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Historic Breakthrough in Epilepsy Treatment at RWJUH! 

| By | News

A major milestone in neurological innovation has just been achieved. Dr. Robert Gross, Study Principal Investigator, has successfully operated on the first participant to receive the investigational gene therapy AMT-260, delivered directly into the brain using a highly advanced MRI-guided procedure.
This landmark procedure is part of a first-in-human (FIH) clinical trial exploring a revolutionary approach to treating adults with unilateral refractory mesial temporal lobe epilepsy (MTLE)—a condition that has remained difficult to treat with conventional therapies. Using MRI-guided convection-enhanced delivery (CED), this cutting-edge technique enables precise, targeted delivery of gene therapy directly to affected brain regions.

✨ A First for RWJUH
This achievement marks the first patient at RWJUH to receive brain-directed gene therapy, a proud and defining moment that highlights the institution’s leadership in advancing next-generation neurological treatments and bringing new hope to patients.

🧠 About the Study
This multi-center, Phase 1/2a, FIH study is designed to evaluate the safety, tolerability, and efficacy of AMT-260 in adults with MTLE.

Participants in the study will:

  • Receive a one-time administration of AMT-260 at one of two dose levels
  • Benefit from a study design with no placebo, ensuring all eligible participants receive the investigational therapy
  • Avoid any need for removal or destruction of brain tissue
  • Retain the option to pursue other treatments in the future
Throughout the study, participants will be closely monitored through site visits, phone check-ins, blood tests, and questionnaires assessing how seizures impact their daily lives. They will also track seizure activity using an electronic diary.

🌟 Why This Matters
This breakthrough represents a bold step forward in the fight against epilepsy—combining precision medicine, innovative delivery technology, and gene therapy into a single, potentially transformative treatment. It’s not just a milestone for RWJUH, but a glimpse into the future of care for patients living with epilepsy.