Breakthrough Regenerative Therapy for Newborns with Metabolic Disorders in Qatar

Breakthrough Regenerative Therapy for Newborns with Metabolic Disorders in Qatar
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Published October 12th, 2010 - 14:03 GMT

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Hamad Medical Corporation
,
Cytonet GmbH & Co. KG
,
Hilal Al Rifai
,
Georg Hoffmann
,
Wolfgang Rüdinger
,
Michael J. Deissner

Hamad Medical Corporation (HMC), Qatar's leading healthcare provider has partnered with the University Children's Hospital in Heidelberg and German biotech company Cytonet GmbH & Co. KG to establish an agreement to conduct the clinical trial SELICA (Safety and Efficacy of Liver Cell Application).

The joint venture between Hamad and the specialists in regenerative medicine (Cytonet) was announced publicly following a launch event on the 11th of October, which was held alongside a Newborn Screening Workshop (the third one to be held in Qatar and one of the few such workshops organized in the GCC).

"This initiative is one of the many medical and healthcare projects we are working on that will raise the health standards in Qatar and lead to a better quality of life for our population," commented Dr. Hanan Al Kuwari, Managing Director of Hamad Medical Corporation. "I am proud of how Hamad has been instrumental in developing this program which will help position Qatar as the regional hub for metabolic disorder diagnosis and treatment techniques.

This close cooperation will facilitate the treatment of children who suffer from urea cycle disorders, which is a genetic disorder caused by a deficiency of an enzymes in the urea cycle responsible for removing ammonia from the blood stream thereby cleaning it of toxins. Under the agreement, these children can be included faster into the liver cell therapy study programme.

Dr. Hilal Al Rifai, Director of the Newborn Screening Program, HMC, commented: "We are delighted to be participating in the further development of this very important option of treating those who are affected. This agreement allows us to now offer our most seriously ill patients the possibility of being quickly included in the SELICA study. Because, apart from a liver transplantation, which is often not possible - especially in affected newborns - there are no alternative curative treatment options for urea cycle defects. I am delighted that Qatar will be the regional center of excellence for this therapy."

Dr. Wolfgang Rüdinger and Michael J. Deissner, the Managing Directors of Cytonet, supported this notion by commenting that: "this international cooperation represents another step forward on the road to making liver cell therapy available and workable at all times in every metabolic center".

The treatment option for urea cycle defects – which includes severe and life-threatening metabolic disorders of the liver – is currently undergoing clinical examination. Within the framework of this cooperation agreement, physicians are also to be given further training as they work together with the metabolic experts at Heidelberg University Hospital.

The only currently available cure for this illness is the transplantation of a whole liver or a liver lobe. However, this treatment is highly problematic for very young children and neonatal patients. Additionally, suitable organs available for transplantation are very rare. A therapy using isolated and processed liver cells from non-transplantable donated livers (manufactured under clinically approved standards) has been investigated and further developed over the past years.

Cytonet is working in close cooperation with internationally leading neonatal and pediatric metabolism centers. The overall goal is an effective compensation of the metabolism disorder by means of the infusion of healthy, fully metabolism-competent human liver cells into the portal vein, with the aim of engrafting these in the liver of the child.

Prof. Dr. Georg Hoffmann, Head of the University Children's Hospital in Heidelberg, is a long-standing partner of Cytonet and HMC and has been a key supporter and promoter of this program. The Metabolism Center at the Center for Child and Adolescent Medicine in Heidelberg has spent over 40 years specializing in the diagnosis and treatment of congenital metabolic diseases.

Prof. Hoffmann commented on the importance of this program: "If they are not detected, congenital metabolic disorders can lead to organ damage, physical or mental disability, and even death. The symptoms are varied and often not specific: They range from serious crises after birth with loss of consciousness, seizures, respiratory disorders, or organ failure, to chronic diarrhea or physical and mental developmental disorders. Many of these diseases are easily treatable today, especially if they are diagnosed early enough". 

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