journal in pedia

8/13/2019 Journal in Pedia

1/6

Brittle-Bone Babies Helped by Fetal Stem Cell Grafts

Dec. 16, 2013 Osteogeneis imperfecta (OI) is a congenital bone disease that causes

stunted growth and repeated, painful fracturing. Ultrasound scans can reveal fractures

already in the fetus, and now an international team of researchers from Sweden, Singapore

and Taiwan have treated two babies in utero by injecting bone-forming stem cells. The

longitudinal results of the treatment are published in the journal Stem Cells Translational

Medicine.

The babies were treated withmesenchymal stem cells, connective tissue cells that can form

and improve bone tissue. The stem cells were extracted from the livers of donors and

although they were completely unmatched genetically, there was no rejection and the

transplanted cells were accepted as self.

Back in 2005, a paper was published from Karolinska Institutet in Sweden describing how

stem cells were given to a female fetus. The present study describes how the girl suffered a

large number of fractures and developed scoliosis up to the age of eight, whereupon the

researchers decided to give her a fresh stem cell graft from the same donor. For the next

two years the girl suffered no new fractures and improved her growth rate. Today she

takes dance lessons and participates more in PE at school.

Another unborn baby with OI, a girl from Taiwan, was also givenstem cell

transplantation by the Karolinska Institutet team and their colleagues from Singapore. The

girl subsequently followed a normal and fracture-free growth trajectory until the age of

one, when it levelled off. She was given a fresh stem cell treatment and her growth


2/6

resumed. The girl started to walk and has since not suffered any new fractures. Today she

is four years old.

"We believe that the stem cells have helped to relieve the disease since none of the children

broke bones for a period following the grafts, and both increased their growth rate," says

study leader Dr Cecilia Gtherstrm, researcher at Karolinska Institutet's Department of

Clinical Sciences, Intervention and Technology. "Today, the children are doing much better

than if the transplantations had not been given. OI is a very rare disease and lacks effective

treatment, and a combined international effort is needed to examine whether stem cell

grafts can alleviate the disease."

The researchers have also identified a patient, a boy from Canada, who was born with OI

caused by exactly the same mutation as the Swedish girl had. The boy was not given

stemcell therapy and was born with severe and widespread bone damage, including

numerous fractures and kyphosis of the thoracic vertebrae, which causes such over-

curvature of the spine that it impairs breathing. The boy died of pneumonia within his first

5 months.

Journal Reference:

1. Cecilia Gtherstrm, Magnus Westgren, S W Steven Shaw, Eva strm, Arijit Biswas,Peter H Byers, Citra N Z Mattar, Gail E Graham, Jahan Taslimi, Uwe Ewald, Nicholas M

Fisk, Allen E J Yeoh, Ju-Li Lin, Po-Jen Cheng, Mahesh Choolani, Katarina Le Blanc andJerry K Y Chan. Pre and postnatal transplantation of fetal mesenchymal stem cells

in osteogenesis imperfecta: a two-center experience. Stem Cells Translational

Medicine, December 2013


3/6

Fetal stem cell grafts successfully help brittle-bone babiesMarie Ellis

In an international collaboration, researchers from Sweden, Singapore and Taiwan

successfully treated two babies with a congenital bone disease that causes stunted

growth and repeated fracturing by injecting them in utero with bone-forming stem

cells.

Results of their longitudinal study have been published in the journal Stem Cells

Translational Medicine.

Osteogenesis imperfecta (OI) not only stunts the growth of those who suffer from this

disease, but the repeatedfracturesit causes are painful.

However, this condition can be recognized prenatally with anultrasound,so researchers

from the Karolinska Institutet in Sweden published a paper in 2005 detailing how

mesenchymalstem cells- connective tissue cells that form and improve bone tissue - were

given to a female fetus in Sweden.

These stem cells were taken from the livers of donors, and the researchers note that

though the donors and recipients were not genetically matched, there was no

rejection.

In this recent study, the team explains how the girl experienced several fractures and

hadscoliosisby age 8. At this point, the researchers gave her a fresh stem cell graft from

the same donor as before.

For the following 2 years, the child did not experience any new fractures and her growth

rate improved. Today, the researchers say, she participates in dance lessons and gym class

at school.
http://www.medicalnewstoday.com/articles/173312.phphttp://www.medicalnewstoday.com/articles/173312.phphttp://www.medicalnewstoday.com/articles/173312.phphttp://www.medicalnewstoday.com/articles/245491.phphttp://www.medicalnewstoday.com/articles/245491.phphttp://www.medicalnewstoday.com/info/stem_cell/http://www.medicalnewstoday.com/info/stem_cell/http://www.medicalnewstoday.com/info/stem_cell/http://www.medicalnewstoday.com/articles/190940.phphttp://www.medicalnewstoday.com/articles/190940.phphttp://www.medicalnewstoday.com/articles/190940.phphttp://www.medicalnewstoday.com/articles/190940.phphttp://www.medicalnewstoday.com/info/stem_cell/http://www.medicalnewstoday.com/articles/245491.phphttp://www.medicalnewstoday.com/articles/173312.php


4/6


5/6

Born with severe widespread bone damage, this boy was not given stem cell therapy like

the girls were, and he experienced numerous fractures and kyphosis of the thoracic

vertebrae - a condition that causes an extreme curvature of the spine, impairing breathing.

The untreated boy died within his first 5 months frompneumonia,the investigators say.

Although the researchers say their findings suggest the stem cell therapy treatment

"appears safe and is of likely clinical benefit," they add that "the limited experience

to date means that it is not possible to be conclusive and that further studies are

required."
http://www.medicalnewstoday.com/articles/151632.phphttp://www.medicalnewstoday.com/articles/151632.phphttp://www.medicalnewstoday.com/articles/151632.phphttp://www.medicalnewstoday.com/articles/151632.php


6/6

I. SummaryII. ReactionIII. Recommendation

journal in pedia

Documents