A multidisciplinary team at UC San Diego Health, participating in a clinical trial sponsored by Neurona Therapeutics, recently injected regenerative brains cells into the brain of a patient in an effort to treat their epileptic seizures. The regenerative brain cell therapy uses interneurons derived from human stems cells and has the potential to provide drug-resistant temporal lobe epilepsy patients with the first non-destruction treatment to potentially cure seizures.
“This experimental therapy offers us the potential to essentially restore the balance in the brain to be able to calmly and ideally stop the seizures, while retaining the normal function of that part of the brain,” said Sharon Ben-Haim, MD, associate professor of neurological surgery at University of California San Diego School of Medicine and surgical director of epilepsy at UC San Diego Health. “Currently, we do not have a therapy that allows us to do that, so this is really exciting.”
The trial hopes to enroll 40 patients across the country to study the therapeutic potential of implantation of stems 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 principal investigator Jerry Shih, MD, 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.”
This new, experimental regenerative therapy could provide an alternative to current treatment methods of seizure reduction which remove or laser-burn the areas of the brain where the seizures originate, or use electrodes implanted in the brain to control seizures.
“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.”
Patients enrolled in the trial, which commenced a year ago with the first procedure performed at SUNY Upstate Medical University in Syracuse, NY, will have two years of regular monitoring to collect data on the effects of the implanted stem cells. Preliminary data reported last month showed a reduction of more than 90% in seizure frequency in the first two patients to receive the therapy.
“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.”
Shih noted that the coordination required to provide the therapy including faculty and staff from neurosciences, neurosurgery, cellular regenerative medicine, radiology, neuropsychology and neuro critical care; California Institute for Regenerative Medicine Alpha Clinic at the Sanford Stem Cell Clinical Center; Advanced Cell Therapy Lab; Center for Multimodal Imaging and Genetics and the Consortium for Regenerative Medicine make it the most complex clinical trial he has participated in his 25 year career of leading trials.
