Building the Infrastructure for Disease Modifying Therapies in Epilepsy Clinical Guidance Package
Purpose

Epilepsy care is entering a new era. For decades, treatment has focused on controlling seizures, but advances in precision medicine are opening the door to therapies that target the underlying causes of disease. This program grew out of the work of the AES Presidential Task Force and reflects an important in conversation the field of epilepsy: as disease modifying therapies move closer to clinical practice, we must consider not only the emerging science, but also how these therapies can be implemented safely, ethically, and equitably.

The program includes one live Ask the Expert webinar and three recorded webinars. In the live session, a panel of experts reviews the evolving pipeline of disease modifying therapies and discusses what it will take to deliver them responsibly, including clinical readiness, infrastructure and workforce needs, informed consent, long-term follow-up, and equitable access for patients in both academic and community settings. The three recorded webinars each take a closer look at a major therapeutic approach: antisense oligonucleotides (ASOs), gene therapy, and stem cell derived therapies. Together, they cover how each approach works, where it stands in development, and what ongoing research means for patients and clinicians.

These sessions give clinicians, researchers, and other members of the epilepsy community a grounded understanding of where disease modifying therapies stand today and how the field can prepare for their arrival in practice.

Disease Modifying Therapies Task Force

  • Erika Axeen, MD
  • Scott Demarest, MD
  • Kunal Gupta, MD, PhD, FAANS
  • John Hixson, MD
  • Sucheta Joshi, MD, MS, FCNS, FAES  
  • Jack Parent, MD
  • Anup Patel, MD, FAAN, FAES, FCNS
  • Veronica Robbins-Hood, PhD
  • Lacey Smith, MS, CGC 
  • Joseph Sullivan, MD
  • Julie Ziobro, MD, PhD
Course Components
Ask the Experts On Demand: Disease Modifying Therapies Clinical Guidance
Description: This webinar will bring together experts to review the evolving therapeutic pipeline of disease modifying therapies for epilepsy and outline key considerations for safe, ethical, and equitable implementation. The session will address clinical readiness, infrastructure and workforce needs, equitable access to advanced therapies, consent and long-term follow-up, and strategies to support both academic and community practices.

Learning Objectives
  • Assess the current and future landscape of disease modifying therapies for epilepsy.
  • Recognize necessary infrastructure to implement and provide follow-up for these therapies.
  • Examine ways to promote equitable access to advanced therapies for appropriate patients.
Faculty
Kunal Gupta, MD, PhD, FAANS 
Sucheta Joshi, MD, MS, FCNS, FAES 
Julie Ziobro, MD, PhD 

Antisense Oligonucleotides (ASO)

Description: Dr Anup Patel of Nationwide Children's Hospital discusses what ASOs are and their use in epilepsy management. ASOs represent a major shift from managing symptoms to disease modifying, precision medicine. 


Learning Objectives

  • Define antisense oligonucleotide (ASO) therapy.
  • Understand use of ASOs in epilepsy.
Faculty
Anup D. Patel, MD, FAAN, FAES, FCNS
 
Targeting the Root Cause: The Current Clinical Landscape of Epilepsy Gene Therapy
Description: For many people living with genetic epilepsies, current treatments manage seizures without addressing the underlying cause. Gene therapy offers the possibility of changing that by targeting the genetic root of disease directly. In this webinar, Dr Joseph Sullivan of Encoded Therapeutics gives a broad overview of how gene therapy works. He briefly reviews the preclinical models used to develop and test these therapies, and discusses which genetic targets are furthest along in the development pipeline. Dr Sullivan then takes a closer look at an ongoing clinical trial of gene therapy for Dravet syndrome.

Faculty

Joseph Sullivan, MD, FAES

Learning Objectives
  • Understand the fundamentals of gene therapy and its potential to address the underlying causes of genetic epilepsies.
  • Describe the current landscape of gene therapy research in epilepsy, including where the field stands today.
  • Discuss how clinical trials, such as the ongoing study in Dravet syndrome, are shaping the future of precision treatment for epilepsy.

Stem Cell Derived Therapies
- Coming December 2026

Faculty
John Hixson, MD  
CONTACT INFORMATION
For questions regarding this activity, please contact the American Epilepsy Society Education Department at education@aesnet.org or call (312) 883 3800. 
Summary
Availability:
On-Demand
Cost:
FREE
Credit Offered:
No Credit Offered
Contains:
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