Injecting foetal stem cells into unborn babies has given hope the procedure will lessen symptoms of incurable brittle bone disease in newborns.
A world-first clinical trial, starting in January, will be led by Sweden’s Karolinska Institute and in the UK by Great Ormond Street Hospital. The donated stem cells will come from terminated pregnancies and provide the correct instructions for growing bone.
Brittle bone disease, or osteogenesis imperfecta, affects one in every 25,000 births and can be fatal with babies born with multiple fractures. Those who survive can have 15 bone fractures a year, brittle teeth, impaired hearing and growth problems.
Prof Lyn Chitty, from Great Ormond Street Hospital, will carry out genetic testing to search for the defects that lead to the condition.
“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,” she tells BBC.
Fifteen babies will have the infusion in the womb and again after they are born. A further 15 will have the treatment after birth and the number of fractures will be compared with untreated patients.
Born with broken arms and a fracture in his spine, Adam Reynolds, 21, says his main problem growing up was “learning to be sensible” when “as a kid you just want to run around and have fun with mates”.
He cannot keep track of how many times he’s broken his bones, but tells BBC the figure is somewhere between 30 and 40.
“Day-to-day life is awkward. If someone stubs their toe on a table they go ‘ow!’, for me it’s ‘did I break my toe’?” he says. “The idea of a cure coming out or something to help at such an early age is just fantastic news.”
Dr Cecilia Gotherstrom, from the Karolinska Institute, says reducing the fracture frequency, strengthening bone and improving growth will have a huge impact.
“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,” she says.
The first infusion will take place 20 to 34 weeks into the pregnancy. The trial will start in January and will recruit patients for two years.
(via BBC, image via Adam Reynolds)