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Babies diagnosed with a hole in their diaphragm could soon benefit from the research of Dario Fauza, MD and his team who are set to apply to the US Food and Drug Administration (FDA) for permission to run the first human trial using fetal stem cells.
If given the go ahead the trial will take place at the Children’s Hospital Boston, Massachusetts.
That’s where Fauza and team have developed their research during the past decade and successfully tested their “minimally invasive” treatment on large animal models - a trial that involved 27 sheep. To test the effectiveness of the amniotic stem cells some of the sheep received a diaphragm patch with amniotic stem cells and some without. The results were then compared.
“The message is that having cells in the graft makes a significant difference,” Fauza told New Scientist.
Following this success the team are now seeking FDA approval for their groundbreaking research to be extended to a trial on twenty babies born with a hole in their diaphragm.
“We’re just waiting for the green light,” Fauza told New Scientist. “If approved, it will be the first trial of this strategy, and the first based on amniotic fetal stem cells.”
A congenital diaphragmatic hernia (CDH) or hole in the diaphragm as they’re more commonly known is often diagnosed during routine ultrasounds. Whilst all babies have a small hole in the diaphragm during the early weeks of pregnancy the majority of those naturally close by the end of the first three months.
Holes that don’t heal fall in to two categories of CDH: a Bochdalek hernia, which is an opening in the back of the diaphragm and a Morgagni hernia, a rarer type that causes a hole in the front of the diaphragm.
Both types result in the stomach, intestines, liver or spleen moving through the hole and in to the chest cavity. This can affect the development of the lung on the affected side and growth of the other lung. Babies suffering from a hole in their diaphragm will need surgery once born and suffer with lung problems and difficulties in breathing, a problem that can remain with them throughout their life.
If Fauza’s bid is successful the trial will begin taking stem cells from the amniotic fluid during pregnancy. These cells will then be grown in a lab, allowing the pregnancy to continue as normal. Once the baby is born, the expanded tissue will be grafted over the hole in the diaphragm during surgery.
The New Scientist article explains: “Fetuses normally shed numerous cells in the surrounding amniotic fluid. Of these about 1 per cent are amniotic stem cells.”
These stem cells are called “amniotic mesenchymal (aMSCs) stem cells,” the New Scientist say and they “multiply twice as fast as any other known type of stem cell, so once collected they can generate enough material to build spare tissue before the baby is born.”
Amniocentesis is the process that extracts cells from the amniotic sac protecting the fetus. The procedure usually takes place between the 15th and 20th week of pregnancy and is more commonly used to test for genetic abnormalities such as Down syndrome or other fetal problems. Concerns are that an amniocentesis can potentially lead to a miscarriage or other complications.
The current treatment for a congenital diaphragmatic hernia is a Teflon diaphragm patch. These grafts have a tendency to detach, resulting in the need for further operations to repair the damage.
With a stem cell implant Fauza believes that the long-term results will be more encouraging.
“The hope is that patches made from a baby’s own tissue will become a permanent part of the diaphragm, growing alongside it.”
Other researchers in the field are hopeful that Fauza’s trial will go ahead, the New Scientist say. “Fauza has been doing a lot on the tissue manufacturing side, and his work is fantastic,” says Anna David of University College London, who is co-leader of a team developing potential treatments for blood disorders based on amniotic fetal stem cells.”
Fauza and his team have also started work on research that explains why “amniotic stem cells may be one of the key factors explaining why wounds heal faster and better inside than outside the womb.”
“It’s biological validation that these cells are already being used by nature to repair things,” Fauza says, “in this case, fetal wounds.”
Amniotic stem cells are the future, Fauza says. It’s his belief that there will come a time when amniotic stem cells are routinely stored for use throughout our lifetime.
| Relevant Links |
| Amniocentesis (Wiki) |
| Using fetal cells to fix birth defects (YouTube) |
| Children’s Hospital Boston |