The grant will fund a new study to evaluate the effectiveness of brain-responsive neuromodulation in patients with LGS. The study is expected to begin accepting patients in the second half of 2022.
The National Institutes of Health (NIH) has awarded a grant to NeuroPace, which will fund an Investigation Device Exemption (IDE) study to evaluate the safety and use of its responsive neurostimulation (RNS) system in treating patients with Lennox-Gastaut syndrome (LGS).1
A total of $9.3 million, funded through the Brain Research through Advancing Innovative Neurotechnologies(BRAIN) Initiative, will be allocated over a period of 5 years. In addition to exploring brain-responsive neuromodulation technology as a new therapy for those living with LGS, individualized therapy may be optimized as a result of continual monitoring and recording of brain activity through the RNS system.
Upon approval from the FDA, the study will be conducted primarily to evaluate the safety and efficacy of RNS system technology to treat seizures associated with LGS. The IDE study will be the first to evaluate a neuromodulation device in patients with LGS and is anticipated to begin accepting patients in the latter half of 2022.
“LGS is a horrific epilepsy syndrome that develops in very young children and results in daily seizures, frequent seizure emergencies and hospitalizations, and significant developmental delays,” Tracy Dixon-Salazar, PhD, executive director, LGS Foundation, said in a statement.1 “Most people living with LGS have tried more than a dozen treatments and yet seizures persist, and families live life waiting for the next seizure crisis. I am so encouraged by the research being done with the RNS system and am hopeful that this treatment can help LGS families who are in desperate need of better therapies.”
A total of 20 patients with LGS and drug-resistant generalize onset seizures will be divided into cohorts of 10, with safety and efficacy results from the preliminary group determining the participation of the second cohort. A total of 8 US academic centers will collaborate in the study, with 6 clinical sites enrolling patients. In an effort to better personalize treatment, 2 additional academic sites will create individualized maps of brain seizure networks for patients.
Martha Morrell, MD, chief medical officer, NeuroPace, was selected as the principal investigator of the study. “We are pleased that the NIH recognizes the promise of responsive neuromodulation to potentially address the current gap in therapeutic options for patients with debilitating seizure disorders such as LGS,” Morell said in a statement.1
“We’re hopeful that treatment with the RNS system can ultimately improve the lives of patients with LGS and their families, and that what is learned from the brain data can also be applied to treatment of other types of generalized onset seizures,” she added.
Approval of new labeling for the RNS system was granted to NeuroPace in March 2020, allowing patients with non-brain MRI requirements to receive treatment from the stimulation device.2 Data collected from the new IDE study will be used in future studies and initiatives to potentially identify a biomarker for when seizures occur, as well as an aid in epilepsy research and clinical response.
This is not the first foray into epilepsy treatment for NeuroPace, with prior data collected from 2 seminal studies of the RNS system suggesting its success in reducing seizures and improve quality of life for people living with refractory epilepsy.
The first study, published in Epilepsia, included 150 patients treated for at least 1 year with the RNS system and included data on serious adverse events (AEs) and the median percent change in disabling seizure frequency from baseline at years 1, 2, and 3 of treatment and at most recent follow-up. The median reduction in seizures was 67% (interquartile range [IQR], 33-99; n = 149) at 1 year, 75% (IQR, 50-94; n = 93) at 2 years, 82% (IQR, 50-96; n = 38) at 3 or more years, and 74% (IQR, 50–96; n = 150) at last follow-up (mean, 2.3 years).3
The second trial was a seminal 9-year, multicenter, long-term treatment study published in Neurology. It was the largest prospective neuromodulation trial in the field of epilepsy, including 230 patients across 34 centers with nearly 1900 patient-implant years of follow-up. In that study, adults treated with the RNS system within 2-year feasibility or randomized controlled trials entered into a long-term prospective open label trial to assess safety, efficacy, and quality of life (QoL) over an additional 7 years. At 9 years, the median percent reduction in seizure frequency was 75% (P <.0001), responder rate was 73%, and 35% of patients had a 90% or more reduction in seizure frequency.4