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Translating Basic Research into Safe and Effective Cell-based Treatments for Respiratory Diseases

Ikonomou, Laertis; Wagner, Darcy E LU ; Turner, Leigh and Weiss, Daniel J (2019) In Annals of the American Thoracic Society 16(6). p.657-668
Abstract

Respiratory diseases, such as chronic obstructive pulmonary disease and pulmonary fibrosis, result in severely impaired quality of life and impose significant burdens on healthcare systems worldwide. Current disease management involves pharmacologic interventions, oxygen administration, reduction of infections, and lung transplantation in advanced disease stages. An increasing understanding of mechanisms of respiratory epithelial and pulmonary vascular endothelial maintenance and repair and the underlying stem/progenitor cell populations, including but not limited to airway basal cells and type II alveolar epithelial cells, has opened the possibility of cell replacement-based regenerative approaches for treatment of lung diseases.... (More)

Respiratory diseases, such as chronic obstructive pulmonary disease and pulmonary fibrosis, result in severely impaired quality of life and impose significant burdens on healthcare systems worldwide. Current disease management involves pharmacologic interventions, oxygen administration, reduction of infections, and lung transplantation in advanced disease stages. An increasing understanding of mechanisms of respiratory epithelial and pulmonary vascular endothelial maintenance and repair and the underlying stem/progenitor cell populations, including but not limited to airway basal cells and type II alveolar epithelial cells, has opened the possibility of cell replacement-based regenerative approaches for treatment of lung diseases. Further potential for personalized therapies, including in vitro drug screening, has been underscored by the recent derivation of various lung epithelial, endothelial, and immune cell types from human induced pluripotent stem cells. In parallel, immunomodulatory treatments using allogeneic or autologous mesenchymal stromal cells have shown a good safety profile in clinical investigations for acute inflammatory conditions such as acute respiratory distress syndrome and septic shock. As yet, no cell-based therapy has been shown to be both safe and effective for any lung disease. Despite the investigational status of cell-based interventions for lung diseases, businesses that market unproven, unlicensed and potentially harmful cell-based interventions for respiratory diseases have proliferated in the U.S. and worldwide. The current status of various cell-based regenerative approaches for lung disease as well as the effect of the regulatory environment on clinical translation of such approaches are presented and critically discussed in this review.

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author
organization
publishing date
type
Contribution to journal
publication status
published
subject
in
Annals of the American Thoracic Society
volume
16
issue
6
pages
657 - 668
publisher
American Thoracic Society
external identifiers
  • scopus:85066435433
ISSN
2325-6621
DOI
10.1513/AnnalsATS.201812-890CME
language
English
LU publication?
yes
id
49affb9b-87ee-4fde-8639-15d4b95ad0e9
date added to LUP
2019-03-31 19:00:20
date last changed
2019-07-09 04:43:01
@article{49affb9b-87ee-4fde-8639-15d4b95ad0e9,
  abstract     = {<p>Respiratory diseases, such as chronic obstructive pulmonary disease and pulmonary fibrosis, result in severely impaired quality of life and impose significant burdens on healthcare systems worldwide. Current disease management involves pharmacologic interventions, oxygen administration, reduction of infections, and lung transplantation in advanced disease stages. An increasing understanding of mechanisms of respiratory epithelial and pulmonary vascular endothelial maintenance and repair and the underlying stem/progenitor cell populations, including but not limited to airway basal cells and type II alveolar epithelial cells, has opened the possibility of cell replacement-based regenerative approaches for treatment of lung diseases. Further potential for personalized therapies, including in vitro drug screening, has been underscored by the recent derivation of various lung epithelial, endothelial, and immune cell types from human induced pluripotent stem cells. In parallel, immunomodulatory treatments using allogeneic or autologous mesenchymal stromal cells have shown a good safety profile in clinical investigations for acute inflammatory conditions such as acute respiratory distress syndrome and septic shock. As yet, no cell-based therapy has been shown to be both safe and effective for any lung disease. Despite the investigational status of cell-based interventions for lung diseases, businesses that market unproven, unlicensed and potentially harmful cell-based interventions for respiratory diseases have proliferated in the U.S. and worldwide. The current status of various cell-based regenerative approaches for lung disease as well as the effect of the regulatory environment on clinical translation of such approaches are presented and critically discussed in this review.</p>},
  author       = {Ikonomou, Laertis and Wagner, Darcy E and Turner, Leigh and Weiss, Daniel J},
  issn         = {2325-6621},
  language     = {eng},
  month        = {03},
  number       = {6},
  pages        = {657--668},
  publisher    = {American Thoracic Society},
  series       = {Annals of the American Thoracic Society},
  title        = {Translating Basic Research into Safe and Effective Cell-based Treatments for Respiratory Diseases},
  url          = {http://dx.doi.org/10.1513/AnnalsATS.201812-890CME},
  volume       = {16},
  year         = {2019},
}