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 NameInstitutionGrant TitleGrant TypeAward Amount
Tien PengUniversity of California, San FranciscoEx vivo fate mapping of human lung stem cell plasticity in fibrotic diseaseFoundation – Discovery Stage Research Projects$1,625,998
Dr. Brigitte N GompertsUniversity of California, Los AngelesOvercoming barriers for airway stem cell gene therapy for Cystic FibrosisFoundation – Discovery Stage Research Projects$1,472,858
Harold A. ChapmanUniversity of California, San FranciscoModulation of human alveolar stem cells to promote lung regeneration and avoid pulmonary fibrosisFoundation – Discovery Stage Research Projects$1,626,001
William van der TouwCelularity Inc.A phase I/II study of human placental hematopoietic stem cell derived natural killer cells (CYNK-001) for the treatment of adults with COVID-19Clinical Trial Stage Projects$315,000
Prof. John A ZaiaCity of Hope, Beckman Research InstituteEvaluation and Characterization of SARS-CoV-2 Antibody in Convalescent Volunteer Plasma Donors for Potential Therapeutic UseClinical Trial Stage Projects$999,999
Evan Y. SnyderSanford Burnham Prebys Medical Discovery InstituteUsing hiPSC-derived lung organoids, a clinically-relevant system, to validate & winnow a list of approved drugs that inhibit SARS-CoV-2 cytopathyDiscovery Research Projects$228,229
Preet M ChaudharyUniversity of Southern CaliforniaChimeric Antigen Receptor Targeting Spike Glycoprotein of SARS-cov2Discovery Research Projects$248,125
Dr Karen ChristmanUniversity of California, San DiegoPro-healing biomaterial for treating lung inflammation associated with COVID-19Discovery Research Projects$221,758
Dr. Gay Miriam CrooksUniversity of California, Los AngelesStem cell-based rapid identification of SARS-CoV-2 T cell epitopes and T cell receptors for therapeutic useDiscovery Research Projects$126,692
Dr. Brigitte N GompertsUniversity of California, Los AngelesIdentifying a lead compound for COVID19 using high throughput screening with lung stem cell organoidsDiscovery Research Projects$149,998
Dr. Brigitte N GompertsUniversity of California, Los AngelesStem Cells for Lung Diseases – Overcoming Barriers to Find New TherapiesConference – Lung Regenerative Medicine Workshop$44,548
Marco QuartaRubedo Life Sciences Inc.Pharmacological regenerative treatment of idiopathic pulmonary fibrosis targeting the senescent niche of lung progenitor cells.Quest – Discovery Stage Research Projects$1,450,876
Prof James S HagoodUniversity of California, San DiegoMesenchymal stem cell extracellular vesicles as therapy for pulmonary fibrosisQuest – Discovery Stage Research Projects$0
Dr. Matthew H PorteusStanford UniversityGenome Editing to Correct Cystic Fibrosis Mutations in Airway Stem CellsQuest – Discovery Stage Research Projects$1,968,456
Semil P ChoksiUniversity of California, San FranciscoGeneration of human airway stem cells by direct transcriptional reprogramming for disease modeling and regenerationInception – Discovery Stage Research Projects$238,408
John RamunasRejuvenation Technologies, Inc.Telomerase mRNA for short telomere related pulmonary fibrosisTherapeutic Translational Research Projects$3,984,942
Dr. Peter C. BelafskyUniversity of California, DavisTissue Engineered Recellularized Laryngotracheal ImplantsDisease Team Therapy Development III$3,181,162
Dr. Brigitte N GompertsUniversity of California, Los AngelesUsing human induced pluripotent stem cells to improve our understanding of Idiopathic Pulmonary FibrosisTissue Collection for Disease Modeling$811,231
Dr. Martin BirchallUniversity of California, DavisAirways for ChildrenDisease Team Therapy Planning I$19,800
Dr. Barry R StrippCedars-Sinai Medical CenterEpithelial progenitors and the stromal niche as therapeutic targets in lung diseaseResearch Leadership$4,841,830
Dr. Brigitte N GompertsUniversity of California, Los AngelesStem Cells in Lung CancerNew Faculty II$2,381,572
Total:
$25,937,483.39

CIRM respiratory disorders videos

Matthew Donne, UCSF – CIRM Stem Cell #SciencePitch

Youngtae Jeong, Stanford – CIRM Stem Cell #SciencePitch

Asaf Presente, UCSD – CIRM Stem Cell #SciencePitch

Find Out More:
Stem Cell FAQ | Stem Cell Videos | What We Fund