20.09.2007 12:00:00
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Children Can be Cured of Sickle Cell Disease and Thalassemia After Sibling Cord Blood Transplantation: Results from ViaCell and Children's Hospital & Research Center Oakland
ViaCell, Inc. (Nasdaq: VIAC) and Children’s
Hospital & Research Center Oakland reported results today that children
with Sickle Cell Disease and Thalassemia can be cured with umbilical
cord blood from a compatible sibling. At the Sickle Cell Disease
Association of America and National Institutes of Health (NIH) 35th
Annual Convention, Dr. Mark Walters, Director of the Blood and Marrow
transplant program at Children’s Hospital &
Research Center Oakland presented research data demonstrating that cord
blood from a relative can be an effective source of stem cells for
transplantation in children affected with Sickle Cell Disease and
Thalassemia and may have advantages over bone marrow transplantation.
"Patients with Sickle Cell and Thalassemia
often lead debilitating lives,” said Dr.
Walters. "Through continued research and
transplant success, sibling umbilical cord blood has proven to be
effective in curing children of these blood disorders. I expect the use
of umbilical cord blood will continue to increase and as we gain more
experience using cord blood stem cells in transplant medicine, I believe
it could outpace the use of bone marrow in transplant medicine.”
The data presented at the Sickle Cell Disease Association of America and
NIH meeting showed outcomes from children treated under The Sibling
Connection Program, a directed sibling transplant program implemented by
ViaCord and Children’s Hospital Oakland
Research Institute (CHORI), the research arm of Children’s
Hospital & Research Center Oakland. This program has resulted in cord
blood treatments in more than 100 children to date. Of the children
treated under the Sibling Connection Program, 17 were transplanted for
Sickle Cell Disease and 23 were transplanted for Thalassemia. The median
age of patients treated for Sickle Cell Disease was 8 years and 5 years
for patients treated for Thalassemia.
Transplantation of sibling umbilical cord blood has demonstrated
clinical advantages over bone marrow transplantation in young children.
In particular, the risk of graft-versus-host (GvHD) disease, a common
side-effect and the leading cause of death in transplant medicine, is
reduced. Of the children treated, six patients with Sickle Cell Disease
had acute GvHD. No patients treated for Sickle Cell Disease had chronic
GvHD. In addition, no acute or chronic GvHD was observed in patients
transplanted for Thalassemia.
The median time to neutrophil recovery (ANC greater than 500 cells per
microliter) and platelet recovery (greater than 20,000 per microliter)
in patients treated for Sickle Cell Disease was 18 days and 36 days,
respectively. 82% of the patients treated for Sickle Cell Disease
survive and are disease-free. The median time to neutrophil recovery
(ANC greater than 500 cells per microliter) and platelet recovery
(greater than 20,000 per microliter) in patients treated for Thalassemia
was 25 days and 47 days, respectively. 96% of the patients treated for
Thalassemia survive and 91% are disease-free.
In 2006, ViaCell and CHORI combined their efforts in the area of
directed transplants for sibling donor umbilical cord blood to form the
Sibling Connection Program. To date, over 100 children have been treated
by cord blood from units collected and processed through this program.
This includes transplants through cord blood collected, preserved and
stored with ViaCord and transplants using cord blood stored through
CHORI's Sibling Donor Cord Blood Program. The Sibling Connection Program
provides ViaCord’s comprehensive cord blood
collection, processing and five years of storage at no cost to families
who have a child diagnosed with a condition that can be treated with
cord blood stem cell transplant and meet the other requirements of the
program.
About Sickle Cell Disease
Sickle Cell Disease is an inherited blood disorder where red blood cells
are sickle or crescent shaped. The abnormally-shaped cells become rigid
and prevent normal flow of oxygen to tissues, causing tissue damage.
Common symptoms include chest pain, frequent infections, jaundice, and
anemia. There is currently no universal cure for Sickle Cell Disease.
Complications of Sickle Cell Disease can be treated with antibiotics,
pain management, intravenous fluids, blood transfusions and surgery.
Over 2.5 million people in the United States carry the trait and over
80,000 have sickle cell anemia. Sickle Cell Disease predominantly
affects African Americans and people of Mediterranean descent. September
is National Sickle Cell Awareness Month.
Umbilical cord blood has been successfully transplanted in patients with
Sickle Cell Disease and Thalassemia as an emerging therapeutic treatment
option. ViaCord released its first cord blood unit from a related source
to treat Sickle Cell Disease in 2000. 30% of all enrollments in the
ViaCord/CHORI Sibling Connection Program are with families who have
children affected with Sickle Cell Disease.
About Thalassemia
Thalassemia is a hereditary blood disorder characterized by decreased
production of hemoglobin, a critical oxygen-carrying protein in red
blood cells. This results in anemia and shortage of red blood cells.
Diagnosis is typically early in childhood with lifelong red blood cell
transfusions and resulting complications. The symptoms of thalassemia
depend on the type and severity of the disease and include anemia,
jaundice, enlarged spleen and liver, abnormal facial bones and poor
growth. It is estimated that over 2 million people in the United States
carry the genetic trait for Thalassemia and approximately 1,000 people
are living with Thalassemia in the U.S.
About Umbilical Cord Blood
Umbilical cord blood is a valuable, non-controversial source of stem
cells with proven therapeutic effect in treating over 40 diseases.
These diseases include cancers such as Acute Lymphoblastic Leukemia
(ALL) and Non-Hodgkin’s lymphoma, certain
bone marrow failure syndromes such as severe aplastic anemia and Diamond
Blackfan anemia, certain blood disorders such as sickle cell anemia,
thalassemia and other genetic disorders. Over 8,000 cord blood
transplants have been performed worldwide. Studies have shown that
umbilical cord blood transplants from a family member, rather than from
a non-relative, have a significantly higher survival rate than
transplants from an unrelated donor.
About ViaCord
ViaCord allows expectant families the opportunity to preserve their baby’s
umbilical cord blood for potential medical use by the child or a related
family member. The child’s cord blood is
collected at the time of birth in the only FDA-approved cord blood
collection bag suitable for use in a sterile field. To date, ViaCord has
preserved over 130,000 newborn’s umbilical
cord blood. The ViaCord Processing Laboratory, located in Hebron,
Kentucky, is an AABB accredited cord blood facility. Additional
information about ViaCord is available online at http://www.viacord.com.
Research at Children’s Hospital & Research
Center Oakland, CA
Research efforts at Children’s Hospital &
Research Center Oakland are coordinated through Children’s
Hospital Oakland Research Institute (CHORI). Children’s
Hospital Oakland is Northern California’s
only freestanding and independent children’s
hospital. CHORI’s internationally renowned
biomedical research facility brings together seven centers of excellence
that are devoted to clinical and basic science research to treat and
prevent disease. CHORI has approximately 300 staff members and an annual
budget of more than $49 million. The National Institutes of Health is
CHORI’s primary funding source. The institute
is a leader in translational research, bringing bench discoveries to
bedside applications. These include providing cures for blood diseases,
developing new vaccines for infectious diseases and discovering new
treatment protocols for previously fatal or debilitating conditions such
as cancers, sickle cell disease and thalassemia, diabetes, asthma,
HIV/AIDS, pediatric obesity, nutritional deficiencies, birth defects,
hemophilia and cystic fibrosis.
About ViaCell
ViaCell, Inc. is a biotechnology company dedicated to enabling the
widespread application of human cells as medicine. The Company markets
ViaCord®, a product offering through which
families can preserve their baby’s umbilical
cord blood at the time of birth for possible future medical use in
treating over 40 diseases including certain blood cancers and genetic
diseases. The Company is also working to leverage its commercial
infrastructure and product development capabilities by developing ViaCyteSM,
a product candidate being studied for its potential to broaden
reproductive choices for women through the cryopreservation of human
unfertilized eggs. ViaCell also conducts research and development
primarily to investigate other potential therapeutic uses of umbilical
cord blood-derived stem cells and on technology for expanding
populations of these cells. ViaCell’s
pipeline is focused in the areas of cancer, cardiac disease, and
diabetes. For more information about ViaCell, visit our website at http://www.viacellinc.com.
Forward-Looking Statements
This press release contains forward-looking statements regarding
expectations about the increased use of cord blood in transplant
medicine. These statements are based on current expectations, and are
subject to a number of risks and uncertainties that could cause actual
results to differ materially from current expectations. For example,
expectations about the increased use of cord blood may be negatively
impacted by data or research that indicates that cord blood is not more
effective than existing or future therapies in treating diseases that
are not currently treatable with cord blood, and any unexpected material
issues, delays or failures in the collection, processing, storage or
transplant of umbilical cord blood. For more detailed information on the
risks and uncertainties associated with these forward-looking statements
and the Company's other activities, see the periodic reports filed by
the Company with the Securities and Exchange Commission. The Company
does not undertake any obligation to publicly update any forward-looking
statements, whether as a result of new information, future events, or
otherwise.
ViaCell® and ViaCord®
are registered trademarks and ViaCyteSM is a
service mark of ViaCell, Inc.
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