New Data Presented at the 2014 Joint Garrod and Canadian Newborn and
Child Screening Symposium
OTTAWA, Ontario & RALEIGH-DURHAM, N.C.--(BUSINESS WIRE)--
New data on Cytonet’s
investigational liver cell therapy (LCT) found it may help temporarily
stabilize pediatric patients with Urea
Cycle Disorders (UCD) while they await liver transplantation. The
study was presented at the 2014
Joint Garrod and Canadian Newborn and Child Screening Symposium in
Ottawa, Ontario on May 31, 2014.
UCD comprise a group of rare, potentially life-threatening disorders of
liver metabolism that affect newborns and infants. In its severe form it
causes ammonia to accumulate in the body, leading to 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.
Liver cell therapy may help keep the patient stable until a liver
transplant is possible. It 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.
Cytonet currently has two ongoing multicenter clinical trials in U.S.
and Canada (SELICA III), and in Germany (SELICA V) exploring the use of
liver cell therapy for patients with UCD.
In his presentation, Aneal
Khan, MD, lead author and assistant professor of medical genetics
and pediatrics at the University of Calgary and Alberta Children’s
Hospital in Calgary, Alberta, Canada, shared case reviews of patients
participating in the SELICA III trial. Between October 2012 and December
2013, Dr. Khan treated four patients under the age of 3 with Cytonet’s
liver cell therapy. All four achieved a period of sustained
normalization of ammonia levels within 30 days, though the duration of
control varied from patient to patient.
One patient was successfully bridged to solid organ liver
transplantation and two are currently stable, awaiting eligibility
clearance. One patient died as a result of complications due to the
underlying disease prior to organ transplantation.
“After receiving liver cell therapy, all four of our patients showed
normal and stable ammonia levels for a period of time – some patients
went up to 10-12 months without a single hyperammonemic episode. In
patients where a suitable donor organ was found, liver cell therapy was
a successful bridge to liver transplant that also helped the patient
recieve adequate nutrition to support growth and development,” said Dr.
Khan. “The much longer survival of boys with a deletion in the OTC gene
after liver cell therapy shows that this approach shows promise in
changing the natural history of a disease that has been universally
fatal in our population up until now.”
In December 2013, Cytonet submitted a Marketing Authorization
Application (MAA) to the European Medicines Agency (EMA) 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), urea
cycle disorders comprise a group of genetic disorders leading to 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.
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 metabolic 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/.
Susan Donath, +49 (6201) 2598-133