Looking for new therapeutic targets in OI – the OI NEWT study
Research type
Research Study
Full title
Developing novel approaches to the treatment of osteogenesis imperfecta (OI) in infants and children
IRAS ID
224666
Contact name
Nick Bishop
Contact email
Sponsor organisation
Sheffield Children's Hospital Foundation Trust
Duration of Study in the UK
1 years, 3 months, 30 days
Research summary
Osteogenesis imprefecta (OI) or brittle bone disease is a genetic disorder characterised by bones that break easily because the bone material is brittle and the structure of the bones is defective. Current medicines cannot affect the material itself, instead they increase the bone strength by making bones wider and 'filling in' the holes in the bone walls that weaken it.
The ultimate aim of any treatment is to cure the disease. Whilst we can’t yet do that, we propose to firstly assess a new approach to treatment that could improve bones and muscles in OI and secondly devise a system for assessing gene therapy methods that might eventually lead to a cure.
Recently, it was found that blocking a chemical called TGF beta had remarkable effects in terms of restoring the bony skeleton of mice with OI. This approach might represent a new way to treat humans with OI in the short term. As well as assessing this, we want to develop a system that allows us to test different gene therapy approaches.
Lots of children with OI have operations. Bone is often removed and discarded at that time. We will use blood taken through the drip that is always in place during an operation, skin from the wound edge and bone tissue that would otherwise be thrown away to measure TGFbeta and other relevant factors.
Some of the discarded bone tissue will be implanted in mice and allowed to grow over time. We can assess the system we are creating and determine whether we can use it for testing approaches to gene therapy – our ultimate aim is to cure OI, and we believe that this is a first step towards that goal.
REC name
Yorkshire & The Humber - Bradford Leeds Research Ethics Committee
REC reference
17/YH/0268
Date of REC Opinion
23 Aug 2017
REC opinion
Favourable Opinion