Stem Cell Report – June 2000

Date: 07/02/2000

Successful Trial of Gene Therapy in France Uses “Adult” Stem Cells

French researchers recently reported what appears to be the first successful trial of human gene therapy, boosting the immune systems of two infants with severe combined immunodeficiency by injecting them with their own stem cells containing a normal copy of the gene that they lacked. Researchers removed stem cells from the infants’ bone marrow, added a working copy of the gene to the cells’ DNA, and injected the repaired stem cells back into the infants (8 and 11 months old at the time of treatment).1

Adult Stem Cells Reverse Diabetes in Mice

Researchers at the University of Florida announced in March that they have reversed diabetes in mice using adult pancreatic stem cells. The pancreatic stem cells were harvested from adult donors and grown in culture, where they formed small functional organs known as islets of Langerhans (the insulin-secreting parts of the pancreas.) The cells were then injected under the skin of the diabetic mice, where they began secreting insulin. Within 7-10 days, the mice were able to regulate the levels of glucose in their blood.2

Bone Marrow Stem Cell Transplant Used to Treat Children with Cartilage Defect

Bone marrow-derived stem cells have been used clinically to treat children with osteogenesis imperfecta, a condition that leads to multiple fractures, severe bony deformities, and considerably shortened stature. Three months after treatment, the three children showed changes indicating new dense bone formation. The report by researchers at St. Jude Children’s Hospital in Memphis indicates the promising possibility for treatment of this as well as similar stem cell disorders.3

Neural Stem Cells Able to Repair Damage Throughout Brain

In studies with mice, neural stem cells have been shown to globally replace damaged brain tissue. The study, performed by researchers at Harvard Medical School, involved transplanting neural stem cells from born mice into the brains of “shiverer” mice, a model for Parkinson’s and similar central nervous system diseases. The neural stem cells were able to migrate throughout the brain to repair damaged tissue. Treated mice showed a decrease in tremors.4

Corneal Stem Cells Help Restore Sight

Corneal stem cells have been used by doctors in Japan to restore useful vision to patients who were legally blind. Transplants of adult corneal stem cells were used for conditions in which normal cornea transplants were unsuitable. One year after treatment, over half of patients had marked improvements in vision.5

  1. M. Cavazzana-Calvo, et al., “Gene Therapy of Human Severe Combined Immunodeficiency (SCID)-X1 Disease,” Science 288, 669-672, April 28, 2000.
  2. V. K. Ramiya, et al., “Reversal of insulin-dependent diabetes using islets generated in vitro from pancreatic stem cells,” Nature Medicine 6, 278-282, March 2000.
  3. E. M. Horwitz, et al., “Transplantability and therapeutic effects of bone marrow-derived mesenchymal cells in children with osteogenesis imperfecta,” Nature Medicine 5, 309-313, March 1999.
  4. B.D. Yandava, et al., “‘Global’ cell replacement is feasible via neural stem cell transplantation: evidence from the dysmyelinated shiverer mouse brain,” Proceedings of the National Academy of Sciences USA 96, 7029-7034, June 8, 1999.
  5. K. Tsubota, et al.,“Treatment of severe ocular-surface disorders with corneal epithelial stem-cell transplantation,” New England Journal of Medicine 340, 1697-1703, June 3, 1999.