specialist centres for treatment. Mobility and disease impact can be challenging for more advance patients, rendering treatment visits more difficult over time. Additionally, the patient’s QoL isn’t always at the forefront of real world decision making- nor is the impact on their mental health status as a subcomponent of their QoL. There remains huge opportunity for pharma to continually support patients from this more holistic perspective- enabling them to navigate the logistical friction of their care, as well as the psychological burden this represents. This is something which is felt quite strongly by our MS expert in conversation: “We need to avoid unnecessary travelling to the specialised centres in particular for those with disability. At the moment we are only working on MS units, MS services, infusion suits… but we still need a domiciliary approach or training at home and follow up to avoid the patient from coming into hospital” Reliable MS symptom and disease activity monitoring is a challenge in traditional MS patient management as disease activity is often reviewed on an infrequent basis through telephone consultations. New monitoring developments are emerging within the clinical trial setting, targeting neurofilaments as a biomarker. As a neuron-specific component, this has promise in dialling up the accuracy of tracking disease activity. Elsewhere, digitization of patient symptom progression monitoring between patient and physician is also an unmet need within care which could alleviate in person centre visits. Some pharmaceutical companies are already taking the lead in this area, but more needs to be done to help embed digitization to help patient symptom monitoring. “I think we need to review disability progression through devices, mobile phones and digital tools as this will be important. These are currently in development and in clinical trials but we don’t use these in clinical practice.” These challenges underline the need for better access to innovative DMT treatments earlier line, and to continue to facilitate more patient centric and individualise treatment approaches centring on patient’s QoL- and emphasises the importance of patient reported outcomes in this area. This may help translate the impact of traditional endpoints such as reduction in relapses or reduced lesions into real term patient’s quality of life impact. Treatments on the horizon BTK inhibitors Bruton’s tyrosine kinase (BTK), a B-cell targeting agent, has gained much traction over the years in liquid cell tumour therapy. More recently, it has also gained the attention of some pharmaceutical companies who are taking a different stance in DMTs by developing MS treatments to target this receptor protein. This was brought to light by the MS key expert feeling that BTK inhibitors are one of the most exciting treatments on the immediate horizon. “The area to look out for is within BTK inhibitors which will be available in the market in the next 3 years. These treatments will be key especially in the absence of immune re-constitution therapies in the short term. Obviously, infusions and injections are more chronic based treatments. Ultimately, we want to get to a position where treatments are not use as frequently to avoid the patient from continuously being immune suppressed.” These molecules are still in clinical trial development and act by targeting B-cells from being activated in the periphery and crossing the blood brain barrier. This treatment involves a more holistic approach by focusing innate microglia cells which are responsible for maintenance of CNS tissue. BTK is also expressed on these cells showing promising signs that these novel DMT can act directly in cells within the blood brain barrier. This advancement in treatment approach can offer a more “accurate” treatment which may benefit their side effect profile compared to existing DMT treatments. Immune reconstitution therapies Another new area of development is the use of immune re-constitution therapies in MS. These therapies are an emerging concept for the treatment in multiple sclerosis (MS) that are given intermittently and can induce long-term remission of MS that is sustained in treatment-free periods. These treatments act on the early stage of leukocyte development and may ignite a revolutionary step forward in transformation of the treatment landscape. This has the potential to trigger a seismic shift in DMT paradigm, substituting the need for chronic and ongoing DMT therapies to be administered to patient’s long term. “The rise of immune reconstitution therapies such as Alemtuzumab and Cladribine are therapies which you use once or on two occasions and that’s it! No need for further medications. This could help alleviate the burden of monitoring and the burden of pills and infusions in the long term” Although these treatments show promising signs for the future landscape in MS, they all focus on prevention or symptom reduction within progressive MS. A key unmet need in the development of pharmaceutical treatments is to create therapies which focus on re-myelination to help re-establish lost functioning. This can facilitate a dual and comprehensive approach of targeting relapse prevention, neurodegeneration and aid lost mobility functioning to ultimately improve MS patients’ prognosis and overall quality of life. I closed the discussion by asking our key MS expert what if anything can be done to help change and support the public perception on MS: “ I feel we need to focus on access to media, digital blogs, information in journals to help bring MS into the forefront of people minds and help support MS patients where we can.” By Vincent Huart Apply Now!