Grant: Foundation Grant

<html> <body> <p> <b>Transformative research that matters.</b> Within 7 years, my program will implement stem cell-based therapies to radically improve the outcome of life-threatening and debilitating neonatal lung diseases.<br> <br>At no other stage in life is the impact of disability-adjusted life years higher than in the neonatal period. Effective interventions at this stage provide exceptional value. Extreme prematurity accounts for &gt;85% of all perinatal complications and death. The chronic lung disease bronchopulmonary dysplasia (BPD) is the most frequent sequelae of prematurity. BPD contributes to life-long respiratory and neurological impairment. The economic burden is &gt;$25B/y of which 10% is just for treating infants with BPD. Pulmonary hypertension (PHT), due to restricted lung vascular growth in BPD and congenital diaphragmatic hernia (CDH), doubles the risk of death. Surfactant protein deficiencies are lethal genetic mutations and account for 10% of all childhood interstitial lung diseases. Our pioneering work suggests stem cells as a transformative approach for these lung disorders. Through 3 core activities, we will now move these therapies into patients:<br> <br> <b>CORE ACTIVITY 1: Resident lung stem/progenitor cells in development and disease</b> <br>Exhaustion/dysfunction of resident stem cells may explain the inability of the lung to repair itself. Knowledge on endogenous repair cells will provide new strategies for lung repair.<br> <br> <b>CORE ACTIVITY 2:</b> <b>Next generation cell therapies for neonatal lung diseases</b> <br>We will focus on 3 cell products that each targets an unmet need:<br>1. Umbilical cord (UC)-derived mesenchymal stromal cells (MSCs) for the prevention of BPD;<br>2. UC blood-derived Endothelial Colony Forming Cells (ECFCs) for neonatal PHT;<br>3. Induced pluripotent stem cells (iPSCs) for surfactant protein deficiencies.<br>We will deliver proof-of-concept, preclinical testing in relevant disease models and novel cell manufacturing strategies to produce superior cell therapies for clinical trials.<br> <br> <b>CORE ACTIVITY 3:</b> <b>Clinical trials to test the safety and efficacy of cell therapies</b> <br>Cell products will be tested in early phase clinical trials. <i>In some cases</i>, we are close to completing the validation and will initiate the world&rsquo;s 1^st cord MSC trial for BPD. <i>In another example</i>, a vascular ECFC therapy is in the early preclinical stage and will be translated into a potentially lifesaving therapy for neonatal PHT. <i>Our 3^rd project</i> will test iPSC-derived cell therapies to cure lethal genetic lung diseases.<br> <br> <b>Dramatic knowledge and healthcare advancement.</b> We will capitalize on the tremendous potential of stem cells to radically improve the outcome of patients with life-threatening lung diseases that currently lack treatments. Knowledge, technology and cell products developed in this program will also impact cystic fibrosis, asthma or COPD, that combined, represent the 3rd killer in humans with an economic burden of $12B/year.<br> <br>To ensure the success of my ambitious program, I have secured national and international collaborators and networks with complementary <b>core expertise</b> in stem cell biology and clinical trials.<br> <br> <b>Unparalleled and exciting training ground.</b> My multidisciplinary program will contribute to capacity building in the bourgeoning field of regenerative medicine. Exposure to the full breadth of translational research on novel, game-changing cell therapies from bench discovery to clinical trials will perfectly launch my trainees&rsquo; careers in academia and non-academic health-related fields.</p> </body> </html>