Development of a human stem cell-derived inhibitory neuron therapeutic for the treatment of chronic focal epilepsy
Grant Award Details
Grant Type:
Grant Number:
TRAN1-11611
Investigator(s):
Disease Focus:
Human Stem Cell Use:
Award Value:
$4,848,505
Status:
Closed
Progress Reports
Reporting Period:
Operational Milestone #4
Grant Application Details
Application Title:
Development of a human stem cell-derived inhibitory neuron therapeutic for the treatment of chronic focal epilepsy
Public Abstract:
Translational Candidate
A cellular therapeutic comprised of inhibitory nerve cells produced from human stem cells
Area of Impact
Drug-resistant chronic temporal lobe epilepsy
Mechanism of Action
The product candidate is intended to be delivered into the seizure focus, integrate, and secrete the inhibitory neurotransmitter GABA to rebalance neural electrical activity in the brain and eliminate/reduce seizures.
Unmet Medical Need
The seizures in approximately one-third of epilepsy patients do not adequately respond to current anti-epileptic drugs. Alternative surgical interventions are highly invasive and damage brain tissues. The proposed product candidate is intended to be restorative and long-acting.
Project Objective
Pre-IND meeting; Pilot material manufactured
Major Proposed Activities
A cellular therapeutic comprised of inhibitory nerve cells produced from human stem cells
Area of Impact
Drug-resistant chronic temporal lobe epilepsy
Mechanism of Action
The product candidate is intended to be delivered into the seizure focus, integrate, and secrete the inhibitory neurotransmitter GABA to rebalance neural electrical activity in the brain and eliminate/reduce seizures.
Unmet Medical Need
The seizures in approximately one-third of epilepsy patients do not adequately respond to current anti-epileptic drugs. Alternative surgical interventions are highly invasive and damage brain tissues. The proposed product candidate is intended to be restorative and long-acting.
Project Objective
Pre-IND meeting; Pilot material manufactured
Major Proposed Activities
- Finalize manufacturing process to be appropriate for future clinical use
- Produce Pilot product using the intended process, confirm efficacy in two rodent models of chronic epilepsy and demonstrate safety at maximum dose
- Select intended clinical cell delivery device and conduct preIND meeting to confirm IND-enabling preclinical requirements
Statement of Benefit to California:
Epilepsy is the fourth most common neurological disorder affecting more than 400,000 people in the State of California. One-third of epilepsy patients are considered to be drug-resistant and have persistent, uncontrolled seizures that can be disabling and affect quality of life. Alternative surgical interventions are highly invasive and may cause lasting impairment. This proposal aims to further develop a cellular therapeutic for treating drug-resistant epilepsy.
