Development of a universal allogeneic human interneuron cell therapy candidate for the treatment of drug resistant focal epilepsy.
Grant Award Details
Grant Type:
Grant Number:
TRAN1-16225
Investigator(s):
Award Value:
$3,828,714
Status:
Pre-Active
Grant Application Details
Application Title:
Development of a universal allogeneic human interneuron cell therapy candidate for the treatment of drug resistant focal epilepsy.
Public Abstract:
Translational Candidate
Development of a universal allogeneic human interneuron cell therapy candidate for the treatment of drug resistant focal epilepsy.
Area of Impact
Non-destructive treatment option for drug-resistant focal epilepsy patients with reduced immunosuppression regimen and universal recipient eligibility
Mechanism of Action
The proposed therapeutic candidate comprises inhibitory interneurons that would be delivered in a one-time procedure into affected region of the brain and is intended to increase local GABAergic inhibitory tone and rebalance neural activity. The proposed candidate represents a cell replacement therapy that could compensate for the loss and dysfunction of the same types of neurons in the epileptic brain and possibly eliminate/significantly reduce seizures.
Unmet Medical Need
Epilepsy ranks as the fourth most common neurological condition in the US. One-third of people with epilepsy are classified as having seizures that are drug-resistant, which can be profoundly disabling and detrimental to their quality of life. Current treatment options do not typically culminate in seizure freedom and/or risk serious adverse events. Hence, there exists a compelling need to develop alternative therapies that are less invasive, non-destructive, safe, effective, and durable.
Project Objective
Pre-IND meeting with the FDA
Major Proposed Activities
Development of a universal allogeneic human interneuron cell therapy candidate for the treatment of drug resistant focal epilepsy.
Area of Impact
Non-destructive treatment option for drug-resistant focal epilepsy patients with reduced immunosuppression regimen and universal recipient eligibility
Mechanism of Action
The proposed therapeutic candidate comprises inhibitory interneurons that would be delivered in a one-time procedure into affected region of the brain and is intended to increase local GABAergic inhibitory tone and rebalance neural activity. The proposed candidate represents a cell replacement therapy that could compensate for the loss and dysfunction of the same types of neurons in the epileptic brain and possibly eliminate/significantly reduce seizures.
Unmet Medical Need
Epilepsy ranks as the fourth most common neurological condition in the US. One-third of people with epilepsy are classified as having seizures that are drug-resistant, which can be profoundly disabling and detrimental to their quality of life. Current treatment options do not typically culminate in seizure freedom and/or risk serious adverse events. Hence, there exists a compelling need to develop alternative therapies that are less invasive, non-destructive, safe, effective, and durable.
Project Objective
Pre-IND meeting with the FDA
Major Proposed Activities
- Evaluation of candidate immunogenicity in a variety of human cellular and humanized mouse models.
- Development and qualification of analytical assays and manufacturing of pilot universal allogeneic human interneuron cell therapy material.
- Pilot Pharmacology, Dose-Finding, and Safety Studies in an established mouse model of TLE.
Statement of Benefit to California:
Epilepsy affects over 400,000 people in California, one-third of whom have seizures that are drug-resistant, can be disabling, and affect quality of life. A universal allogeneic inhibitory neuron cell therapy candidate will benefit California’s diverse population by providing a non-tissue destructive, regenerative treatment alternative for drug resistant focal epilepsy that does not require concurrent immunosuppression and improves safety, accessibility, and clinical utility for all patients.