Development and Evaluation of Stem Cell based Therapy for Diffuse Parenchymal Lung Diseases

Funding Type: 
Basic Biology III
Grant Number: 
RB3-02157
Investigator: 
ICOC Funds Committed: 
$0
oldStatus: 
Closed
Public Abstract: 
The life expectancy of persons diagnosed with Idiopathic Pulmonary Fibrosis (IPF) is 2 to 3 years, worse than many cancers. It belongs to the family of idiopathic interstitial pneumonias (IIPs), and represents the most aggressive form of diffuse parenchymal lung disease (DPLD). As many as 500,000 patients are presently affected in the EU and US. We and others think that progression of lung fibrosis in IPF and related forms of DPLD is primarily based on an overwhelming, albeit insufficient progenitor cell response, which exceeds the regenerative capacity of the alveolar lining epithelium. Therefore, stem cell-based therapies appear as a promising and probably the only effective approach to change the dire fate of this disease. Goal: To devise and optimize novel pre-clinical stem cell based therapeutic solutions to DPLD. Future Aim. To show that amniotic fluid derived stem cells are a novel, safe, scalable and well immune tolerated source of ethically neutral cells that have major pro-homeostatic effects on the alveolar milieu in DPLD and thus to justify an IND filing for a phase 1 trial of these cells in human patients with terminal DPLD. Significance: If, as we expect, and our preliminary data supports, this novel stem/progenitor cell based therapeutic approach works and is not toxic in murine models of IPF, it will be a major innovation, leading to a potentially important translational advance for several human DPLDs, including IPF, which presently have no effective therapy.
Statement of Benefit to California: 
The life expectancy of persons diagnosed with Idiopathic Pulmonary Fibrosis (IPF) is 2 to 3 years, worse than many cancers. It belongs to the family of idiopathic interstitial pneumonias (IIPs), and represents the most aggressive form of diffuse parenchymal lung disease (DPLD). As many as 500,000 patients are presently affected in the EU, US and California. We and others think that progression of lung fibrosis in IPF and related forms of DPLD is primarily based on an overwhelming, albeit insufficient progenitor cell response, which exceeds the regenerative capacity of the alveolar lining epithelium. Therefore, stem cell-based therapies appear as a promising and probably the only effective approach to change the dire fate of this disease. If proven effective, this innovative treatment would provide a major translational and biotechnology business opportunity for the State and citizens of California.

© 2013 California Institute for Regenerative Medicine