Regenerative Brain Cell Procedure for Epilepsy Performed

UC San Diego Health has become one of the first health systems in the country to inject regenerative cells into the brain to treat epileptic seizures, which could lead to a breakthrough advancement in the treatment of temporal lobe epilepsy.

UC San Diego Health’s multidisciplinary team performed the third experimental regenerative brain cell treatment procedure earlier this month as part of a national clinical trial. UC San Diego Health is the region’s only nationally designated Level 4 Adult Epilepsy Center.

Under the bright lights of the operating room, Sharona Ben-Haim, M.D., associate professor of neurological surgery at the University of California San Diego School of Medicine and surgical director of epilepsy at UC San Diego Health, made multiple injections of inhibitory brain cells into mapped out precision points of the patient’s brain.

Interneurons are cells produced from human stem cells. If successful, NRTX-1001, the first-ever regenerative human cell experimental therapy, could offer drug-resistant temporal lobe epilepsy patients the first non-destructive option for potentially curing their seizures.

Ben-Haim meticulously analyzed the intra-operative magnetic resonance imaging images that pinpoint her every step while setting up several trajectory points on the patient’s brain prior to cell insertion.

While digitally rotating the brain three-dimensionally on screen to inspect her work, Ben-Haim explained, “This experimental therapy offers us the potential to essentially restore the balance in the brain to be able to calm and ideally stop the seizures, while retaining the normal function of that part of the brain. Currently, we do not have a therapy that allows us to do that, so this is really exciting.”

The Neurona Therapeutics-sponsored clinical trial is looking to enroll 40 people across the country to study the effects of implanting stem cells that produce gamma-aminobutyric acid (GABA) — a neurotransmitter that blocks overactive impulses between nerve cells in the brain.

“In drug-resistant temporal lobe epilepsy, some of the normal brain cells in the temporal lobe have been damaged or are dead,” said Jerry Shih, M.D., professor of neurosciences at UC San Diego School of Medicine, neurologist and director of the Epilepsy Center at UC San Diego Health. “This experimental cell therapy implants healthy human brain cells into the damaged temporal lobe with the hope that those new cells will begin establishing connections in the patient’s brain, to ultimately make a healthier temporal lobe.”

According to the Centers for Disease Control and Prevention, epilepsy is the fourth most frequent neurological ailment in the United States, trailing migraine, stroke, and Alzheimer’s disease. Epilepsy affects an estimated 3.4 million Americans, and around one-third of those people do not react to anti-seizure drugs.

Traditional seizure reduction methods include removing or laser-burning the areas of the brain where seizures originate, as well as implanting deep-brain electrodes to control seizure activity. This novel experimental regenerative therapy might potentially treat different regions of the brain without removing tissue, giving drug-resistant epilepsy patients new hope.

“This first-in-human clinical trial represents a paradigm shift in the way we treat this disease process, shifting from procedures that destroy bad tissue to procedures that repair the bad tissue,” Shih said. “Our hope is that this procedure has such a high success rate and good tolerability that it becomes the standard of care for all drug-resistant focal epilepsies.”

Ben-Haim returned to the operating room and thoroughly examined her work on her patient’s brain. He is the third person in the US to have the treatment, which began in June 2022 at SUNY Upstate Medical University in Syracuse, New York, and will be completed in November 2022 at Oregon Health Sciences University in Portland, Oregon.

“These patients are willing to try an experimental procedure in this clinical trial to get control of their seizures, and I think they are incredibly brave,” Ben-Haim said. “We are already seeing improvements in as early as one month. Our ultimate goal is to improve a patient’s long-term quality of life.”

Patients in the trial will be evaluated on a regular basis for two years following the procedure to study the impact of the implanted stem cells. Preliminary findings presented in June show a more than 90% reduction in seizure frequency in the first and second patients one year and seven months after treatment, respectively.

Shih, the principle investigator for UC San Diego Health’s participation, said the study is the most complex clinical trial he’s worked on in his 25-year career doing clinical trials at three prominent academic institutions throughout the country.

“This study can only be conducted in an institution with a strong clinical and research infrastructure, which we are fortunate to have here at UC San Diego Health,” Shih said.

He went on to say that it took a lot of collaboration among UC San Diego teams, including faculty and staff from neurosciences, neurosurgery, cellular regenerative medicine, radiology, neuropsychology, and neurocritical care; the California Institute for Regenerative Medicine Alpha Clinic at the Sanford Stem Cell Clinical Center; the Advanced Cell Therapy Lab; the Center for Multimodal Imaging and Genetics, and the Consortium for Regenerative Medicine.

“We would not have been able to participate in this study without the active collaboration of all these integral groups. It truly takes a village.”

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