Grant: Operating Grant: Clinical Trials Projects
Ottawa Hospital Research Institute
$2,300,649.00
Helping Underdeveloped Lungs with Cells (HULC-2) - A Randomized Controlled Trial to test the efficacy of umbilical cord-derived mesenchymal stromal cells (UC-MSC) in reducing respiratory morbidity in preterm infants at risk of bronchopulmonary dysplasia (BPD)
FY 2023-24
Summary
Bronchopulmonary dysplasia (BPD) is a chronic lung disease that follows ventilator and oxygen treatment for acute failure to breathe in babies born too soon (before 28 weeks of gestation instead of 40). BPD is a major complication of prematurity with a short- and long-term burden for infants and their families. Damage to the still developing lung stops the normal formation of the alveoli (the air sacs in the lung that allows the uptake of oxygen and release of waste carbon dioxide). Currently there is no treatment for this disease. Our laboratory is seeking new medications to either protect or heal the damaged lung.
Our body contains stem cells, a pool of cells that have the ability to become any type of cell (i.e. bone, muscle, liver or blood cell, etc.). Stem cells can be found in the bone marrow but also in the umbilical cord (UC) of babies. The UC is discarded after birth but can be safely collected without risk to the mother or the baby. Our lab was one of the first to show that a specific type of cells from the UC, called mesenchymal stromal cell (MSC) can repair lungs in laboratory models of BPD. Extensive studies have led to Health Canada approval for our Phase 1 clinical trial that showed the safety of a single intravenous injection of UC-MSCs in extreme preterm infants at high risk of developing BPD.
Here, we propose the next logical step: a Phase 2 trial to test the efficacy and safety of UC-MSCs compared to placebo. This will be a Canadian multicentre trial enrolling 168 patients to determine if UC-MSC can reduce the time babies spent on a ventilator and whether this leads to better outcomes for premature babies. Our findings will be relevant to the treatment of other lung diseases that currently lack efficient treatments such as COVID-19 and other pandemic agent-related acute respiratory distress syndrome.
Department
Program
Operating Grant: Clinical Trials Projects
Recipient
Location
Ontario, Canada
Recipient Type
Other
Type
Grant
Source
CIHR Funding Decisions Database
As published
Id
497369
Name
Thebaud Bernard
Region
Ontario
Country
Canada
Orgtype
Institution Paid; Research Institution
Instname2
Human Development, Child and Youth Health
Theme2
Clinical
Prcname2
Clinical Trials Projects - Full Application
Projecttitle
Helping Underdeveloped Lungs with Cells (HULC-2) - A Randomized Controlled Trial to test the efficacy of umbilical cord-derived mesenchymal stromal cells (UC-MSC) in reducing respiratory morbidity in preterm infants at risk of bronchopulmonary dysplasia (BPD)
Programtype2
Operating Grants
Orgnameinp2
Ottawa Hospital Research Institute
Cihramount2
$2,300,649
Cihrequipment2
$0
Programname2
Operating Grant: Clinical Trials Projects
Keyworddelim
Clinical Trial; Lung Injury; Mesenchymal Stromal Cells; Newborn; Outcome; Preterm Birth; Regenerative Medicine; Stem Cells
Abstract
Bronchopulmonary dysplasia (BPD) is a chronic lung disease that follows ventilator and oxygen treatment for acute failure to breathe in babies born too soon (before 28 weeks of gestation instead of 40). BPD is a major complication of prematurity with a short- and long-term burden for infants and their families. Damage to the still developing lung stops the normal formation of the alveoli (the air sacs in the lung that allows the uptake of oxygen and release of waste carbon dioxide). Currently there is no treatment for this disease. Our laboratory is seeking new medications to either protect or heal the damaged lung.
Our body contains stem cells, a pool of cells that have the ability to become any type of cell (i.e. bone, muscle, liver or blood cell, etc.). Stem cells can be found in the bone marrow but also in the umbilical cord (UC) of babies. The UC is discarded after birth but can be safely collected without risk to the mother or the baby. Our lab was one of the first to show that a specific type of cells from the UC, called mesenchymal stromal cell (MSC) can repair lungs in laboratory models of BPD. Extensive studies have led to Health Canada approval for our Phase 1 clinical trial that showed the safety of a single intravenous injection of UC-MSCs in extreme preterm infants at high risk of developing BPD.
Here, we propose the next logical step: a Phase 2 trial to test the efficacy and safety of UC-MSCs compared to placebo. This will be a Canadian multicentre trial enrolling 168 patients to determine if UC-MSC can reduce the time babies spent on a ventilator and whether this leads to better outcomes for premature babies. Our findings will be relevant to the treatment of other lung diseases that currently lack efficient treatments such as COVID-19 and other pandemic agent-related acute respiratory distress syndrome.
Competitiondate
202311
Approvedterm2
3 yrs 0 mth
Pinamesdelim
Thebaud, Bernard; Robeson, Paula; Fergusson, Dean A
Approvedterm
3 ans 0 mois
Cihrcontribution2
~ $2,300,649~ $0~