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Successful liver cell therapy in newborns

Cytonet GmbH & Co. KG, Germany’s leading cell therapy company, has provided the first clear evidence for the efficacy of its liver cell therapy in severe congenital metabolic disorders of newborns. The study was conducted in cooperation with the University Children’s Hospital in Heidelberg.

According to initial therapeutic results of researchers from the University Children’s Hospital in Heidelberg and from Cytonet GmbH & Co. KG, Germany’s leading cell therapy company, it is possible for liver cell suspensions to redress the lack of enzyme activity in the liver of newborns suffering from severe congenital metabolic disorders associated with mental disability. Many metabolic disorders are the result of enzyme defects in liver cells. It is hoped that liver cell therapy will contribute to stabilising the metabolism of newborns suffering from such disorders as well as preventing neurological damage whilst they await a liver transplant.
Prof. Dr. Georg F. Hoffmann, Managing Director of the University Children’s Hospital in Heidelberg and Cytonet’s long-time cooperation partner says: “This is the first clear evidence of the efficacy of liver cell therapy for this disease, which is often associated with mental disability and premature death.” A ten-week old boy received liver cells that were produced by Cytonet. After 15 months, the boy received a split-liver transplant (editor’s note: i.e. a donated liver is split in two and each half is transplanted into a different patient).
When the child who was injected with the liver cell suspension had his liver removed and examined, it revealed a total enzyme activity of 5 per cent of a healthy liver. According to Dr. Jochen Meyburg, a neonatologist from Heidelberg and also one of Cytonet’s cooperation partners, this is a considerable increase in enzyme activity since the boy’s liver showed no activity at all prior to cell therapy. In addition, the injected liver cells not only accumulated in the child’s liver, but also proliferated. It was assumed that proliferation occurred because the measured enzyme activity was four times higher than calculations had led to expect it would be.
Schematic presentation of how the liver cell suspension is injected into the liver. (Figure: Cytonet)
The liver cell suspension compensates for the lack of enzyme activity in the liver of affected patients. Instead of being converted into urea which is removed from the body with the urine, ammonia accumulates in urea cycle disorder patients leading to irreversible brain damage or death. The disease is characterised by severe crises that occur at unforeseeable intervals. The doctors hope that the liver cell therapy will lead to the stabilisation of the children’s metabolism and prevent neurological damage for the time that it takes for the children to gain enough body weight to undergo liver transplant. Cytonet’s CEO, Dr. Dr. Wolfgang Rüdiger, and Cytonet’s CFO, Michael J. Deissner, highlighted that the liver cell therapy is a completely new option in the long-term therapy of congenital metabolic defects of the liver. These therapeutic findings are part of the clinical SELICA II (Safety & Efficacy of Liver Cell Application) study that is about to begin at the University Children’s Hospital in Heidelberg. In April 2008, the Paul Ehrlich Institute granted Cytonet approval for the study. SELICA’s objective is the systematic proof of the efficacy of liver cell suspensions in the treatment of congenital enzyme defects in the urea cycle of newborns. Prof. Hoffmann pointed out that the liver cell suspension is the first cell-based medicament produced according to GMP standards, which involved comprehensive investigations on the drug’s microbiological safety. Source: Cytonet GmbH & Co. KG, Weinheim - 25 August 2008
In 2006, the European drug regulatory agency, EMEA, granted the liver cell therapy orphan drug status recognising it as a pharmaceutical for rare diseases. This status gives applicants the prospect of an accelerated approval process.

Further information:
Michael Tschugg
Cytonet GmbH & Co. KG
Albert-Ludwig-Grimm-Str. 20
69469 Weinheim
Tel.: +49 (0)6201 – 259819
Fax: +49 (0)6201 – 259828
E-mail: presse@cytonet.de

Website address: https://www.gesundheitsindustrie-bw.de/en/article/press-release/successful-liver-cell-therapy-in-newborns