Clinical Applications

In the United States alone, nearly 120,000 patients each year qualify as candidates for a bone marrow transplant with the administration of hematopoietic stem cells. Unfortunately, about 100,000 of these patients (or 80 percent) do not receive a transplant due to the lack of a match between recipient and donor cells.

To address this enormous unmet clinical need, scientists have identified alternative sources of hematopoietic stem cells for bone marrow transplants. In particular, umbilical cord blood contains a rich reservoir of these stem cells. Umbilical cord blood-derived stem cells are more readily available and usage is not burdened with the same strict requirements for matching as seen with cells obtained from the bone marrow. However, since only small numbers of these cells are obtained, their use has historically been limited to transplantation in children weighing less than 25 kg.

ASC-101 and ASC-201 offer the potential to overcome this limitation and allow expanded use of these small numbers of umbilical cord blood cells not only for children but also adults.

The beneficial effect of these technologies may potentially extend to transplantation of mature peripheral blood stem cells as well. In addition to utilization in hematopoietic stem cell transplants for lymphoma, leukemia, myeloma, anemia, and inherited red cell abnormalities, ASC-101 and ASC-201 profile as platform technologies based on their potential to enable stem cells to better home to targeted organs and treat 70+ diseases. In particular:

  • Treatment of areas of inflammation caused by chemotherapy and irradiation for cancer patients


  • Treatment of nonmalignant diseases such as autoimmune disease and improving functional neurological recovery following strokes


  • Use of cord blood treated with ASC-101 in place of regular blood transfusions for cancer patients that require frequent blood transfusions to supplement intense doses of chemotherapy and irradiation


  • Non-hematopoietic applications such as enhancing the homing of stem cells to diseased and cancer-ravaged organs like the heart, liver, kidneys, breast to allow the regenerative capacity of these stem cells to aide in the recovery of these tissues


  • Treatment of other types of stem cells with ASC-101, such as mesenchymal and adipose cells, to promote their homing to target tissues

References:

  1. Bailen K., New Trends in Umbilical Cord Blood Transplantation, Blood, 2005, 105:3788- 3792


  2. Xia L., et al., Surface Fucosylation of Human Cord Blood Cells Augments Binding to P- Selectin and E-Selectin and Enhances Engraftment in Bone Marrow, Blood 2004, 104:3091- 3096


  3. Broxmeyer HE., Human Umbilical Cord Blood as a Potential Source of Transplantable Hematopoietic Stem/Progenitor Cells, Proc Natl Acad Sci USA 1989, 86:3828-3832.


  4. Mini-Review on Hematopoietic Stem Cells by Dr. Bert Wognum at Stem Cell Technologies

    5. http://www.stemcell.com/technical/29015_Hematopoietic.pdf

  5. Goodwin HS., et al., Multilineage Diferentiation Activity by Cells Isolated from Umbilical Cord blood: Expression of Bone, Fat, and Neural Markers.  Biol Blood Marrow Transplant 2001, 7:581- 588


  6. Vendrama M.  et al., Infusion of Human Umbilical Cord Blood Cells in a Rat Model of Stroke Dose-Dependently Rescues Behavioral Deficits and Reduces Infarct Volume.  Stroke 2004 36: 2390 – 2395.