AMAG Announces Autologous Cord Blood Cells Improve Motor Function in Some Children with Cerebral Palsy
November 08 2017 - 8:00AM
Cord Blood Registry (CBR) from AMAG Pharmaceuticals (Nasdaq:AMAG)
today announced research recently published in the journal Stem
Cells Translational Medicine found that children with cerebral
palsy who received an infusion of their own neo-natal cord blood
(collected & stored at birth) saw improved motor function and
brain connectivity one year after treatment, when receiving a high
cell dose (>20 million total nucleated cells per kilogram). The
prospective, randomized, double-blind, placebo-controlled Phase 2
clinical study examining the efficacy of autologous cord blood in
treating children with cerebral palsy was led by researchers at
Duke University.
“The study results are compelling for further study of the use
of autologous blood cord infusions in children with cerebral
palsy,” said Joanne Kurtzberg, M.D., the study’s principal
investigator and director of the Carolinas Cord Blood Bank and The
Duke Pediatric Blood and Marrow Transplant Program. Dr. Kurtzberg
is also chief scientific officer of the Robertson Clinical and
Translational Cell Therapy Program at Duke University.
Following a Phase 1 study which showed the use of autologous
cord blood to be safe in children with neurologic disorders, the
aim of the Phase 2 trial was to determine efficacy using autologous
cord blood for treatment of cerebral palsy in pediatric patients
ages one through six. Improvement in the study was measured using
the Gross Motor Function Measure (GMFM-66), a standardized
assessment to evaluate motor function (e.g. walking ability).
Whole brain connectome (neural mapping) exploratory analysis was
also performed using magnetic resonance imaging (MRI) of the brain.
Sixty-three children were randomized into a treatment (n = 32) or
placebo (n = 31) group. Of the 32 children receiving treatment,
those who were given a dose greater than 20 million total nucleated
cells per kilogram showed statistically significant, and clinically
meaningful improvements in motor function measured on a validated
scale for assessing motor function in children with cerebral palsy
compared to children who received lower doses of cord blood
(p=0.05) or the placebo (p=0.04). Additional analysis showed that
children who received higher cord blood cell doses also showed a
greater increase in normalized whole brain connectivity compared to
children who received lower cell doses. While these results are
encouraging, limitations of the study include a small sample size,
heterogeneity of the participants, and greater than historically
observed gains in motor function observed in all groups.
More than one-third of the children in this study—or 22 out of
63 total study participants—had their cord blood preserved at birth
with CBR. CBR is one of the only private cord blood banks that
collects health status information from families storing in order
to facilitate participation in clinical research. CBR aims to
advance families' access to new clinical applications of cord blood
stem cells for conditions that currently have no cure through
regenerative medicine and is committed to advancing the science on
the use of cord blood stem cells. The results of this study
highlight the need for continued research of autologous cord blood
infusions to treat cerebral palsy. CBR is partnering with research
institutions to support FDA-regulated clinical trials investigating
the use of cord blood in regenerative medicine applications across
a wide variety of conditions, including autism, acquired hearing
loss, and two clinical trials on cerebral palsy at Augusta
University and The University of Texas Health Sciences Center.
“We are excited about these results from this early trial
regarding the potential for autologous cord blood in children with
cerebral palsy and the impact it may have on both the participants
and their families,” said Heather Brown, vice president, Scientific
and Medical Affairs, CBR. “It is encouraging to see that the
results of this trial suggest that in some of the children with
cerebral palsy, when dosed ≥2 × 107 cells per kg, an infusion
of a child’s own cord blood improves whole brain connectivity and
motor function.”
About Cord Blood Registry®
(CBR®) CBR, from AMAG Pharmaceuticals, Inc. (NASDAQ:AMAG),
is the world’s largest private newborn stem cell company. Founded
in 1992, CBR is entrusted by parents with storing approximately
700,000 umbilical cord blood and cord tissue units. CBR is
dedicated to advancing the clinical application of newborn stem
cells by partnering with research institutions on FDA-regulated
clinical trials for conditions that have no cure today. For more
information, visit www.cordblood.com
About AMAGAMAG is a biopharmaceutical company
focused on developing and delivering important therapeutics,
conducting clinical research in areas of unmet need and creating
education and support programs for the patients and families we
serve. Our currently marketed products support the health of
patients in the areas of maternal and women’s health, anemia
management and cancer supportive care. Through CBR®, we also help
families to preserve newborn stem cells, which are used today in
transplant medicine for certain cancers and blood, immune and
metabolic disorders, and have the potential to play a valuable role
in the ongoing development of regenerative medicine. For additional
company information, please
visit www.amagpharma.com.
Forward-Looking Statements This press release
contains forward-looking information about AMAG Pharmaceuticals,
Inc. within the meaning of the Private Securities Litigation Reform
Act of 1995 and other federal securities laws. Any statements
contained herein which do not describe historical facts, including,
among others, statements about the safety and efficacy of
autologous cord blood use in children with neurological disorders,
including cerebral palsy; the ability of CBR to advance families'
access to new clinical applications of cord blood stem cells for
conditions that currently have no cure through regenerative
medicine and to advance the science on the use of cord blood stem
cells; the ability of autologous cord blood to be used in children
with cerebral palsy and the potential impact it may have on both
the children and their families; AMAG’s belief that the results of
the trial suggest that in some children with cerebral palsy, when
dosed ≥2 × 107 cells per kg, an infusion of a child’s own cord
blood improves whole brain connectivity and motor function; and
beliefs that newborn stem cells have the potential to play a
valuable role in the development of regenerative medicine are
forward-looking statements which involve risks and uncertainties
that could cause actual results to differ materially from those
discussed in such forward-looking statements.
Such risks and uncertainties include, among others, those risks
identified in AMAG’s filings with the U.S. Securities and Exchange
Commission (SEC), including its Annual Report on Form 10-K for the
year ended December 31, 2016, its Quarterly Reports on Form 10-Q
for the quarters ending March 31, 2017, June 30, 2017 and September
30, 2017 and subsequent filings with the SEC. Any such risks and
uncertainties could materially and adversely affect AMAG’s results
of operations, its profitability and its cash flows, which would,
in turn, have a significant and adverse impact on AMAG’s stock
price. AMAG cautions you not to place undue reliance on any
forward-looking statements, which speak only as of the date they
are made.
AMAG disclaims any obligation to publicly update or revise any
such statements to reflect any change in expectations or in events,
conditions or circumstances on which any such statements may be
based, or that may affect the likelihood that actual results will
differ from those set forth in the forward-looking statements
AMAG Pharmaceuticals® and Feraheme® are registered trademark of
AMAG Pharmaceuticals, Inc. MuGard® is a registered trademark of
Abeona Therapeutics, Inc. Makena® is a registered trademark of AMAG
Pharmaceuticals IP, Ltd. Cord Blood Registry® and CBR® are
registered trademarks of Cbr Systems, Inc. Intrarosa® is a
registered trademark of Endoceutics, Inc.
AMAG Pharmaceuticals, Inc. Contacts:
Investors:Christi WaarichAssociate Director, Investor Relations
617-498-7638
Media: Rushmie NofsingerExecutive Director, Corporate
Communications & Alliance Engagement617-498-3332
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