The Hamad Medical Corporation has been identified as the regional centre of excellence within the Gulf for the treatment of severe liver diseases and other inherited metabolic disorders affecting newborns and infants.
The Corporation, which has signed an agreement with specialists in regenerative medicine at the Germany-based University Children’s Hospital in Heidelberg and German Biotech Company – Cytonet, will identify and include children suffering from disorders, especially urea cycle defect, in Qatar into an ongoing liver cell therapy study programme SELICA (Safety and Efficacy of Liver Cell Application).
University Children’s Hospital head professor Georg Hoffmann is the principal investigator of the clinical trial.
Another study is just starting in the US in co-operation with the Urea Cycle Disorder Consortium while a third study for the treatment of further inherited metabolic disorders is already in preparation in Europe.
The joint venture between the three institutions was announced following a launch event on October 11 held alongside a Newborn Screening workshop, the third one to be held in the country and one of the few organised in the GCC.
Under the agreement, the clinical trial will be conducted apart from facilitating the treatment of children who suffer from urea cycle defect in Germany.
Urea cycle disorders is a genetic disorder caused by deficiency of enzymes in the urea cycle, which is responsible for removing ammonia from the blood stream thereby cleaning toxins.
Currently, the only available cure for the illness is the transplantation of a whole liver or a liver lobe, however, the treatment was highly problematic for very young children and neonatal patients and also, suitable organs available for transplantation are very rare.
Speaking at a dinner organised to announce the co-operation, HMC managing director Dr Hanan al-Kuwari said the initiative was one of the many medical and healthcare projects the Corporation was working on that will raise the health standards in the country and lead to a better quality of life for the population.
About seven years ago, the HMC in co-operation with the University Children’s Hospital of Heidelberg, started to develop the first comprehensive and extended newborn screening programme (NBS) for metabolic and endocrine disorders in the whole of Middle East.
So far, over 90,000 babies have been screened with around 205 newborns found with different types of disorders.
NBS director Dr Hilal al-Rifai gave an overview of the programme at the launching of the study.He explained that apart from a liver transplantation, which was often not possible, especially in newborns, there were no alternative curative treatment options for urea cycle defects.
Gulf Times