By: Catherine Joachin
Stem Cell Therapy and Epilepsy
Introduction
Around one-third of patients diagnosed with epilepsy do not respond to conventional anti-seizure medication (Hlebokazov et al., 2021). This condition, known as refractory or drug-resistant epilepsy, is marked by persistent uncontrolled seizures and can significantly impact the mental, physical and social well-being of those living with it (Janson & Bainbridge, 2021).
Medically refractory epilepsy is traditionally managed using surgical and interventional methods; however, growing research interest in stem cell therapy has driven the development of alternative treatment options to treat the condition (Alayli et al., 2023).
What is stem cell therapy?
Stem cells are undifferentiated cells with the potential to self-renew and develop into various cell types (Mayo Clinic, 2024). They can be classified into different categories based on their origin:
- Embryonic stem cells (ESCs), which are derived from the inner cell mass of the early developing human embryo
- Perinatal stem cells, derived from placental tissue such as the umbilical cord
- Adult stem cells (ASCs) include neural stem cells (NSCs) and mesenchymal stem cells (MSCs), which originate from bone marrow and adipose tissue
- Induced pluripotent stem cells (iPSCs), which are genetically engineered adult stem cells with the same properties as ESCs
The application of stem cells in epilepsy care offers the possibility to replace damaged or lost neurons, restore brain function and stabilize excitatory-inhibitory balance while eliminating the need for real-time seizure detection techniques (Alayli et al., 2023).
Stem Cell Therapy in Epilepsy
Recent studies on the use of stem cell therapy in seizure control have demonstrated promising therapeutic results, with early animal models exhibiting a reduction in both the frequency and duration of seizures (Alayli et al., 2023). An American study even found that transplanting hiPSCs derived GABAergic interneurons in epileptic mice mitigated seizures and improved cognitive function, aggressive behaviour and mood, highlighting possible restorative benefits (Cunningham et al., 2024).
In theory, stem cell transplantation could have the potential to provide important therapeutic effects for epilepsy patients, however, high-quality clinical data on its application is limited (Aligholi et al., 2021).
Recently, researchers found that anti-epileptic drug treatment combined with mesenchymal stem cell (MSC) injection derived from the bone marrow of the same individual led to long-term reduction to complete suppression of seizures (Hlebokazov et al., 2021).
Moreover, the first in-human investigation of stem cell therapy for refractory epilepsy was initiated in 2025. It involved transplanting candidate NRTX-1001 inhibitory interneurons into the hippocampus of affected patients to correct hyperexcitability caused by seizures. This experimental cell therapy targets focal epilepsy and is derived from human pluripotent stem cells inhibiting GABAergic neurons. So far, reports indicate that treatment is well tolerated and leads to seizure reduction across participants, suggesting potential long-term benefits for drug-resistant focal epilepsy, but more trials are still needed to confirm its effectiveness (Hixson et al., 2025).
References
Alayli, A., Lockard, G., Gordon, J., Connolly, J., Monsour, M., Schimmel, S., Dela Peña, I., & Borlongan, C. V. (2023). Stem Cells: Recent Developments Redefining Epilepsy Therapy. Cell Transplantation, 32, 9636897231158967. https://doi.org/10.1177/09636897231158967
Chang, B.-L., & Chang, K.-H. (2022). Stem Cell Therapy in Treating Epilepsy. Frontiers in Neuroscience, 16, 934507. https://doi.org/10.3389/fnins.2022.934507
Cunningham, M., Cho, J.-H., Leung, A., Savvidis, G., Ahn, S., Moon, M., Lee, P. K. J., Han, J. J., Azimi, N., Kim, K.-S., Bolshakov, V. Y., & Chung, S. (2014). hPSC-Derived Maturing GABAergic Interneurons Ameliorate Seizures and Abnormal Behavior in Epileptic Mice. Cell Stem Cell, 15(5), 559–573. https://doi.org/10.1016/j.stem.2014.10.006
Hixson, J., Spencer, D., Burchiel, K., Babu, H., Beach, R., Shih, J., Ben-Haim, S., O’Dwyer, R., Sani, S., Southwell, D., Luedke, M., Blum, D., Banik, G., Bershteyn, M., Bulfone, A., Feld, B., Finefrock, H., Fuentealba, L., Jung, J.-H., … Nicholas, C. (2025). First-in-Human Trial of NRTX-1001 GABAergic Interneuron Cell Therapy for Focal Epilepsy – Updated Clinical Trial Results (P8-9.003). Neurology, 104(7_Supplement_1). https://doi.org/10.1212/WNL.0000000000210525
Hlebokazov, F., Dakukina, T., Potapnev, M., Kosmacheva, S., Moroz, L., Misiuk, N., Golubeva, T., Slobina, E., Krasko, O., Shakhbazau, A., Hlavinski, I., & Goncharova, N. (2021). Clinical benefits of single vs repeated courses of mesenchymal stem cell therapy in epilepsy patients. Clinical Neurology and Neurosurgery, 207, Article 106736. https://doi.org/10.1016/j.clineuro.2021.106736
Janson, M. T., & Bainbridge, J. L. (2021). Continuing Burden of Refractory Epilepsy. The Annals of Pharmacotherapy, 55(3), 406–408. https://doi.org/10.1177/1060028020948056
Mayo Clinic. (2024). Stem cells: What they are and what they do. Mayo Clinic. https://www.mayoclinic.org/tests-procedures/bone-marrow-transplant/in-depth/stem-cells/art-20048117
