Mayo Clinic has embarked on a groundbreaking clinical investigation exploring regenerative therapy for patients with drug-resistant epilepsy. The multicenter study, launching in January 2025, marks the first human trial of its kind, testing the safety of implanting specialized neural cells called interneurons into the hippocampus of affected patients.
The innovative treatment approach targets a fundamental mechanism of epilepsy - the loss of inhibitory interneurons in the hippocampus following repeated seizures. These specialized cells are derived from human embryonic stem cells through a process that prevents reversion to their stem cell state, offering a potential breakthrough in epilepsy treatment.
"This work has the potential to rewrite the care pathway for epilepsy," explains Dr. Jonathon J. Parker, neurosurgeon at Mayo Clinic in Phoenix/Scottsdale and co-principal investigator. "We hope that replacing these inhibitory cells can restore the normal excitatory balance and reduce or even eliminate seizures."
Novel Therapeutic Approach
The experimental therapy involves a single stereotactic intracerebral administration of interneurons, specifically targeting individuals with drug-resistant unilateral mesial temporal lobe epilepsy. This approach represents a paradigm shift from traditional treatment methods.
Dr. Amy Z. Crepeau, neurologist and co-principal investigator at Mayo Clinic's Arizona campus, highlights the significance: "For years, patients have asked me about stem cell treatment. It's exciting now to explore a treatment that might impact seizure mechanisms, not in a way that is destructive to tissue but in a regenerative way."
Advanced Imaging and Technical Expertise
The study leverages Mayo Clinic's comprehensive technical capabilities across its three Level 4 Epilepsy Centers. The procedure requires specific imaging protocols for MRI-guided cell implantation, developed through collaboration between Dr. Justin Cramer, a neuroradiologist, and Dr. Yuxiang Zhou, a medical physicist.
Mayo Clinic's advanced imaging capabilities include:
- 7T MRI scanning, which detects abnormalities in 30% of cases deemed normal on standard 3T MRI
- Magnetoencephalography for precise electrode placement
- Photon-counting CT for enhanced visualization
- SISCOM (Subtraction ictal SPECT coregistered to MRI) for precise seizure-related blood flow measurement
Comprehensive Treatment Approach
The regenerative therapy trial adds to Mayo Clinic's existing arsenal of treatment options. Dr. Parker explains the current landscape: "Medications try to correct the abnormal circuitry pharmacologically. Traditional surgery tries to ablate or disconnect the circuitry, and stimulation therapies try to rehabilitate it. Regenerative therapy could be transformative."
Patient Care and Future Implications
Mayo Clinic emphasizes individualized patient care in epilepsy treatment. The team recommends early consultation for various patient groups, including those who:
- Continue experiencing seizures after trying two medications
- Show brain lesions on imaging despite controlled seizures
- Have generalized epilepsy resistant to medical therapy
"Conversations with patients early in the disease course are very important," emphasizes Dr. Parker. "We want to offer people every appropriate opportunity to treat their seizures. We have to move the field forward."
