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26 Oct 2015

First Ever Stem Cell Treatment for Babies Inside the Womb

In UK, a new stem cell trial begins next year for treating fetuses inside the uterus affected with brittle bone disorder. This is the first trial in which stem cells will be transplanted into babies who are still inside the womb of the mother. This clinical trial is specially designed to study and treat brittle bone disorder, Osteogenesis imperfecta which causes multiple bone fractures and growth issues.

What is Osteogenesis Imperfecta?

Also known as brittle bone disease, it affects 1 in every 25,000 births. This condition is caused by errors in the baby’s DNA which results in the poor development of collagen. In developing babies, collagen forms the structure of the bone and gives strength which may be missing or of poor quality in patients suffering from this condition. The affected children are usually born with multiple broken bones and after birth they may often experience regular fractures, growth irregularities, brittle teeth, hearing loss etc.

Adam’s Experience with Brittle Bone Disease

Adam Reynolds from Hampshire was born with spinal cord fracture and broken arms due to brittle bone disease. While growing up, Adam has broken his bones for about 20-30 times and says he can’t keep track of the number of bone fractures that he had experienced over the years. In 2009, he had to undergo 12 surgeries in six years to fix his broken leg. Now, at the age of 21, he feels that the disorder has never held him back in life. He has successfully graduated from college with first class honours degree in accounting and finance.

Stem Cells to Treat Brittle Bone Disease

Stem cells are capable of renewing and repairing the damaged tissue. However in this case, the stem cells are expected to correct the errors of DNA by providing the right instruction for the bones to grow. This improves the bone strength and minimizes the bone fractures in babies. The donated stem cells are available from terminated pregnancies, thus the usage of these cells do not pose any ethical issues. The risk of transplant rejection is also considered to be very low.

Stem Cell Therapy for In Utero Babies

The objective of the trial is to see if stem cells infused into the fetus can help to minimize the effect of the disease. About 15 affected babies will be identified using genetic testing for the trial that begins next year. The stem cells will be infused to the babies before and after birth while 15 other babies will have the treatment only after birth to understand the effectiveness of ‘In utero’ stem cell infusion.

The researchers hope that this treatment could reduce the frequency of bone fractures, strengthen the bones and improve their growth. In-the-womb stem cell transplant is expected to give better results as they are given when the bone is developing and growing rapidly. If successful, this therapy could pave way for the treatment of muscular dystrophy and other bone disorders in future.



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