CNS10-NPC-GDNF delivered into the motor cortex for the treatment of ALS
Grant Award Details
Grant Type:
Grant Number:
CLIN2-12319
Investigator(s):
Disease Focus:
Human Stem Cell Use:
Award Value:
$11,990,372
Status:
Active
Grant Application Details
Application Title:
CNS10-NPC-GDNF delivered into the motor cortex for the treatment of ALS
Public Abstract:
Therapeutic Candidate or Device
CNS10-NPC-GDNF - a neural progenitor cell secreting GDNF
Indication
Amyotrophic Lateral Sclerosis (ALS)
Therapeutic Mechanism
This therapy will replace damaged astrocytes. The new astrocytes will release paracrine factors. As the cells have been modified to release GDNF they will also provide this neuroprotective factor to dying motor neurons in the motor cortex.
Unmet Medical Need
There are currently only two FDA-approved therapies with minimal benefits, but there is no cure for ALS. Thus there is a huge unmet medical need to find additional therapies with longer-lasting benefits.
Project Objective
Phase 1/2a completed
Major Proposed Activities
CNS10-NPC-GDNF - a neural progenitor cell secreting GDNF
Indication
Amyotrophic Lateral Sclerosis (ALS)
Therapeutic Mechanism
This therapy will replace damaged astrocytes. The new astrocytes will release paracrine factors. As the cells have been modified to release GDNF they will also provide this neuroprotective factor to dying motor neurons in the motor cortex.
Unmet Medical Need
There are currently only two FDA-approved therapies with minimal benefits, but there is no cure for ALS. Thus there is a huge unmet medical need to find additional therapies with longer-lasting benefits.
Project Objective
Phase 1/2a completed
Major Proposed Activities
- Enrollment of 16 patients to a Phase 1/2a trial to demonstrate safety and preliminary efficacy of the cellular product
- GMP manufacturing of a new CNS10-NPC-GDNF working cell lot for future clinical trials in ALS.
Statement of Benefit to California:
ALS is a devastating disease and there are over 6,000 cases in CA. If this treatment works it will provide one of the only ways to slow down motor neuron disease progression. This illness costs the state of California millions of dollars in healthcare costs and immense suffering to those Californians affected by the disease. Additionally, because over 90% of the funds will be used in California, it will help stimulate the economy.