At the California Institute for Regenerative Medicine (CIRM), we highlight achievements from the research we support.
We recently spoke with Cory Nicholas, PhD, CEO and Co‑Founder of Neurona Therapeutics, about a major milestone for the clinical‑stage company.
Neurona received the FDA’s Regenerative Medicine Advanced Therapy (RMAT) designation for NRTX‑1001, a CIRM‑funded neural cell therapy for drug‑resistant focal epilepsy. The designation affirms the company’s early clinical data and highlights NRTX‑1001’s potential to suppress seizures long‑term.
In this interview, Dr. Nicholas discusses the significance of the RMAT designation, NRTX‑1001’s promise, and what it may mean for patients and the future of regenerative medicine.
Some responses have been edited for clarity and brevity.
Q: Can you describe the research you and your team are doing at Neurona for neural cell therapies and why you’re so passionate about it?
Neurona Therapeutics develops allogeneic, off‑the‑shelf regenerative cell therapies manufactured from a single cell source for all eligible patients. Our therapies are designed as single‑dose treatments for neurological disorders. Epilepsy is our lead indication, and we are committed to delivering safe, effective options for people with drug‑resistant disease.
My interest in neural cell therapy began years ago through personal experiences with family members facing serious illnesses, including stroke. Studying stem cell and developmental biology led me to focus on brain development and repair, where I saw tremendous potential to advance neurology.
I became driven to use stem cell technologies to improve health and looked for ways to translate them to the clinic. I’ve had the privilege of working with outstanding mentors and colleagues who shared this vision. With their guidance and our dedicated team of innovators, we’ve pushed the boundaries of what’s possible.
Q: Can you please tell us about Neurona’s NRTX-1001 cell therapy? Can you explain how the cell therapy works to a lay audience?
NRTX-1001 is a cell therapy that involves the administration of inhibitory neurons into the nervous system, aimed at restoring balance within the brain for individuals with drug-resistant focal epilepsy.
Drug-resistant epilepsy is characterized by uncontrolled seizures caused by a hyperactive imbalance in the brain’s circuitry. The core idea behind our therapy is to supply the missing inhibitory elements to rebalance these circuits.
By administering these inhibitory neurons into targeted parts of the brain, we aim to provide the necessary “brake” that is missing in patients with epilepsy. Once these neurons are in place, they are designed to effectively rebalance the brain’s circuits and stop the seizures.
This approach not only addresses the symptoms but targets the underlying cause of the seizures, offering hope for a long-term solution for those with this form of epilepsy.
Q: Why is the FDA’s Regenerative Medicine Advanced Therapy (RMAT) designation for Neurona’s NRTX-1001 cell therapy significant for patients with epilepsy?
The RMAT designation for NRTX‑1001 marks a key milestone and recognizes Neurona’s progress toward treating uncontrolled seizures. Early data from our ongoing Phase 1/2 trial show that NRTX‑1001 is well‑tolerated and may significantly reduce seizures. The designation enables deeper FDA engagement to explore ways to accelerate development, offering hope to affected individuals and families.
Epilepsy is widely misunderstood as a minor nuisance, which is false. It is a serious, common neurological disorder that harms quality of life and can be fatal. Over 3 million Americans have epilepsy, and despite more than 30 anti‑seizure drugs, 56 percent of adults continue to experience uncontrolled seizures.
Seizures of any frequency can limit independence, prevent driving and employment, and lead to dangerous situations including accidents, falls, and sudden unexpected death. Drug‑resistant seizures and their treatments can also cause memory loss, mood disorders, sleep disruption, drowsiness, social isolation, and psychosis.
Q: How does NRTX-1001 differ from the current standard of treatments?
Current treatments for drug‑resistant epilepsy are often ineffective and can cause severe side effects. Some patients may undergo lobectomy, a high‑risk surgery that can lead to permanent loss of brain function.
We believe cell therapy offers a safer alternative. Our approach aims to regenerate lost inhibitory neurons and restore circuit balance without removing brain tissue. NRTX‑1001 may provide a more effective option and address a significant unmet need.
This is an early step, but it holds strong promise for improving treatment options for people worldwide living with drug‑resistant seizures.
Q: What are some milestones Neurona is hoping to achieve next for NRTX-1001?
We are conducting clinical trials to evaluate the safety, efficacy, and durability of NRTX‑1001. Ten patients with unilateral temporal lobe epilepsy have been treated, and we are enrolling six more in an expansion cohort.
We are also evaluating additional focal epilepsy populations that may benefit from NRTX‑1001. A second trial has begun in ten patients with bilateral temporal lobe epilepsy. This study is just starting, and we plan to expand into other focal epilepsy disorders.
Our initial focus remains epilepsy. We are also performing preclinical research to assess NRTX‑1001 and other neural cell types for disorders with similar hyperactive circuit pathology. These include subsets of Alzheimer’s disease, chronic neuropathic pain, and Parkinson’s disease, where our inhibitory cell therapy may help quiet and repair overactive circuits.
We are excited about these possibilities and committed to advancing research that may help patients with challenging neurological conditions.
Q: How has CIRM funding supported the advancement of Neurona’s NRTX-1001 cell therapy?
CIRM’s support has been vital in advancing epilepsy research and driving NRTX‑1001 from bench to bedside. Their support spans nearly 20 years, beginning with our UCSF grants and continuing after Neurona’s 2015 launch. At the time, human pluripotent stem cells were new, and CIRM’s backing accelerated our research.
We would not be here today without CIRM. Neurona and our regenerative cell therapy approach honor CIRM’s original mission. It also reflects years of dedicated work. We remain deeply grateful to CIRM and their team for their unwavering support.
Learn more about Neurona Therapeutics at their website www.neuronatherapeutics.com.
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