Potential For Cellbased Therapies

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Umbilical cord blood is commonly used in cell-based therapies today for reconstitution of the bone marrow after bone marrow ablation for cancers of the blood (36). There are some new experimental therapies using bone marrow transplant with cord blood cells being developed for other diseases. Umbilical cord blood transplantation in Wiskott Aldrich syndrome, which results in severe immune deficiency and early death if not treated, was found to result in rapid and reliable recovery of immune function, with low risk of graft-vs-host disease (81). Using umbilical cord blood stem cells taken from unrelated donors, Staba et al. (82) treated children with Hurler's syndrome, who lack of a functional enzyme, alpha-l-iduronidase. These researchers were able to treat these patients without bone marrow ablation and to have improvement in survival and less neuronal degeneration than Hurler's patients who received bone marrow transplants. The researchers speculate that stem cells from cord blood may transport a-l-iduronidase across the blood-brain barrier more effectively. In addition, they are younger cells and do not have to be matched as closely. Research is under way to expand the use of umbilical cord blood cells to treat other disorders such as p-thalassemia (83).

Animal models suggest that umbilical cord blood cells may be useful in treatment of amyotrophic lateral sclerosis by slowing motor neuron degeneration when injected intravenously (84). Ende and coworkers found that intravenous injection of umbilical cord blood cells could extend the survival of several mouse knockout models of human disease, including amyotrophic lateral sclerosis (85), Alzheimer's (85), Huntington's (86), Parkinson's (87), and type 1 diabetes (88). Human umbilical cord blood cells also improve the mobility of rats with spinal cord injuries when injected intravenously. Cord blood cells were observed in the areas of injury of spinal cord but not others and never seen in the control, uninjured animals (89). Similarly, umbilical cord blood cells were able to improve function in a stroke model in the rat when injected intravenously. The human umbilical cord blood cells differentiated into cells that expressed glial or neuronal markers (90). This suggests that umbilical cord blood cells have the ability to target to and heal neurologic defects.

Cells from umbilical cord matrix may also be a source of cells for treatment of neurodegenerative disease. Medicetty et al. (91) treated rats with a unilateral 6-hydroxydopamine (6-OHDA) lesion that caused parkinsonian-like symptoms. Four weeks after the 6-OHDA lesion, rats were injected with umbilical cord matrix cells or sham transplants. Four weeks after transplantation, there was a significant decrease in apomorphine-induced rotatory behavior in the parkinsonian rats that received umbilical cord matrix cell transplants as compared with parkinsonian rats that received a sham transplant. Normal rats, without 6-OHDA lesions, were transplanted with umbilical cord matrix cells but showed no changes in behavior. This work suggests that umbilical cord matrix cells can target areas of neurodegeneration and play a role in healing of neural tissue. Amniotic cells may have a similar potential (92). Labeled amniotic epithelial cells were injected into monkeys with spinal cord injuries. Some labeled neurons were subsequently found in the spinal cord. Glial scar formation was decreased compared with animals that did not receive amniotic epithelial cells. More importantly, the function of the animals improved suggesting that amniotic epithelial cells help in axon regrowth. These studies suggest that cells from umbilical cord blood and other cells from extraembryonic tissues may be an important source of stem cells for a variety of therapeutic applications.

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