Clinical trial evaluates ex vivo cultured cord bloodkarate
Umbilical cord blood (UCB) is a rich source of hematopoietic stem and progenitor cells (HSPCs) that can be used for bone marrow transplantation; however, UCB transplantation is hampered by low numbers of HSPCs per donation, which delays engraftment and immune reconstitution.
In this issue of the Journal of Clinical Investigation, Mitchell Horwitz and colleagues at Duke University Medical Center conducted a phase I clinical trial to test the long term engraftment capability of UCB HSPCs that were expanded ex vivo for 21 days in the presence of nicotinamide and then combined with the noncultured T cell fraction of the UCB (NiCord). Eleven adults with hematological malignancies received 1 unit of NiCord and a second unit of unmanipulated UCB following myeloablative bone marrow conditioning. Eight patients exhibited complete or partial engraftment of the NiCord unit and achieved earlier hematopoietic recovery compared to historical controls. These results indicate that further clinical study of NiCord is warranted.
TITLE: Umbilical cord blood expansion with nicotinamide provides longterm multilineage engraftment
The tumor can have both mesodermal and ectodermal features, making it difficult to determine the cellular origin. In this issue of the Journal of Clinical Investigation, Takuro Nakamura and colleagues at the Japanese Foundation for Cancer Research developed a murine model of Ewing's sarcoma by introducing EWSETS fusion genes into a late gestational cell fraction enriched for osteochondrogenic progenitors from the embryonic superficial zone (eSZ) of the long bones The model tumors shared a gene expression profile with human Ewing's sarcoma and both murine and human tumors exhibited upregulated of WNT/βcatenin and EGF signaling. Finally, inhibitors of the WNT/βcatenin pathway attenuated murine tumor growth. This model provides a tool to evaluate potential therapies for treatment of this rare disease.