First 'in womb' stem cell trial announced

The donated stem cells should provide the correct instructions for growing bone.

PTI| Updated: Oct 12, 2015, 16:14 PM IST

London: The first clinical trial to inject foetal stem cells into babies still in the womb to lessen the symptoms of incurable brittle bone disease will begin in January next year.

The trial will be led by Sweden's Karolinska Institute and in the UK by Great Ormond Street Hospital and the stem cells will come from terminated pregnancies.

Officially called osteogenesis imperfecta, brittle bone disease affects around one in every 25,000 births.

It can be fatal with babies born with multiple fractures. Even those who survive face up to 15 bone fractures a year, brittle teeth, impaired hearing and growth problems.

The disease is caused by errors in the developing baby's DNA, that means the collagen supposed to give bone its structure is either missing or of poor quality.

It is hoped the stem cells, which are able to transform into a range of tissues, will lessen symptoms of incurable brittle bone disease.

The donated stem cells should provide the correct instructions for growing bone, 'BBC News' reported.

"This is a very serious disease. Our objective is to see if in utero (in the womb) stem cell therapy can ameliorate the condition and the number of fractures," Professor Lyn Chitty, from Great Ormond Street Hospital, said.

A type of stem cell which develops into healthy bone, cartilage and muscle will be infused directly into the affected foetuses.

Fifteen babies will have the infusion in the womb and again after they are born.

A further 15 will only have the treatment after birth and the number of fractures will be compared with untreated patients.

"It is the first in-man trial and, if successful, it will pave the way for other pre?natal treatments when parents have no other option," Dr Cecilia Gotherstrom, from the Karolinska Institute added.