MEDIPOST America Inc. has announced that the US Food and Drug Administration (USFDA) has approved their product Pneumostem for a Phase 1/2 clinical trial. This Phase 1/2 trial will assess the safety and efficacy of Pneumostem on prematurely born infants who are at high-risk of developing Bronchopulmonary Dysplasia.
Bronchopulmonary dysplasia (BPD) is a serious lung condition that affects infants. BPD usually affects premature infants who need oxygen given through nasal prongs, a mask, or a breathing tube in order to properly breathe.
Most infants who develop BPD are born more than 10 weeks before their due dates and weigh less than 2 pounds (about 1,000 grams) at birth, and have breathing problems. Respiratory infections that hit before or shortly after birth also can contribute to BPD.
Some infants who suffer from BPD may need long-term breathing support from breathing (NCPAP) machines or ventilators. BPD is the leading cause of mortality and severe complications in premature infants. Currently there is no approved therapies or drugs exist for BPD. This pneumostem trial is expected to draw global attention in the field of neonatal medicine, since it would provide a potential treatment for BPD where none presently exists.
Pneumostem is an off-the-shelf product made from human Umbilical Cord Blood-derived Mesenchymal Stem Cells (hUCB-MSCs). hUCB-MSCs show a terrific ability to grow in the laboratory and can also differentiate into multiple types of cells or tissues. They are immune-privileged and thus if they are used in patients other than from whom they are isolated, they do not cause adverse immune reactions. hUCB-MSCs harvested from cord blood show the lowest levels of immunogenicity compared to those by other types of adult stem cells. Thus, instead of provoking immunogenicity, they rather modulate the adverse immune reactions within the host, which makes hUCB-MSCs an ideal candidates for mass-producible stem cell drug for allogeneic use. These cells seem to facilitate regeneration of lung tissue and suppress the inflammatory responses in the lungs of premature infants.
Pneumostem has received Orphan Drug designation in Korea by the Ministry of Food and Drug Safety (MFDS) and the Korean Phase 2 study is 80% complete. The US FDA also granted Orphan Drug designation for Pneumostem demonstrating its medical value and commercial potential.
Presently, MEDIPOST America is rapidly moving begin this Pneumostem trial in the U.S. At the same time, Medipost will continue its licensing and technology transfer negotiations with multinational pharmaceutical companies.
The approval of this Pneumostem clinical trial by the US FDA, whose regulation of medicinal products is very strict (including stem cell products), might boost clinical trial approvals in other European and Asian countries.
Clinical development of Pneumostem was partly supported by Translational Stem Cell & Regenerative Medicine Consortium grant as a part of Public Health and Medicinal Technology R&D Project funded by the Korea Ministry of Health & Welfare and the Korea Health Industry Development Institute.