Respiratory Disease Fact Sheet

CIRM funds many projects seeking to better understand respiratory disease and to translate those discoveries into new therapies.

Description

Severe blockage of the major airway,  or trachea, is relatively rare but is severely debilitating and often causes death. Estimates suggest 200 new cases occur in California each year. The blockage most often occurs after injury, treatment for a tumor or following insertion of a breathing tube for other medical procedures. Physicians currently treat these blockages through surgery or by using a stent to hold the airway open. But neither approach produces very good or lasting results. Replacing the damaged trachea with a new one could be a potential cure. A few teams around the world are working with different types of natural and synthetic scaffolds to try to grow new tracheas for transplant using stem cells.

Clinical Stage Programs

University of California, Davis

The team uses a trachea from a cadaver as a scaffold, removing the soft tissue cells and then seeding the remaining scaffold with two types of stem cells from the patient. That construct is grown in a bioreactor until it is ready for transplant in the patient. The team has already used the procedure through the European compassionate use exemption in five dying patients, saving three of their lives. They plan to use this award to do tests in non-human primates to better understand the role of each type of stem cell used to seed the scaffold.

CIRM grants targeting respiratory disorders

Researcher name Institution Grant Title Grant Type Award Amount
Thomas Novak Cellular Dynamics International Generation and characterization of high-quality, footprint-free human induced pluripotent stem cell lines from 3,000 donors to investigate multigenic diseases hiPSC Derivation $16,000,000
Deborah Requesens Coriell Institute for Medical Research The CIRM Human Pluripotent Stem Cell Biorepository – A Resource for Safe Storage and Distribution of High Quality iPSCs hPSC Repository $9,942,175
Peter Belafsky University of California, Davis Tissue Engineered Recellularized Laryngotracheal Implants Disease Team Therapy Development III $3,181,162
Barry Stripp Cedars-Sinai Medical Center Epithelial progenitors and the stromal niche as therapeutic targets in lung disease Research Leadership $4,841,830
Brigitte Gomperts University of California, Los Angeles Stem Cells for Lung Diseases - Overcoming Barriers to Find New Therapies Conference - Lung Regenerative Medicine Workshop $44,548
Matthew Porteus Stanford University Genome Editing to Correct Cystic Fibrosis Mutations in Airway Stem Cells Quest - Discovery Stage Research Projects $1,968,456
Semil Choksi University of California, San Francisco Generation of human airway stem cells by direct transcriptional reprogramming for disease modeling and regeneration Inception - Discovery Stage Research Projects $238,408
James Hagood University of California, San Diego Mesenchymal stem cell extracellular vesicles as therapy for pulmonary fibrosis Quest - Discovery Stage Research Projects $0
Brigitte Gomperts University of California, Los Angeles Stem Cells in Lung Cancer New Faculty II $2,381,572
Martin Birchall University of California, Davis Airways for Children Disease Team Therapy Planning I $19,800
Brigitte Gomperts University of California, Los Angeles Using human induced pluripotent stem cells to improve our understanding of Idiopathic Pulmonary Fibrosis Tissue Collection for Disease Modeling $811,231
Total:
$39,429,182.00

CIRM respiratory disorders videos

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