The world’s first in womb stem cell clinical trial will begin in January. The trial, which involves foetal stem cells from terminated pregnancies being injected into babies in the womb, will be conducted by the UK’s Great Ormond Street Hospital and Sweden’s Karolinska Institute.
In a joint statement, the researchers say it is hoped the cells, which are able to transform into a range of tissues, will reduce the symptoms of incurable brittle bone disease. The disease officially called osteogenesis imperfecta, affects around one in every 25,000 new born babies and can be fatal for babies born with multiple fractures.
Great Ormond Street Hospital’s Prof Lyn Chitty says even those babies with the disease that survive face a life of as many as 15 bone fractures each year, brittle teeth, growth problems and impaired hearing
She says the disease is caused by mistakes in the developing baby’s DNA, resulting in the collagen that is meant to give bones their strength and structure being of poor quality or totally missing. It is hoped the donated and injected stem cells will “provide the correct instructions for growing bone”.
“This is a very serious disease. Our objective is to see if in utero stem cell therapy can ameliorate the condition and the number of fractures.”
She says a stem cell which develops into healthy bone, cartilage and muscle will be injected directly into 15 babies in the womb and then again after they are born.
Dr. Cecilia Gotherstrom from the Karolinska Institute says, “If we could reduce the fracture frequency, strengthen bone and improve growth it would have a huge impact.”
In-the-womb foetal stem cell transplants have been tried in two cases of osteogenesis imperfecta. But without a proper clinical trial, it is impossible to know how effective the therapy is. Dr. Gotherstrom says: “It is the first in-man trial and, if successful, it will pave the way for other prenatal treatments when parents have no other option.”
The trial will begin in January and will recruit patients for two years.
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