Celgene Cellular Therapeutics receives FDA clearance for first
placenta-derived stem cell clinical study
18 November 2008
Celgene Cellular Therapeutics (CCT), a wholly
owned subsidiary of Celgene Corporation (NASDAQ: CELG),
has received US FDA clearance for a clinical trial using PDA001, an immunomodulatory therapy utilizing human placenta-derived
stem cells obtained via CCT’s proprietary processes.
Clinical development will begin by the end of the year with
the initiation of this Phase I, multi-center clinical trial
in the US for patients with moderate-to-severe Crohn’s
disease, who are refractory to oral corticosteroids, such as
prednisone, and immune suppressants.
PDA001 is a novel culture-expanded stem cell population that has
broad therapeutic potential in areas such as immunology, inflammation, haematology and oncology. Pre-clinical evaluation of PDA001 has
demonstrated its safety and potent immune-suppressive properties. A
placenta-derived stem cell therapy, PDA001 has several potential
significant benefits in that the cells are derived from a safe and
almost unlimited source and are scalable to a traditional pharmaceutical
level.
CCT owns an array of proprietary technologies directed to novel
placental cell types and cell populations, as well as methods for
collecting, processing and storing many types of stem cells from the
placenta. PDA001 is the first product to be developed as a result of
this expanding portfolio.
“This first placental-derived stem cell clinical trial is of vital
importance given the significant number of people suffering from this
debilitating disease,” said lead investigator Dr Lloyd F Mayer,
Professor and Director of the Immunology Institute, Professor of
Medicine and Chief of the Divisions of Clinical Immunology and
Gastroenterology at Mount Sinai in New York City. “With the positive
results from in vivo biodistribution and safety studies, our hope is
that PDA001 will suppress the atypical immune and inflammatory reactions
involved in the pathogenesis of Crohn’s disease, thereby resulting in a
decrease in symptoms and improved quality of life for patients.”
Crohn’s disease, a chronic inflammatory condition of the
gastrointestinal tract, impacts almost one million people in the United
States alone. Traditional treatment for Crohn’s disease has focused on
non-specific anti-inflammatory or immunosuppressive agents.
Unfortunately, a considerable proportion of patients develops
intolerable side-effects, requires surgery or becomes unresponsive to
therapy.
“This is the first step in our program to initiate clinical
evaluations in a range of indications including not only Crohn’s
disease, but other serious inflammatory and autoimmune diseases, such as
multiple sclerosis and rheumatological disorders as well,” said Robert
Hariri, M.D., Ph.D., CEO of Celgene Cellular Therapeutics. “By creating
a novel cell therapy from a readily available source that does not
require human leukocyte antigen-matching, we are hopeful that we can
treat a large number of patients with a variety of devastating
diseases.”
About placenta-derived stem cells
Human Placenta-Derived Cells (PDA001) is a cellular immune modulatory
agent with significant therapeutic potential. PDA001 is a novel cell
population derived from normal, full-term human placental tissue.
PDA001 is a culture-expanded plastic adherent cell, undifferentiated
in vitro cell population that expresses the nominal phenotype CD34-,
CD10+, CD105+ and CD200+. PDA001 cells constitutively express moderate
levels of HLA Class I and undetectable levels of HLA Class II, and they
do not express the co-stimulatory molecules CD80 and CD86.
PDA001 is genetically stable, displaying a normal diploid chromosome
count, normal karyotype and exhibit normal senescence after prolonged in
vitro culture. PDA001 is capable of immunomodulation and suppresses
T-cell proliferation. PDA001 also exhibits immunomodulatory effects on
other cell types involved in an immune response such as T-cell subsets,
macrophages and dendritic cells.
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