A girl who was critically ill with heart failure is doing well after receiving an experimental treatment made from her sister’s umbilical cord stem cells, in the first case of its kind.
The girl, from Germany, has an inherited form of pulmonary arterial hypertension, or high blood pressure in the arteries of the lungs. This causes malformation of the blood vessels in the lungs, leading to progressive and usually fatal heart failure. Doctors had recommended that the girl, who is now 6, have a lung transplant at 3 years old, a procedure that is usually carried out on children who have less than a year to live.
Instead, she was given the experimental treatment, in which Georg Hansmann at Hannover Medical School in Germany and his colleagues harnessed stem cells from the umbilical cord of the girl’s sister.
The cells were grown in a dish. Periodically, the nutrient liquid they were bathed in was changed, and the old liquid was stored. Three years ago, it was infused into blood vessels in the girl’s lungs and heart over six months.
The girl, who was breathless at rest and could only walk slowly, improved over the following months. She now has no limits to her exercise capacity. She also grew 10 centimetres within the first three months of treatment, having had no growth in height or weight in the preceding year.
However, she still has high blood pressure in her lungs and may need further treatment, says Hansmann. Stem cells have the potential to grow into different kinds of tissue and are being tested in many experimental treatments. They can be obtained in small quantities from various parts of the body and made in the laboratory from skin cells.
Stem cell treatments usually involve putting the cells into someone’s body, which can cause an immune reaction. In the girl’s case, the cells were grown in a dish, where they released biochemicals into the liquid they were bathed in. It is these biochemicals that seem to promote tissue healing.
The girl’s treatment used mesenchymal stem cells, which are involved in making and repairing skeletal tissues.
These cells have been tested to see if they could repair heart muscle after heart attacks. While this didn’t lead to lasting benefits and studies found no trace of the transplanted cells in the heart muscle, some recipients had short-term improvements, suggesting that the cells released chemicals that promote healing.
The girl had been given two standard medicines for her condition before the stem cell treatment, which may have helped, says Martin Wilkins at Imperial College London.
But when Hansmann’s team investigated samples of the stem cell liquid she received, they found high levels of biochemicals that are thought to promote healing while suppressing inflammation, including prostaglandin E2.
This biochemical tends to be rapidly broken down in the body, so other compounds may be having an effect, says Wilkins.
“There does appear to have been an improvement both in her biochemical [measurements] and in her functional capacity,” he says. “It’s reasonable to assume there’s something going on here”.
