Translational Research Symposium | Translating 25 Years of Therapeutic Discovery to Special Populations Across the Lifespan

Since the approval of levetiracetam in 1999, anti-seizure therapy development has expanded beyond traditional ion channel modulation toward rationally-designed small molecules, neuroinflammatory modulators, gene-directed therapies, and biologically-informed treatment strategies. In parallel, new methodologies such as humanized organoid and cellular models, syndrome-specific animal models, and AI-assisted discovery, are accelerating the pace of innovation. The next generation of epilepsy therapies provide the unprecedented capability for precision management.

Yet translation into clinical practice lags far behind scientific discovery, especially for vulnerable populations. Pediatric patients, patients with genetic and developmental epileptic encephalopathies (DEEs), and older patients face distinct pharmacokinetic, pharmacodynamic, genomic, and safety considerations. In parallel, novel therapeutic targets, humanized organoid and cellular approaches, AI-assisted target discovery, and syndrome-specific translational animal models are accelerating therapeutic development.

This session provides a framework for understanding how next-generation therapies can be applied to vulnerable populations. Speakers integrate mechanism of action, clinical efficacy and safety data, pharmacokinetic and drug-interaction considerations, translational pipelines, practical prescribing strategies, and vignettes of successes and failures. Rather than a medication-by-medication review, this symposium takes a population-based translational perspective. The goal is to equip clinicians, investigators, and trainees with a practical framework for implementing emerging therapies effectively - and safely - across the lifespan.