Development of a new therapeutic for directing target specific stem cell migration and treatment
Grant Award Details
Grant Type:
Grant Number:
DISC2-12666
Investigator(s):
Disease Focus:
Human Stem Cell Use:
Award Value:
$1,129,512
Status:
Active
Grant Application Details
Application Title:
Development of a new therapeutic for directing target specific stem cell migration and treatment
Public Abstract:
Research Objective
A drug-stem cell combination therapy wherein the drug will direct and promote the delivery and distribution of stem cells to the disease site for the optimal therapeutic effect of the stem cells
Impact
Amyotrophic lateral sclerosis (ALS) and the way to deliver and enhance stem cell-based treatment of ALS
Major Proposed Activities
A drug-stem cell combination therapy wherein the drug will direct and promote the delivery and distribution of stem cells to the disease site for the optimal therapeutic effect of the stem cells
Impact
Amyotrophic lateral sclerosis (ALS) and the way to deliver and enhance stem cell-based treatment of ALS
Major Proposed Activities
- Complete the additional in vitro studies and initiate the in vivo studies in SOD1 mouse model
- Determine whether the combined effect of hNSCs intraparenchymally augmented/guided by SDV1a has a synergistic effect on improving disease onset/progression & symptom-free survival in the SOD1 mouse
- Establish the preliminary toxicity and pharmacokinetics profiles of SDV1a in mouse model
- Elucidation of structure and other characteristics; development and validation of analytical procedures
- Process development and characterization in lab scale, stability study
Statement of Benefit to California:
This new therapeutic will address a significant unmet medical need in the treatment of amyotrophic lateral sclerosis (ALS) and have important benefits to the patients with ALS and impact on the healthcare and bio industry in California.