Stem Cell-Derived Therapy Trial Enrolls First Michigan Patient for Drug-Resistant Epilepsy
Key Insights
Wayne State University enrolled the first Michigan patient in a Phase I/II trial testing stem cell-derived interneuron implantation to reduce seizure frequency.
The trial, "A First-In-Human Study of Inhibitory Interneurons (NRTX-1001)," targets drug-resistant unilateral mesial temporal lobe epilepsy (search) (MTLE (search)).
The six-hour procedure involves robotic and stereotactic techniques to deliver GABA-secreting cells into the hippocampus, aiming for a 75% seizure reduction.
Wayne State University School of Medicine has enrolled the first patient in Michigan in a Phase I/II clinical trial to determine if implanting stem cell-derived gabanergic interneurons into the brain can reduce seizure frequency in patients with epilepsy (search). The patient underwent the procedure on Nov. 6.
Maysaa Basha, M.D., FAES, FANA, Associate Professor and Chair of the Department of Neurology, is the principal investigator for the Wayne State-based "A First-In-Human Study of Inhibitory Interneurons (NRTX-1001) in Drug-Resistant Unilateral Mesial Temporal Lobe Epilepsy (search) (MTLE (search))." Dr. Parthasarathi Chamiraju, a clinical associate professor of Neurosurgery, performed the procedure.
Addressing Drug-Resistant Epilepsy
"Patients who qualify for this study and other types of approved surgeries are considered to have drug-resistant epilepsy (search). Their quality of life is negatively impacted due to associated psychiatric and cognitive impairment," Dr. Basha said. "They also have social factors impacting them because seizures limit their ability to drive and sometimes to continue to work. They can be socially isolated due to recurrent seizures."
The NRTX-1001 Trial Procedure
The six-hour procedure utilizes advanced robotic and stereotactic techniques to create a burr hole in the skull. A probe is then inserted into the hippocampus, the area of the brain identified as the source of the seizures. Guided by magnetic resonance imaging, specialized cells that secrete the inhibitory neurotransmitter gamma-aminobutyric acid (GABA) are deposited at two-centimeter intervals, with approximately 10 cell deposits along the hippocampus.
The primary goal of this phase of the trial is to assess the safety of the procedure. "A secondary outcome will be a change (decrease) in seizure frequency in those patients that receive NRTX-1001 compared to baseline and later on compared to those who receive sham treatment," Dr. Basha added.
Measuring Success and Long-Term Follow-Up
Initial success will be measured by the procedure's safety and subsequent reduction in seizure frequency. Responders are defined as those achieving a 75% reduction in seizures from their baseline. An estimated 40 patients will then take an immunosuppressant drug for one year to prevent cell rejection. The study will assess safety, tolerability, neural cell viability, local inflammation, and effects on epilepsy (search) symptoms for two years post-transplant. Patients will be followed for an additional 13 years, with quarterly phone contact and annual visits.