International Epilepsy Day 2025: Looking Back at Progress for Cell and Gene Therapy
In observance of International Epilepsy Day, held annually on the second Monday of February, we took a look back at the past year's news in cell and gene therapy for epilepsy indications.
According to the Epilepsy Foundation, 65 million people in the world, including over 3.4 million in the United States, live with epilepsy.1 Furthermore, over 150,000 people in the US are diagnosed with epilepsy each year, and 1 in 26 people in the US are expected to be diagnosed with the condition at some point in their lifetime. Notably, a third of people with epilepsy continue to have uncontrolled seizures, as no commercially available treatments are efficacious for these patients.
An important area of interest for new therapeutic development in epilepsy is cell therapy, gene therapy, and other advanced modalities such as RNA therapy. A number of companies and academic institutions are now pursuing the development of such advanced treatments, with several clinical trials currently active. In honor of International Epilepsy Day, observed annually on the second Monday of February by the patient and clinician communities, CGTLive® is taking a look back at the progress that has been made for advanced therapeutics for epilepsy indications, including Dravet syndrome, over the past year. Click the "READ MORE" buttons for more details and information about each item.
Neurona’s Focal Epilepsy Cell Therapy NRTX-1001 Garners FDA RMAT Designation
June 21, 2024 — Neurona Therapeutics’ NRTX-1001, an investigational allogeneic regenerative neural cell therapy intended to treat drug-resistant mesial temporal lobe epilepsy (MTLE), has been granted regenerative medicine advanced therapy (RMAT) designation by the FDA.
NRTX-1001 consists of human interneurons that provide long-term secretion of gamma-aminobutyric acid, an inhibitory neurotransmitter, which is expected to repair neural networks. It is currently being evaluated in a phase 1/2 clinical trial (NCT05135091) for the treatment of drug-resistant unilateral MTLE, which is in the midst of treating patients, and a separate phase 1/2 clinical trial (NCT06422923) for patients with drug-resistant bilateral MTLE, which has not yet begun recruiting patients according to its clinicaltrials.gov page, which was most recently updated on May 31, 2024.
“We’re pleased the FDA has recognized the potential of NRTX-1001, and we see the RMAT designation as validating both our positive initial data as well as our novel regenerative approach to treating focal epilepsy,” Cory R. Nicholas, PhD, the cofounder and CEO of Neurona Therapeutics, said in a statement. “In the near-term, we are focused on completing the ongoing phase 1/2 study [in unilateral MTLE] and engaging with the FDA to discuss our product development plan. The Neurona team is committed to bringing our cell therapy to people living with drug-resistant focal epilepsy as safely and expeditiously as possible.”
Stoke Therapeutics’ Dravet Syndrome ASO STK-001 Demonstrates Ability to Reduce Seizure Frequency on Top of SOC
April 11, 2024 — Stoke Therapeutics’ STK-001, an investigational antisense oligonucleotide (ASO), has demonstrated the ability to reduce seizure frequency in patients with Dravet syndrome in data from several clinical trials and open-label extension (OLE) studies. Notably, the seizure reductions occurred on top of reductions already observed from standard of care (SOC) antiseizure medications the patients had been taking.
The data comes from the phase 1/2a MONARCH clinical trial and its OLE SWALLOWTAIL and the phase 1/2a ADMIRAL clinical trial and its OLE LONGWING. Data from 19 patients treated in MONARCH and ADMIRAL with 1-3 doses of STK-001 at 70mg per dose was combined for analysis. It was found that at 3 months posttreatment, the 8 patients who received a single 70mg dose showed a mean 43% reduction from baseline in convulsive seizure frequency and the 10 patients (1 patient’s data was excluded because of a change in background antiseizure medication) who received 2 or 3 70mg doses showed a mean 85% reduction from baseline in convulsive seizure frequency. At 6 months posttreatment, 7 of the patients who received a single 70mg dose (1 patient’s data was excluded because more than 50% of the seizure diary was missing) showed a mean 57% reduction from baseline in convulsive seizure frequency and 9 of the patients who received 2-3 70mg doses (an additional patient’s data was excluded because of a change in background antiseizure medication) showed a mean 74% reduction from baseline in convulsive seizure frequency. It was noted that participants in MONARCH and ADMIRAL were highly refractory to SOC treatment and were taking the best available antiseizure medications, with 85% of participants taking 3 or more medications for seizure control and 54% of participants taking 4 or more medications for seizure control. Fenfluramine was being taken by half of the participants.
The Promise of Cell Therapy Approaches in Epilepsy
December 17, 2024 — The Mind Moments podcast features exclusive interviews with leaders in neurology discussing the latest research and disease management strategies across the breadth of the field, including epilepsy, multiple sclerosis, Parkinson disease, dementia, sleep disorders, and more.
In this episode, "The Promise Behind Cell Therapy Approaches in Epilepsy" Jonathan Parker, MD, PhD, an assistant professor of neurosurgery at Mayo Clinic Arizona, speaks on the emerging interest in cell therapy as a potential means to address epilepsy. The discussion, which took place at the 2024 American Epilepsy Society (AES) Annual Meeting in Los Angeles, California, goes into the rationale behind this approach and the advantages it may have over traditional surgical approaches that can result in detrimental cognitive effects.
Salvador Rico, MD, PhD, on Developing Gene Regulation Therapy for Dravet Syndrome
June 22, 2024 — CGTLive spoke with Salvador Rico, MD, PhD, chief medical officer, Encoded Therapeutics, to learn more about ETX-101 and what differentiates it from other novel genetic therapies in the investigational field. He shared that the company should soon be initiating clinical trials. He also gave an overview of the company’s strategy with its Dravet program, including the clinical trials, sharing data from natural history studies that Encoded has conducted, and connecting with patient advocacy groups to better understand what end points represent clinically meaningful changes for patients with Dravet and better inform clinical trial design.
