Celgene Cellular Therapeutics (CCT), a wholly owned subsidiary of Celgene Corporation, announced a ground breaking clinical application of human placenta-derived stem cells (HPDSCs) for haematopoietic reconstitution.
The transplant occurred at the LSU Health Sciences Centre Children's Hospital (LSU) on March 28, 2008 to treat a paediatric patient with acute lymphoblastic leukaemia (ALL), a cancer of the bone marrow and blood, according to Celegene. Following the birth of the patient's sibling in December 2007, HPDSCs, along with cord blood, were collected and cryo-preserved and both products were used in the transplant.
CCT owns proprietary technologies for collecting, processing, and storing HPDSCs. HPDSCs are immature and versatile stem cells with potentially broad therapeutic applications in, for example, leukaemia and other haematological malignancies, solid tumour cancers, and autoimmune diseases. CCT is also conducting research on other types of stem cells derived from the placenta that are obtained via additional proprietary methodologies.
The transplant is part of a multicenter clinical trial being conducted at LSU, together with Morgan Stanley Children's Hospital of New York-Presbyterian and Columbia University Medical Centre using HPDSCs for patients with a range of disorders including cancers and non-malignancies. It is a single-arm study with a primary objective of assessing the safety of transplantation of umbilical cord blood augmented with HPDSCs from the same donor, with a secondary objective of assessing potential restoration of normal haematopoiesis and immune function with this combination of cells. Patients will be monitored carefully post-transplant for up to 24 months to monitor safety outcomes, engraftment, and survival.
"The patient is doing extremely well and, in fact, was discharged from the hospital one-to-two weeks earlier compared to traditional cord blood transplants," said Lolie C. Yu, M.D., Professor of Paediatrics, Division Chief of the Paediatric Heme-Onc Programme, Director of the BMT Programme at LSUHSC/Children's Hospital. "Neutrophil engraftment occurred earlier than anticipated in this proof-of-principle study further raising our hopes regarding the benefits of this treatment."
There is considerable anticipation around the potential of HPDSCs. An independent study headed by researchers from UCLA published earlier this year declared that blood-forming stem cells originate in the placenta in laboratory animals.
"We are excited to advance our study of the placenta as a source of stem cells that have the ability to effectively treat patients worldwide," said Robert Hariri, CEO, Celgene Cellular Therapeutics. "We are pleased with this first positive step in our effort to bring this cutting-edge therapy to the clinic to treat sufferers with a variety of devastating diseases."
"For the first time, we are transplanting human placenta-derived stem cells to treat patients with a variety of cancers and non-malignant diseases," said Mitchell Cairo, M.D., who leads the clinical trial at Morgan Stanley Children's Hospital and Columbia University Medical Centre, and is Chief of the Paediatric Blood and Marrow Transplantation Division at Morgan Stanley Children's Hospital of New York-Presbyterian and Professor of Paediatrics, Medicine and Pathology at Columbia University College of Physicians and Surgeons. "Our hope is that HPDSCs, in conjunction with cord blood stem cells, will expedite engraftment and improve treatment for patients."