Imagine living with daily seizures from the age of four. It happens so often, it eventually becomes the norm for you and your family. This is Devon’s story, and it is more common than you may think. Today, more than 3 million people in the U.S. are living with epilepsy, including rare epilepsy syndromes¹ — neurological disorders typically characterized by recurrent, unprovoked seizures.² Devon — now 25 years old — is living with one of these conditions, Lennox-Gastaut syndrome (LGS). Throughout his childhood, he experienced multiple daily seizures. About three years ago, he started a new medication where he now experiences weeks of seizure-free intervals. Devon can now do some of things that many of us take for granted—playing mini golf, doing puzzles, playing cards, or spending time with his family.

For three decades, UCB has been committed to people living with and caring for those with epilepsy – like Devon and his family. Surrounding the patient and caregiver through every step of their care journey from diagnosis to treatment to support, UCB helps them navigate those moments in their lives that matter most - including the moments of vulnerability, fear, and uncertainty. At our core, we have a responsibility to elevate the healthcare ecosystem. Because of this, we have established our legitimacy on the front lines of epilepsy research, development, and treatment innovation. But our past breakthroughs are only a prologue to our future. At UCB, will continue to reimagine how we care for patients, leveraging today's expertise for a better tomorrow. With so much experience behind us and so much potential ahead, we are more invested than ever in profoundly improving the lives of those living with or caring for those with epilepsy or a rare epilepsy syndrome – through our relentless pursuit of a seizure-free life.

In the below interview, Brad Chapman, head of U.S. epilepsy and rare syndromes at UCB, provides insights from his personal experience listening to and partnering with people like Devon and his family, showcasing how their stories are fueling innovations and programs that are making a tangible difference for those affected by rare forms of epilepsy.

Why is patient awareness and education of rare epilepsies so low, and why does this need to change?

Our current healthcare system is simply not built for people living with rare epilepsies. When we think about other conditions, we see a care continuum that goes into effect from the moment of diagnosis. But in the rare epilepsy space, the focus has been much narrower. For instance, people born with a severe group of rare epilepsies called developmental and epileptic encephalopathy (DEE) — including Dravet syndrome (DS) and LGS — which are characterized by seizures as well as encephalopathy, or significant developmental delay or loss of developmental skills, are often not diagnosed until the age of two.³ And even when this is reached and treatment is administered, the system is not built to help facilitate or support the rest of an individual’s holistic care journey. The impact of DEEs also reaches far beyond seizures, with a wide range of non-seizure clinical manifestations, including intellectual disability, motor and movement disorders, and sleep issues.⁴ There must be an urgency to treat not just the seizures but also these non-seizure outcomes that can severely impact overall quality of life. If we're able to get people living with rare epilepsy on the right treatments faster, we can help reduce people’s seizure occurrence and provide appropriate support, resources, and education over the long term.

From your perspective, why is it so important that the patient and caregiver communities are not only considered but also consulted throughout the processes of drug development?

Our approach has always integrated the needs and experiences of people living with serious diseases into our development process — and we consistently engage and partner with advocacy groups to get a better understanding of how people living with epilepsy and rare epilepsy approach diagnosis and treatment, as well as navigate the landscape. We’ve found that while there are many empowered and engaged people living with epilepsy, there are also gaps in resources that help educate about seizures. We’re also focused on partnering with healthcare providers, epileptologists, and the broader neurology community to improve patient communication and create tools to facilitate productive conversations with their doctors that help them better define what they are experiencing and more clearly understand the options that are available to them.

What are some of the challenges faced by people living with a rare form of epilepsy? 

We know the healthcare system is not built for people living with rare epilepsies and their loved ones. As the external ecosystem continues to rapidly evolve with advanced solutions on the horizon, we feel this even more urgently. We have listened to those who care for a loved one with a rare epilepsy. We know there are significant obstacles surrounding the existing standard of care that extend far beyond safety and efficacy. While others may assume that the side effects of traditional epilepsy treatment methods — such as extreme fatigue, heightened emotions, or drifting out of conversations — would pose the biggest challenges, these are not even factors for rare epilepsy families. Instead, people living with rare epilepsies have comorbidities and stressors which often are their most significant challenges. Frustrations about the simplest daily tasks you and I do each and every day without blinking an eye – going to the bathroom independently, sleeping, communicating and even eating. Caregivers also report a historical lack of awareness that they should be receiving specialist epilepsy care (rather than addressing with their general practitioners), as well as challenges navigating the transition from pediatric to adult healthcare settings.⁵ With all these factors in mind, for some, full seizure control is an impossibility. We must find solutions for seizures that won’t stop, not only to preserve the moments that matter, but to protect the minds and lives of those living with rare epilepsies. 

So how does UCB address this wide range of challenges the epilepsy community faces? 

We have a profound responsibility to elevate the healthcare ecosystem where applicable for these individuals. It is a responsibility that extends to the very heart of our purpose – patients and their families. In our collaborations and partnerships across the epilepsy and rare epilepsy community, we’re taking a comprehensive approach informed by patient and caregiver voices to ensure that their journeys are supported from diagnosis through treatment and beyond. We frequently consult our Rare Epilepsy Caregiver Advisory Panel, a group of parents and other caregivers of people living with rare forms of epilepsy, to solicit feedback that informs our approach to discovery, development, and delivery of our solutions at UCB. This includes input on designing future clinical trials, what language resonates with caregivers, and more. We are shifting our development approach, moving away from treatments for broader populations in favor of more targeted, precision-based interventions – which is critical to support those who have historically been difficult to treat. This requires us to consistently push the scientific limits of how we understand disease to better address patient unmet needs. A cure for epilepsy is always on our minds, and we use our experience and scientific expertise to drive toward it by striving beyond symptom management to focus on true disease modification. But it doesn’t stop there. We are committed to helping improve the quality of life for the entire family, which is why we collaborate with the community to provide support through our family of resources including our Canine Assist program and our VIP Sibling program that addresses the impact on siblings who have a brother or sister with a rare epilepsy.

As patient care continues to evolve, so do we – embracing advances in technology to help keep us more connected to the patients and caregivers we serve. As leaders in neurology, we are utilizing technology across our therapeutic areas. We are focused on investing in technologies that enable better detection and earlier prediction of seizures, as well as improving the coordination of care for people living with epilepsy. Recent data shows that people living with epilepsy may report less than half of their seizures to clinicians⁶ – and according to families living with someone with a rare epilepsy, who could experience hundreds of seizures per day⁷, that statistic is even lower. This lack of consistency in reporting can contribute to large gaps in patient care, which deeply affect treatment decisions and patient outcomes. Because of this, medical devices that are worn by patients that collect vital signs, seizure monitoring, and digital biomarkers, are becoming an increasingly important tool to remove subjectivity and ensure that neurologists can treat the right patient with the right medicine at the right time. 

To that end, we’re investing in detection and monitoring devices like those developed by Neurava, Nextsense, Eysz, and Byteflies to ensure people living with epilepsy or a rare epilepsy syndrome are receiving treatment options that elevate and advance the standard of care. In addition, we’re exploring the utility of gene therapy and other agents targeting the underlying mechanisms of epileptogenesis — the process by which a previously normal brain is transformed to an epileptic brain, capable of generating spontaneous seizures. We have also partnered with the Child Neurology Foundation to create resources that raise awareness of potential causes of death among people with epilepsy. This toolkit helps individuals and their families understand preventative measures to decrease the risk of sudden unexpected death in epilepsy (SUDEP), such as having a seizure action plan or a rescue medication. By strategically partnering and investing in meaningful relationships with organizations that have the technology and data capabilities, we can further drive our healthcare transformation in the most impactful way.

How will UCB continue supporting the community of people living with rare epilepsies? 

At UCB, we know our past breakthroughs are only a prologue to our future. We will continue to reimagine the holistic care for those living with epilepsy and rare epilepsy syndromes, leveraging today’s expertise for a better tomorrow. We’ve set ambitious goals, through our research and discovery work. We have one million compounds per drug screening. We have reviewed over 500 protein targets through our AI/digital pathobiology framework. We are currently running more than 250 interventional studies with more than 25,000 people enrolled. All this investment and effort is dedicated to developing an understanding of the biological pathways that will lead to new disease-modifying solutions in the future. To be a responsible leader in epilepsy and rare syndromes, we must continue to show up for the community in both words and actions. And we are strong in our commitment, because genuine innovation requires a bold approach, and we are relentless in our focus on patient care and our pursuit of a cure.  

To learn more about how UCB’s efforts in improving the lives of those living with epilepsy, visit UCBCares.

References:

1. Centers for Disease Control and Prevention. Epilepsy Fast Facts. Accessed September 2023. https://www.cdc.gov/epilepsy/about/fast-facts.htm 
2. Nationwide Children’s. What is Epilepsy? Accessed September 2023. https://www.nationwidechildrens.org/conditions/epilepsy 
3. Lo Barco T, Kuchenbuch M, Garcelon N, et al. Improving early diagnosis of rare diseases using Natural Language Processing in unstructured medical records: an illustration from Dravet syndrome. Orphanet J Rare Dis. 2021;16(309). https://doi.org/10.1186/s13023-021-01936-9
4. Strzelczyk A, Schubert-Bast S. Psychobehavioural and Cognitive Adverse Events of Anti-Seizure Medications for the Treatment of Developmental and Epileptic Encephalopathies. CNS Drugs. 2022 Oct;36(10):1079-1111. doi: 10.1007/s40263-022-00955-9. Epub 2022 Oct 4. PMID: 36194365; PMCID: PMC9531646. 
5. Rajendran S, Iyer A. Epilepsy: addressing the transition from pediatric to adult care. Adolesc Health Med Ther. 2016; 27(7):77-87. doi: 10.2147/AHMT.S79060. PMID: 27390536; PMCID: PMC4930239. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4930239/
6. Burneo JG. The real truth behind seizure count. Epilepsy Curr. 2008;8(4):92-3. doi: 10.1111/j.1535-7511.2008.00251.x. PMID: 18596872; PMCID: PMC2442148. 
7. Epilepsy Foundation. Development and Epileptic Encephalopathy. Access September 2023. https://www.epilepsy.com/what-is-epilepsy/syndromes/developmental-and-epileptic-encephalopathy 

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