[March 11, 2014] |
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First Data on Cytonet's Liver Cell Therapy for Urea Cycle Disorders Presented at the Annual Meeting of the Society for Inherited Metabolic Disorders (SIMD)
WEINHEIM, Germany & RALEIGH-DURHAM, N.C. --(Business Wire)--
Interim data on the use of liver cell therapy (LCT) for the treatment of
pediatric patients with Urea
Cycle Disorders (UCD) were presented today at the Annual
Meeting of the Society for Inherited Metabolic Disorders (SIMD) in
Pacific Grove, California.
UCD is a rare, potentially life-threatening liver metabolism disorder
that in its severe form can affect newborns and infants, causing toxic
levels of ammonia to accumulate in the body, which can lead to massive
and irreversible damage of the nervous system including the brain. For
patients suffering from severe neonatal UCD, liver transplantation
remains the only option for long term stabilization. However, liver
transplantation is still very difficult in small infants and success
rates clearly increase in older infants. Therefore, the current goal for Cytonet's
liver cell therapy is to treat severe UCD patients in order to stabilize
them until they have reached an age and size that allows liver
transplantation to be performed with a high probability of success
and/or until a suitable donor organ becomes available.
Thomas Opladen, MD, from the University of Heidelberg presented data
from a combined interim analysis of two studies performed in Germany,
the U.S. and Canada. Patients less than six months of age with a
confirmed diagnosis of UCD (OTCD, CPS1D or ASSD (citrullinemia) were
treate with liver cell therapy and were compared to a historical
control group treated with the current standard of care. Advantages for
the treated group were seen for parameters such as event free survival
time or incidence of moderate or severe hyperammonemic events. Reduction
of such events is the objective of the liver cell therapy in order to
avoid irreversible brain damage. The liver cell therapy was well
tolerated.
"The markedly prolonged event free time and the less severe
hyperammonemic events after treatment with liver cell therapy are
promising and suggest that liver cell therapy may play an important role
in managing patient's ammonia levels," said Dr. Opladen.
Cytonet's liver cell therapy involves collecting healthy cells from
donated livers not suitable for organ transplantation. These cells are
infused into the portal vein in six sessions on six consecutive days.
The Company is currently conducting two on-going multicenter clinical
trials in U.S. and Canada (SELICA III), and in Germany (SELICA V).
In December 2013, Cytonet submitted a Marketing Authorization
Application (MAA) to the European Medicines Agency (EMA (News - Alert)) seeking
approval for its liver cell therapy for the treatment of Urea Cycle
Disorders in children.
ABOUT UREA CYCLE DISORDERS
According to the National Urea Cycle Disorders Foundation (NUCDF), a
urea cycle disorder is a genetic disorder caused by a deficiency of one
of the six enzymes in the urea cycle which is responsible for removing
ammonia from the blood stream. These include carbamoyl phosphate
synthetase I (CPS I) deficiency, N-acetylglutamate synthetase (NAGS)
deficiency, ornithine transcarbamylase (OTC) deficiency,
argininosuccinate synthetase (ASS) deficiency (which is also known as
citrullinemia), argininosuccinate lyase (ASL) deficiency and arginase 1
deficiency (hyperargininemia). The urea cycle involves a series of
biochemical steps in which nitrogen, a waste product of protein
metabolism, is removed from the blood and converted to urea. Normally,
the urea is transferred into the urine and removed from the body. In
urea cycle disorders, the nitrogen accumulates in the form of ammonia, a
highly toxic substance, and is not removed from the body resulting in
hyperammonemia (elevated blood ammonia). Ammonia then reaches the brain
through the blood, where it causes irreversible brain damage, coma
and/or death.
Urea cycle disorders are included in the category of inborn errors of
metabolism. Inborn errors of metabolism represent a substantial cause of
brain damage and death among newborns and infants. Because many cases of
urea cycle disorders remain undiagnosed and/or infants born with the
disorders die without a definitive diagnosis, the exact incidence of
these cases is unknown and underestimated. It is believed that up to 20
percent of Sudden Infant Death Syndrome cases may be attributed to an
undiagnosed inborn error of metabolism such a urea cycle disorder. For
more information, please visit NUCDF at www.nucdf.org.
ABOUT CYTONET
Cytonet is an international biotechnology company which is located in
Weinheim and Heidelberg in Germany and in Durham, NC in the U.S. The
Company develops and produces cellular products for therapeutic
purposes. Cytonet's goal is to provide alternatives to existing
therapies for many diseases with a particular emphasis on liver
diseases. Cytonet is a pioneer and leader in the field of regenerative
medicine. For the past several years, Cytonet has worked with
internationally-leading metabolism and neonatal centers to study its
liver cell therapy which uses healthy and metabolically functional human
liver cells collected from donated livers not suitable for transplant
for infusion to treat urea cycle disorders in children. For more
information, please visit Cytonet's website at http://www.cytonetllc.com/

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