Phase 1/2 Study of NRTX-1001 Neural Cell Therapy in Subjects with Drug-Resistant Unilateral Mesial Temporal Lobe Epilepsy
Grant Award Details
Grant Type:
Grant Number:
CLIN2-13355
Investigator(s):
Disease Focus:
Human Stem Cell Use:
Award Value:
$7,999,999
Status:
Active
Grant Application Details
Application Title:
Phase 1/2 Study of NRTX-1001 Neural Cell Therapy in Subjects with Drug-Resistant Unilateral Mesial Temporal Lobe Epilepsy
Public Abstract:
Therapeutic Candidate or Device
NRTX-1001 is an inhibitory neuron cell therapy derived from human embryonic stem cells.
Indication
Focal epilepsy; drug-resistant mesial temporal lobe epilepsy.
Therapeutic Mechanism
NRTX-1001 is an inhibitory neuron cell therapy derived from human embryonic stem cells (hESCs) for the prospective treatment of drug-resistant focal epilepsy (MTLE). NRTX-1001 is delivered as a single dose by intracranial injection into the seizure focus, and is intended to distribute locally, functionally integrate, and release the inhibitory neurotransmitter GABA. NRTX-1001 is expected to persist long-term following a single dose and not require repeated administration.
Unmet Medical Need
Current treatments for drug-resistant MTLE include surgical resection and ablation; both are tissue-destructive and can cause serious, irreversible adverse effects. There is a clear need to develop targeted, non-tissue-destructive, and long-lasting therapies that are safe and effective for MTLE.
Project Objective
Initiate/conduct Phase 1; Plan for Phase 2
Major Proposed Activities
NRTX-1001 is an inhibitory neuron cell therapy derived from human embryonic stem cells.
Indication
Focal epilepsy; drug-resistant mesial temporal lobe epilepsy.
Therapeutic Mechanism
NRTX-1001 is an inhibitory neuron cell therapy derived from human embryonic stem cells (hESCs) for the prospective treatment of drug-resistant focal epilepsy (MTLE). NRTX-1001 is delivered as a single dose by intracranial injection into the seizure focus, and is intended to distribute locally, functionally integrate, and release the inhibitory neurotransmitter GABA. NRTX-1001 is expected to persist long-term following a single dose and not require repeated administration.
Unmet Medical Need
Current treatments for drug-resistant MTLE include surgical resection and ablation; both are tissue-destructive and can cause serious, irreversible adverse effects. There is a clear need to develop targeted, non-tissue-destructive, and long-lasting therapies that are safe and effective for MTLE.
Project Objective
Initiate/conduct Phase 1; Plan for Phase 2
Major Proposed Activities
- Initiate and conduct FIH Study of NRTX-1001 in subjects with drug-resistant MTLE (NCT05135091). Includes preclinical and technical support.
- Clinical operations supporting planning of Stage 2. Includes preclinical and technical support.
Statement of Benefit to California:
Epilepsy affects more than 3 million people in the United States; one-third of epilepsy patients are drug-resistant. Transplantation of inhibitory neuron cell therapy is a novel therapeutic strategy that has shown promise in preclinical studies and could potentially provide a non-tissue-destructive therapeutic option for suppressing seizures in people with drug-resistant focal epilepsy. California medical institutions will participate in the NRTX-1001 clinical trial.
