UCB is driven by the everyday stories of people living with epilepsy, learning about and understanding those moments in their lives that matter most. Moments both big and small, where we focus on the person, not just their disease. Patients and caregivers have shared with us that, in addition to seizure control, improved quality of life matters to them and their families. These are everyday moments, such as when a mother sees her child succeed, or is able to spend meaningful time with her family; or a father gets some relief from the worries and daily stress of caring for his child living with a rare epilepsy syndrome; or a sibling plays with his sister because she is more alert and experiencing fewer seizures. We saw results like this in a recently published real-world study on our treatment for people living with Dravet syndrome (DS) and their families.1
Dravet syndrome is a rare, devastating, and life-long epileptic encephalopathy that begins in infancy and is marked by frequent treatment-resistant seizures, significant developmental and motor impairments that persist into adulthood, and an increased risk of sudden unexpected death in epilepsy (SUDEP).2,3 The severe and devastating effects of DS underscore just how important these moments are for people living with rare epilepsy syndromes, and their families.
UCB’s heritage in epilepsy treatment challenges us to do more for people living with epilepsy, to go deeper, by developing a global epilepsy program that focuses on designing meaningful, patient-centric treatment options, because for us, it’s personal.
Recently, we’ve made two changes to UCB’s organization to enable us to better help patients. We acquired Zogenix earlier this year, bringing a treatment for DS and Lennox-Gastaut syndrome (LGS) into UCB’s portfolio. We have also unified the epilepsy organization under one global umbrella that spans research to development to commercialization. Within this new structure, and as I take on the role of the Global Head of Epilepsy and Rare Syndromes, we have established three goals:
- Identifying disease pathologies
- Amplifying a more targeted approach to treatment
- Leading in patient care and outcomes
Identifying Disease Pathologies
Since identifying the synaptic vesicle protein 2A (SV2A) as a novel target for treating seizures, UCB has learned a great deal about epilepsy, new ways to treat it, and the implication of new treatments. We focus on disease pathways, not just the symptoms, moving from seizure suppression into targeted pathobiology. This allows us to identify molecular pathways, whatever the etiology, and target the root cause of the disease for specific patient groups.
Our goal is to understand and break down the cause of epilepsy so we can better understand how a targeted treatment could be used. For instance, DS and LGS are two serious developmental epileptic encephalopathies (DEE), defined by drug-resistant seizures and encephalopathy, which is manifested with cognitive and developmental delay. Both epilepsy syndromes have pediatric onset and are associated with significant morbidity and mortality. DS is a more homogeneous syndrome, in that many of the patients have a common genetic mutation of the SCN1A gene.2 Over the last several years, DS has become more recognized through clinical diagnosis and genetic testing; patients are being diagnosed earlier. In contrast, LGS is recognized as being more heterogeneous, with multiple causalities and lack of consensus on diagnostic criteria. The classic triad for diagnosis is refractory epilepsy, developmental delay, and classic EEG findings, but this triad is often still not recognized in all affected patients.4 Because of this, an appropriate diagnosis and efforts to provide more precise and innovative therapies for patients is so important.
To further deepen our discovery within these pathologies and understand what types of treatments are needed, it is crucial to partner with advocacy groups, patients, and caregivers, who are doing research into the types of epilepsy or rare syndrome that affect them. For example, the LGS Foundation just received a grant from the Patient-Centered Outcomes Research Institute (PCORI) to conduct a study that will help parents and providers make more informed choices about treatment for children with LGS.5 We plan to use their insights to identify areas of opportunity and provide the best possible health care for people living with LGS and their families.
A More Targeted Approach
We have been partnering with patients and caregivers to learn, and have been honing our approach to become more targeted in identifying epilepsy subpopulations – with the goal of developing treatments that bring meaningful change to people’s everyday lives.
We believe that early intervention is imperative for patients with epilepsy, and have seen the benefits of patients receiving the right medicine at the right time. In fact, we’ve heard that doctors prescribing the newest treatment innovations as first-line therapy at disease onset may lead to less drug-resistant epilepsy in the future.6
UCB wants to make a difference in the treatment of epilepsy, and specifically in rare syndrome subpopulations, like DS and LGS. Because these populations have a higher degree of serious neurodevelopmental, cognitive, and motor-function impairment, early treatment may help prevent brain damage and cognition challenges in patients.7 Additionally, SUDEP is a major concern for people living with rare epilepsy syndromes;8 by creating more targeted approaches for rare epileptic encephalopathies, we hope to mitigate some of these risks.
Identifying disease pathologies opens the way to the new generation of therapies that are coming. For example, we are developing a rescue medication with the potential for rapid epileptic seizure termination, with the hope of preventing a more serious clinical event. We are also diving deeper into developing and testing new antibody treatments and gene therapies for patients.
Leading in Patient Care and Outcomes
Patient outcomes are at the heart of everything we do. Results like the ones found in the real-world caregiver study show that UCB is invested in protecting and preserving the moments that matter for patients; from reducing daily stress levels to improving sleep,1 UCB cares deeply for patients and their families. We are also invested in partnerships with companies to help identify seizure forecasting through digital innovation to help make each day more predictable for patients living with epilepsy.
Recognizing and addressing gaps in care is another focus area for UCB, in order to identify treatment trends and the importance of developing tools and resources. For instance, seizure action plans and rescue medications are important for patients to know about and access for a seizure emergency, in addition to their daily epilepsy therapy. We are also investing in research in adults living with LGS and DS to understand their experience and mitigate gaps in care. Our hope is to reach patients not only by treating their condition, but also through the creation of helpful and relevant resources.
UCB also has a great focus on social determinants of health (SDoH) and the connection to health equity. In the first published book on SDoH in epilepsy, The Role of Social Determinants of Health in Epilepsy, UCB’s work to better understand how SDoH affect epilepsy patients living in Arizona is featured as a case study. We are also addressing barriers in the Hispanic community, which is one of the largest subpopulations of people living with epilepsy. We have created bilingual and culturally competent materials for all of our treatment options in order to equip Hispanic patients living with epilepsy and their families with information they need to feel prepared and empowered during medical appointments. Finally, we have just begun to engage in research related to health disparities for Black Americans living with epilepsy, and have been exploring research on women of child-bearing age, so that anyone living with the condition can feel supported in their everyday moments.
UCB’s global efforts are focused on improving the day-to-day patient experience. Our proximity to patients and legitimacy within neurology will allow us to partner better with HCPs on research - with the ultimate goal to modify the underlying causes of disease - and with patients, as we test and learn, so that they can enjoy those moments that matter most.
- Jensen MP, Gammaitoni AR, Galer BS, Salem R, Wilkie D, Amtmann D. Treatment for Dravet Syndrome: Real-world benefits on quality of life from the caregiver perspective. Epilepsy Research. https://www.sciencedirect.com/science/article/abs/pii/S0920121122001279. Published July 7, 2022. Accessed September 2022.
- Dravet C. The Core Dravet Syndrome Phenotype. Epilepsia. 2011 Apr;52 Suppl 2:3-9.
- Dravet C. Dravet syndrome history. Dev Med Child Neurol. 2011 Apr;53 Suppl 2:1-6.
- Asadi-Pooya, A. Lennox-Gastaut syndrome: a comprehensive review. Neurol Sci. 2018. 39:403–414
- Comparative effectiveness of palliative surgery versus additional anti-seizure medications for Lennox-Gastaut syndrome. PCORI. https://www.pcori.org/research-results/2021/comparative-effectiveness-palliative-surgery-versus-additional-anti-seizure-medications-lennox-gastaut-syndrome. Published August 31, 2022. Accessed September 2022.
- Kotulska K, Kwiatkowski DJ, Curatolo P, Weschke B, Riney K, et al. Prevention of Epilepsy in Infants with Tuberous Sclerosis Complex in the EPISTOP Trial. Ann Neurol. 2021 Feb;89(2):304-314.
- Arzimanoglou A, French J, Blume WT, et al. Lennox-Gastaut syndrome: a consensus approach on diagnosis, assessment, management, and trial methodology. Lancet Neurol. 2009;8(1):82-93.
- Harden C, Tomson T, et al. Practice guideline summary: Sudden unexpected death in epilepsy incidence rates and risk factors: Report of the Guideline Development, Dissemination, and Implementation Subcommittee of the American Academy of Neurology and the American Epilepsy Society. Neurology. 2017 Apr 25;88(17):1674-1680.
Content of this article intended for a U.S. audience.
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