Daily Management Review

First 'in womb' Stem Cell Trial in Clinical Setting to Begin


10/12/2015




First 'in womb' Stem Cell Trial in Clinical Setting to Begin
Come January of next year, the attempts to lessen the symptoms of incurable brittle bone disease through the injection of foetal stem cells into babies while they are still in the womb is to begin in the first of its kind clinical trial.
 
The stem cells for the trial would come from terminated pregnancies and the trial will be led by Sweden's Karolinska Institute and in the UK by Great Ormond Street Hospital. The donated stem cells should provide the correct instructions for growing bone.
 
The genetic testing to search for the defects that lead to the condition, will be carried out by Prof Lyn Chitty of the Great Ormond Street Hospital.
 
"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 said to the media.
 
"If we could reduce the fracture frequency, strengthen bone and improve growth it would have a huge impact," said Dr Cecilia Gotherstrom, from the Karolinska Institute.
 
Brittle bone disease affects around one in every 25,000 births and is technologically called osteogenesis imperfect.
 
It can be fatal with babies born with multiple fractures. For the babies that manage to scrape through the danger in the infant stage live with the risk of up to 15 bone fractures a year, brittle teeth, impaired hearing and growth problems.
 
Errors in the developing baby's DNA is the primary cause of the problem which essentially means that the collagen supposed to give bone its structure is either missing or of poor quality.
 
Stem cells that are able to transform into a range of tissues is hoped to lessen symptoms of incurable brittle bone disease. The donated stem cells should provide the correct instructions for growing bone.
 
Experts and researchers of the project claimed that the aim of the clinical trials is to see if in utero or in the womb, stem cell therapy can ameliorate the condition and the number of fractures.
 
The trials would entail the direct infusion of a type of stem cell which develops into healthy bone, cartilage and muscle into the affected foetuses. The infusion would be conducted on Fifteen babies while they are in the womb and again after they are born.
 
A type of stem cell which develops into healthy bone, cartilage and muscle will be infused directly into the affected foetuses.
 
The number of fractures of another set of 15 children who would be given the treatment after birth could be compared with the ones administered the injection in the womb.
 
The first in-man trial of its type, the success of this project would create a way for other pre-natal treatments when parents have no other option.
 
The researchers hope that the injected cells would be able to transform into a range of tissues.
 
Research has shown that stem cells transplantation has the potential to ease symptoms of brittleness in children. If this process is started at an even earlier stage when the bone is developing and growing rapidly, the results can be more effective, say researchers.
 
(Source:www.thedailystar.net) 






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