Qatar to become liver cell therapy regional hub by 2013.
The ground-breaking research on the liver cell therapy is currently being developed by the Cytonet in partnership with the University Hospital of Heidelberg. The Cytonet Group has chosen Qatar as its strategic partner and reference centre in the Middle East. The team engaged in this project is now about to wrap up its ongoing clinical studies and expects to receive approval from the European Medicines Agency by the end of next year and from the US Food and Drug Administration by 2013.
As part of the agreement, the Hamad Medical Corporation (HMC) will assist in the research by identifying children who require the treatment. A select group of physicians will be trained in Heidelberg to make proper use of the liver cell therapy.
Speaking to the media on the sidelines of the Newborn Screening Workshop organised by the HMC on Saturday, Cytonet Managing Director Wolfgang Rudinger said, "The liver cell therapy is a new therapeutic approach which can be used for severe liver disorders. We have started clinical experiments to test the effectiveness of this therapy in newborn children with inherited metabolic disorders, defect of liver metabolism and the first indication of urea cycle disorder. It could be a future therapy for metabolic disorders, acute as well as chronic liver failure. The aim is to do this exclusively in Doha and to put the HMC in a position where it can invite children from other countries in the GCC region for treatment here." Speaking about the new therapeutic procedure, Rudinger said, "It is done by infusing functional human hepatocyte into the baby's liver to substitute the missing enzyme activities inside. It's a very simple and non-invasive method. To introduce the liver cell therapy at HMC, two issues have to be addressed: setting up of its own storage unit for liver cells where human liver cells can be kept stored for years frozen in liquid nitrogen at a temperature of approximately -500 degree Celsius and training of a medical personnel fully capable of handling the cutting-edge treatment technique." Rudinger also said that liver cells would be provided to Qatar by the Cytonet and training of the HMC staff would be done in Heidelberg.
Director of Qatar Newborn Screening Programmes and Chief of Staff at the Women's Hospital Dr Hilal al Rifai said that urea cycle disorder was a rare disease with just five patients being diagnosed with the condition in Qatar. They all died because there was no treatment for the diseaseworldwide.
"The occurrence of the disease is in the ratio of one in every 8,500 babies with one case being diagnosed in Qatar each year." He cited the case of Baby Fatima, who underwent a successful liver cell therapy and organ transplant. The family of baby Fatma had two other babies who died due to the fatal disease.
"With the discovery of the liver cell therapy, the next child diagnosed with the disease here will, hopefully, not require any surgery and will be treated only with the injection of liver cells. Risk involved in this method of treatment is minimal as compared with the surgical transplantation. It's a breakthrough advance in medical science," he explained.
According to Professor Georg Hoffmann, Head of Department of General Paediatrics at the University Hospital of Heidelberg, clinical studies for the research are conducted in Germany and the United States.
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