Did you know groundbreaking epilepsy research is transforming patient care? UCB just presented 26 abstracts at a major international congress, offering new hope for those with Dravet and Lennox-Gastaut syndromes. What could these advancements mean for the future of treatment?
UCB, a leading global biopharmaceutical company, recently presented an impressive 26 scientific abstracts at the International Epilepsy Congress (IEC) in Lisbon, Portugal, highlighting its unwavering dedication to advancing epilepsy research and clinical care. These presentations unveiled a diverse portfolio of studies, focusing on critical areas within epilepsies, including developmental and epileptic encephalopathies (DEEs), prolonged seizures, and emergent treatment pathways. The breadth of data underscores UCB’s commitment to addressing the complex and often unmet needs of individuals living with severe epileptic conditions, striving to foster a better future through innovative solutions.
Among the significant findings was an open-label extension study detailing the long-term safety and sustained benefits of FINTEPLA® (fenfluramine) for children and adults afflicted with Dravet syndrome or Lennox-Gastaut syndrome. This crucial data, derived from a cohort of 412 patients, reported no new or unexpected safety signals, reinforcing fenfluramine’s role in managing these severe forms of epilepsy. The study’s insights into global functioning offer valuable perspectives on improving the quality of life for patients and their caregivers.
Further research presented at the congress shed light on the persistent diagnostic gaps for DEEs in adult care settings, contrasting with the more routine diagnoses in pediatric populations. A qualitative study, based on interviews with caregivers and healthcare professionals across Europe, revealed that a confirmed diagnosis significantly enhances holistic care. This can lead to improved quality of life (QoL), better communication between patients, caregivers, and medical teams, and a reduced risk of hospitalization for adults with these challenging conditions.
The profound daily impacts of unpredictable seizures and disruptive behaviors on caregivers of individuals with DEEs were explored through an extensive internet-based survey. Interim results from this vital study indicated that nearly half of caregivers reported high rates of disruptive seizures and behaviors, which frequently led to a temporary loss of abilities for the patients. These findings underscore the urgent need for better support and interventions to mitigate the substantial burden experienced by families managing these complex neurological disorders.
Several presentations focused intently on prolonged seizures, their definitions, prevalence, and devastating real-world impact on patients and caregivers. Research highlighted significant unmet needs and the profound short-term and long-term negative effects on quality of life. Data from Adelphi’s Prolonged Seizure Disease Specific Programme™ characterized patient populations, revealing that individuals often experience considerable seizure worry and that prolonged seizures frequently escalate to status epilepticus or seizure clusters, necessitating emergency care despite existing best practices.
Innovative analyses of video-EEG recordings were presented, proposing statistical cutoffs for defining possible and probable prolonged seizures across different seizure types. These studies supported the established 2-minute cutoff for tonic-clonic seizures and suggested refined 1 to 3-minute cutoffs for other seizure types, indicating that many seizures become abnormally prolonged at two minutes or less. Additionally, a qualitative study examined seizure emergency pathways, advocating for stronger outpatient guidelines to empower caregivers and patients in managing acute seizure events, potentially averting serious complications.
Recognizing the diagnostic complexities of Lennox-Gastaut syndrome due to its heterogeneous clinical presentation, a group of epilepsy experts collaborated to develop a user-friendly checklist for non-specialists. This tool, framed by International League Against Epilepsy criteria, aims to improve LGS diagnosis. Complementing these findings, UCB sponsored a symposium titled “Time matters in developmental and epileptic encephalopathies,” which sought to broaden understanding of DEEs’ impact in adulthood, emphasizing improved diagnosis, treatment journeys, and addressing barriers to care. The overarching goal of these clinical advancements is to drive better individual outcomes for patients.
Beyond current treatments, UCB also provided glimpses into its early pipeline research, showcasing promising avenues for future therapies. Presentations included studies on AAV gene therapy in mouse models of STXBP1 haploinsufficiency and rescue of neuronal activity in iPSC-derived STXBP1 in vitro disease models. This cutting-edge exploratory work exemplifies UCB’s forward-looking approach to discovering novel solutions for devastating neurological conditions, ensuring a continuous stream of potential breakthroughs in the field.
Ultimately, the collective body of work presented by UCB Pharmaceuticals at the IEC reinforces its global mission to transform the lives of people living with severe diseases. By deepening insights into disease mechanisms, optimizing treatment outcomes, and meticulously considering the experiences of both patients and caregivers, UCB remains at the forefront of neurological innovation. This comprehensive approach ensures that the company is not only developing new medicines but also shaping a more supportive and informed future for those affected by epilepsy.
