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For many patients with such blood diseases, including sickle cell disease, the only hope for curing them requires transplanting normal blood stem cells. However, in many instances, suitable normal blood stem cells cannot be found or there are insufficient numbers of cells for transplantation. The consortium seeks to address this critical need with an innovative system to expand stem cells outside the body using specialized blood vessel cells — known as a vascular niche — to support and nurture the stem cells as they do inside the body.
The consortium will conduct two clinical trials using this platform to expand hematopoietic stem cells. The first trial uses the vascular niche to expand umbilical cord blood stem cells for transplantation in patients with blood cancers that cannot be cured by chemotherapy or available donors. The second trial aims to correct the genetic abnormality in blood stem cells from patients with sickle cell anemia and then return these cells to the patients to supply healthy, functioning stem cells. If successful, the techniques may provide safer, more broadly available stem cell transplants to many thousands of patients affected by blood disorders.
Source: Weill Cornell Medical College