The Lennox-Gastaut Syndrome (LGS) Foundation has announced plans for nationwide walks to raise funds for LGS research in the US. By the next decade, LGS hopes to move at least two disease-modifying therapies into trials, but first, it must clinch several low-hanging fruits.
LGS Foundation launches Walk for LGS research
Amid research gaps and a glut in funding, LGS Foundation is organizing walks across the US to “change the landscape of LGS forever.” Scheduled in April, the event will bring together clinicians, researchers, and families affected by LGS under the shared mission.
LGS, a rare and severe form of epilepsy, currently affects over one million individuals globally. Despite years of effort, progress has stalled with LGS Foundation citing fragmented care systems and delayed diagnosis as key concerns.
Apart from raising money with the walks, the 18-year-old foundation hopes participating families will enroll in Phase 2 of the Learn from Every Patient Database, the organization’s outcomes registry and natural history program. Charity walks have surged in recent years, with the events raising funds for research and awareness.
“Walk participants can also connect with a team member for assistance with enrollment,” said LGS Foundation COO Amber Mathas. “We see the Walk not just as a fundraiser but as a recruitment and engagement moment for the registry itself.”
Funds realized from the nationwide walk will support the LGS Foundation’s Powering Breakthroughs initiative. Aware of the steep challenges, the foundation outlined three grand challenges under the initiative.
The first, dubbed Precision Diagnosis, will aim to reduce the time between the first seizure and an accurate LGS diagnosis from years to months. Furthermore, there are plans to roll out at least two disease-reversing or disease-modifying therapies into clinical trials in the coming years.
Prioritizing biomarker development
The third grand challenge is the lowest-hanging fruit for the LGS Foundation. As part of the challenge, the foundation intends to address inequities in access by expanding recognized LGS clinics and promoting its unified outcomes registry.
However, biomarker development is at the top of the pyramid with its Medical Science Advisory Council and research partners terming it “the single most urgent funding priority.” Mathas told Charity Journal that the reason is that “validated biomarkers are the prerequisite” for a disease-modifying therapy trial.
Zooming in, the team is prioritizing study on biomarkers that can define measurable endpoints for clinical trials, particularly EEG-based signatures like generalized paroxysmal fast activity and slow spike-wave patterns. Furthermore, biomarkers that can identify pre-LGS states will receive priority attention.
“The goal of moving at least two disease-modifying therapies into trials this decade is ambitious but achievable,” said Mathas. “It depends on establishing these shared, validated endpoints that industry and academic partners can build on together.”