Beyond Seizure Control: Building the Infrastructure for Disease-Modifying Therapies in Epilepsy
Year: 2026
Type: article
Abstract: Rapid advances in disease-modifying therapies (DMTs) for epilepsy, including gene-based therapies, antisense oligonucleotides, and stem cell-derived approaches, are reshaping the treatment landscape. As these therapies move through clinical trials and toward regulatory review, they introduce complex clinical, operational, ethical, and equity considerations that extend well beyond traditional antiseizure medication management. Successful integration will require careful attention to patient selection, genetic and biomarker testing, multidisciplinary care models, infrastructure readiness, referral pathways, long-term safety monitoring, informed consent for novel and potentially irreversible interventions, access to clinical trials, and cost and reimbursement challenges. Significant investments in personnel, systems, and facilities may be necessary, raising important questions about implementation across diverse practice settings and the risk of widening disparities in care. Here, we review the evolving therapeutic pipeline and outline key considerations for safe, ethical, and equitable implementation.
Institutions: University of Michigan, University of Virginia, Children's Hospital Colorado, Medical College of Wisconsin, University of California System, University of Southern California, Children's Hospital Los Angeles, Nationwide Children's Hospital, Dravet Syndrome Foundation, Boston Children's Hospital, VA Ann Arbor Healthcare System
Language: English
Subfield: Clinical Neurology
Field: Medicine
Domain: Health Sciences
Open Access status: diamond
OpenAlex ID: W7225814329