FDA Approves Pneumostem Clinical Trial for Bronchopulmonary Dysplasia


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.

Big Strides in Stem Cell Treatments for Neonatal Lung Diseases


Bernard Thébaud works at the Ottawa Hospital Research Institute (OHRI) and Children’s Hospital of Eastern Ontario (CHEO), and is also a member of the Ottawa Stem Cell Initiative. Dr. Thébaud has proposed a new therapy that utilizes umbilical cord stem cells to treat a lung disease called bronchopulmonary dysplasia (BPD), which was previously thought to be untreatable.

Thébaud described BPD in this way: “BPD is a lung disease described 45 years ago in which we have made zero progress. And now, with these cord-derived stem cells there is a true potential for a major breakthrough. I am confident that we have the talent and the tools here at CHEO and OHRI to find a treatment for BPD. These findings published today are helping us get there.”

Every year, BPD affects ~10,000 premature newborns in Canada and the US. The lungs of infants with BPD are not developed enough to function properly, and consequently the baby has to be placed on a ventilator in order to receive sufficient quantities of oxygen. Mechanical respirators, however, are very hard on such young, friable lungs, and the lungs then to fray and this prevents them from developing properly. The longer the baby stays in the neonatal intensive care unit, the greater the degree of multiorgan damage (retina, kidneys, and the brain). Therefore, the baby needs oxygen to survive, but the very act of giving them oxygen eventually hastens their death.

Thébaud’s research team used new-born rats that were given oxygen soon after their premature birth. Some were given stem cell treatments and others were not. These experiments produced five new findings:

1) Mesenchymal stem cells (MSCs) from human umbilical cord can protect the lungs when injected into the lungs as the animals were put on oxygen.
2) MSCs had a tendency to stimulate repair of the damaged lungs when injected two weeks after the animals were put on oxygen.
3) The medium in which the MSCs were grown (conditioned medium) was injected into the lungs instead of the cells, this medium had the same reparative and protective effects as the cells themselves.  This suggests that it is the cocktail of growth factors and other supportive molecules secreted by the MSCs that provide their healing properties.  Such a mechanism, in which the cells secrete molecules that affect nearby cells and tissue, is known as a “paracrine” mechanism.
4) When examined six months after treatment (the equivalent of 40 human years), the treated animals had better exercise performance and more normal lung structure.
5) MSC administration did not adversely affect the long-term health of the laboratory animals. None of the MSC-treated animals had any tumors and MSCs given to control animals that did not have BPD were also normal six months later.

Thébaud would like to conduct a pilot clinical (Phase I) study within two years with around 20 human patients in order to determine if this treatment is feasible and safe. If the treatment turns out to be safe, Thébaud would like to initiate a randomized controlled (Phase II) clinical trial.

See Maria Pierro et al., “Short-term, long-term and paracrine effect of human umbilical cord-derived stem cells in lung injury prevention and repair in experimental bronchopulmonary dysplasia,” Thorax 2012: DOI:10.1136/thoraxjnl-2012-202323.