Researchers discussed the development of a new generation of epilepsy treatments, reviewing 13 experimental treatments presented at this year’s EILAT XVII. Eight of the 13 treatments target developmental and epileptic encephalopathies (DEEs), many of which are tied to specific genetic causes where current medications often don’t work well enough. Several approaches target the underlying biology rather than simply suppressing seizures. Several others use newer approaches, including gene therapy and antisense medicines, which are intended to change how the body’s cells produce particular proteins. Some drugs discussed were completely new, while others are existing medicines being repurposed for epilepsy.
For the genetic therapies and antisense drugs, an especially important unanswered question is whether changing the underlying molecular flaw will produce long-lasting improvements in seizures and cognition/development, rather than merely reducing seizure frequency. The EILAT progress report covered 21 investigational treatments in 2026, compared with 14 in 2024, 12 in 2022, and 13 in 2020. This increase is meaningful not only because there are more drugs, but also because the development strategy itself is changing.
In the past, the paradigm was focused on finding a drug that suppresses neuronal excitability to reduce seizures; however, the emerging paradigm is to identify the molecular cause/mechanism and intervene selectively to potentially improve seizures and other manifestations of the disease.
That is particularly significant for severe DEEs, where conventional antiseizure medications can reduce seizures without adequately addressing developmental, cognitive, behavioral, or other neurological consequences. The hope is that newer, more targeted treatments could do more than simply reduce seizure frequency—they might also improve other problems associated with severe genetic epilepsies.