Ask the Experts Webinar: Disease Modifying Therapies Clinical Guidance-AskTheExperts

Video Transcription

Video Summary

The webinar explored disease-modifying therapies (DMTs) for epilepsy—treatments intended to address underlying causes rather than only control symptoms. Approaches include stem cell-derived inhibitory interneuron transplants, RNA-targeting therapies such as antisense oligonucleotides (ASOs), and DNA-level gene replacement, editing, or regulation. Some are in clinical trials, while others remain in preclinical development. Genetic testing and further research into disease mechanisms will be essential to identify suitable patients.<br /><br />Implementing DMTs requires substantial infrastructure: multidisciplinary clinical teams, institutional regulatory and ethics review, specialized pharmacies and laboratories, trained surgical and infusion staff, long-term safety monitoring, and strong institutional leadership. Delivery methods—including intravenous, intrathecal, and direct brain administration—differ in invasiveness, targeting, risks, and resource requirements. Sustainable reimbursement and adequate coverage will also be critical.<br /><br />The speakers emphasized that access must be addressed early. Clinical trials and specialized epilepsy centers often serve less diverse populations, while language barriers, travel and participation costs, referral patterns, and limited site capacity can delay or prevent treatment. Recommendations include improving trial awareness and translated materials, strengthening referrals and genetic testing, coordinating specialist and local care, and embedding equity considerations into clinical practice, education, research, and advocacy.<br /><br />Discussion highlighted the need to prepare trainees and adult neurologists for DMT administration, follow-up, and patient transitions. While complex therapies may initially be concentrated at experienced centers, partnerships and investment could expand access. The speakers described DMTs as a rapidly approaching opportunity to transform epilepsy care, alongside significant unanswered questions about durability, long-term effects, and equitable delivery.

Keywords

epilepsy

disease-modifying therapies

DMTs

gene therapy

antisense oligonucleotides

stem cell transplantation

genetic testing

clinical trials

health equity

specialized epilepsy centers