MRI Guided Delivery of Neural Progenitor Cells Secreting GDNF for the Treatment of Parkinson’s disease
Grant Award Details
Grant Type:
Grant Number:
CLIN1-11059
Investigator(s):
Disease Focus:
Human Stem Cell Use:
Award Value:
$5,757,078
Status:
Closed
Grant Application Details
Application Title:
MRI Guided Delivery of Neural Progenitor Cells Secreting GDNF for the Treatment of Parkinson's disease
Public Abstract:
Therapeutic Candidate or Device
CNS10-NPC-GDNF is a neural progenitor cell line transfected with glial cell line derived neurotrophic factor (GDNF)
Indication
Mid-stage Parkinson's disease (UPDRS stage III or lower)
Therapeutic Mechanism
Degeneration of dopaminergic neurons that project from the substantia nigra to the striatum causes the primary motor symptoms of Parkinson's disease. CNS10-NPC-GDNF cells will be transplanted into the putamen, and are expected undergo limited migration to areas of degeneration, induce sprouting of dopaminergic terminals and protect dopamine cell bodies. The cells can mature into astrocytes that may provide additional protection of degenerating regions through secretion of supportive factors.
Unmet Medical Need
Current treatments provide symptomatic relief of Parkinson's disease (PD), but become less effective over time as they have no effect on the disease process. CNS10-NPC-GDNF is expected to slow the disease progression by inducing sprouting of dopaminergic terminals and protecting dopaminergic cells.
Project Objective
Complete pre-clinical studies, and file an IND.
Major Proposed Activities
CNS10-NPC-GDNF is a neural progenitor cell line transfected with glial cell line derived neurotrophic factor (GDNF)
Indication
Mid-stage Parkinson's disease (UPDRS stage III or lower)
Therapeutic Mechanism
Degeneration of dopaminergic neurons that project from the substantia nigra to the striatum causes the primary motor symptoms of Parkinson's disease. CNS10-NPC-GDNF cells will be transplanted into the putamen, and are expected undergo limited migration to areas of degeneration, induce sprouting of dopaminergic terminals and protect dopamine cell bodies. The cells can mature into astrocytes that may provide additional protection of degenerating regions through secretion of supportive factors.
Unmet Medical Need
Current treatments provide symptomatic relief of Parkinson's disease (PD), but become less effective over time as they have no effect on the disease process. CNS10-NPC-GDNF is expected to slow the disease progression by inducing sprouting of dopaminergic terminals and protecting dopaminergic cells.
Project Objective
Complete pre-clinical studies, and file an IND.
Major Proposed Activities
- Manufacture of CNS10-NPC-GDNF to supply the proposed clinical trial
- Demonstrate longterm lack of tumorigenicity and safety in rats
- Demonstrate safety and tolerability of CNS10-NPC-GDNF in aged MPTP lesioned non-human primate model of Parkinson's disease
Statement of Benefit to California:
Parkinson's disease is a debilitating disease, which puts a huge burden on state resources through the need for care givers and medical care. While primarily an effort to reduce patient and family suffering, this project will also ease the cost of caring for PD patients in California if successful. This in turn will attract scientists, clinicians, and biotech companies to the state of California thus increasing state revenue and state prestige in this rapidly growing field.