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New Findings from LGS-CORE Registry Highlight Challenges in Seizure Treatment for Lennox-Gastaut Syndrome

The Lennox-Gastaut Syndrome (LGS) Foundation is set to unveil significant preliminary findings from its LGS Collaborative Outcomes Registry (LGS-CORE) during the upcoming NORD Rare Diseases Orphan Products Breakthrough Summit. This data aims to shed light on the efficacy of current treatment options for Lennox-Gastaut Syndrome, a complex and severe form of epilepsy that predominantly affects children, evidenced by the alarming statistic that only 4% of reported treatments have been effective in stopping all seizures.

Background on Lennox-Gastaut Syndrome

Lennox-Gastaut Syndrome is characterized by multiple seizure types that are often resistant to medical treatment. It typically manifests in childhood and poses persistent challenges, not just medically but also in educational and social aspects, with roughly 50,000 patients in the United States and over 1 million globally currently grappling with its effects. Moreover, most individuals with LGS suffer from moderate to severe intellectual disabilities, and currently, there are no disease-modifying therapies or cures available.

Insights from the Early Registry Data

Initial data collection for LGS-CORE began in February 2026 and has since enrolled around 100 families, seeking to reach a target of 1,000. The registry emphasizes real-world experiences and outcomes of patients through longitudinal surveys completed by caregivers, who provide insights into the effectiveness and side effects of treatments administered. Preliminary results from 104 different medications indicate that:

• - Only 4% of the treatments fully stopped seizures.
• - 21% achieved some reduction in all seizure types, whereas 47% were reported as having diminished only some seizure frequency.
• - 22% of respondents reported uncertainty regarding the efficacy of the medications used.

These results point to a grim reality for caregivers and patients alike, as they navigate a complex landscape of treatment options without clear indications of what works.

Treatment Patterns and Their Implications

The data show a concerning trend toward heavy polypharmacy, with 40% of patients currently on at least four anti-seizure medications. The most frequently prescribed drugs include cannabidiol, clobazam, valproic acid, and clonazepam. This raises essential questions about possible interactions between medications and the challenge of identifying which drugs contribute to clinical improvements versus side effects.

In terms of seizure frequency, the data highlight a pressing issue: most patients experience daily or weekly seizures. Additionally, 75% of the patients had seizures lasting over five minutes, with 27% requiring emergency medical intervention due to seizure-related incidents. A troubling statistic indicates that for 64% of patients, the underlying cause of their seizures remains unidentified, further complicating treatment efforts. Surgical interventions are not uncommon, with 61% of participants having undergone surgery, primarily for vagus nerve stimulation, a procedure designed to help reduce seizure frequency.

Addressing the Evidence Gap

Helen Chen, PhD, the Research Director at the LGS Foundation, underscores the critical need for more effective treatment strategies. According to her, families participating in the registry express the difficulty in assessing the effectiveness of multiple medications, leading to a significant evidence gap that requires attention. The insights shared by caregivers indicate a dire need for clarity on treatment efficacy, which is crucial for better-targeted therapies.

Future Directions for LGS-CORE

Looking ahead, the LGS-CORE registry plans to expand its sample size to meet the desired goal of enrolling 1,000 families. Continued data collection will allow for richer insights into treatment efficacy and help identify more optimal prescribing patterns. The registry aims to share data with researchers and trial sponsors interested in advancing studies related to LGS.

In conclusion, the initial findings from the LGS-CORE registry paint a sobering picture of the current state of Lennox-Gastaut Syndrome treatment. As the LGS Foundation presents these findings at the forthcoming summit, they hope to initiate further discourse on improving the lives of those impacted by this challenging condition.