Beyond Seizure Control: Building the Infrastructure for Disease-Modifying Therapies in Epilepsy
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.
Authors
- Kunal Gupta (ORCID: https://orcid.org/0000-0002-9847-0099)
- Julie M. Ziobro (ORCID: https://orcid.org/0000-0002-7250-7308)
- Jack M. Parent (ORCID: https://orcid.org/0000-0002-2972-1528)
- Anup D. Patel (ORCID: https://orcid.org/0000-0001-9313-1541)
- John Hixson (ORCID: https://orcid.org/0000-0003-3053-6474)
- Erika Takle Axeen (ORCID: https://orcid.org/0000-0001-9913-1972)
- Veronica L. Hood (ORCID: https://orcid.org/0000-0002-1988-4980)
- Scott Demarest (ORCID: https://orcid.org/0000-0002-4544-3724)
- Lacey A. Smith (ORCID: https://orcid.org/0000-0002-4456-0072)
- Joseph E. Sullivan (ORCID: https://orcid.org/0000-0003-2081-8988)
- Sucheta M. Joshi (ORCID: https://orcid.org/0000-0002-1205-6103)
Institutions
- University of Southern California (US)
- Boston Children's Hospital (US)
- Nationwide Children's Hospital (US)
- Children's Hospital of Los Angeles (US)
- Medical College of Wisconsin (US)
- University of Michigan (US)
- Dravet Syndrome Foundation (US)
- University of California System (US)
- Children's Hospital Colorado (US)
- VA Ann Arbor Healthcare System (US)
- University of Virginia (US)
Publication Details
- Journal
- Epiliepsy currents/Epilepsy currents
- Published
- 2026-11-11
- DOI
- https://doi.org/10.1177/15357597261497536
- Primary Topic
- Epilepsy research and treatment
- Type
- article
- Field-Weighted Citation Impact
- 0.00