Gene Therapy With Stem Cells

Gene therapy has its challenges, but could be done for virtually any type of genetic disease that has been discovered. Genetic therapy using stem cells would currently need to be done by contract with an individual patient (which basically means the sufferer would need to pay for it) since many genetic conditions are rare and thus it is not commercially feasible to manufacture one particular gene transfected stem cell in hopes that patients will line up to get the therapy. This is a sad, but true state of life. But hopefully with stem cell therapy becoming increasingly used, studied and available, all sufferers will have access. One example of a rare genetic disease is Progeria, a "rapid aging disease" in children, with only 42 known cases spread out over 20 countries.

A somewhat more common genetic ailment is cystic fibrosis. It is estimated that there are over 60,000 sufferers worldwide, and half of them reside in the United States. Consequently, a fair amount of research has been done for this genetic disorder. One group of studies showed that patients with cystic fibrosis (CF) could potentially be benefitted using their own stem cells that have been maneuvered by gene therapy. In fact, in 2004, researchers at five different institutions (including the University of Pittsburgh School of Medicine) demonstrated that human bone marrow-derived adult stem cells can be persuaded to differentiate into airway epithelial cells. Furthermore, by programming these cells with the gene that is defective in CF it was shown to restore an important cellular function necessary for keeping the airways clear of mucus and air-borne irritants. [Combined stem cell-gene therapy approach seen as potential treatment for cystic fibrosis Contact: Lisa Rossi, University of Pittsburgh RossiL@upmc.edu]

It is not currently legal in the United States, except in research, or in a limited number of diseases (mostly cancer) to use gene transfected stem cells on humans. A veterinarian can do it for your dog, but your doctor can't do it for you. This, however, is the reason we need to keep plunging ahead with the use and research of adult bone marrow and umbilical cord stem cells for all manner of disease. The stigma against the free and open use of stem cells is because people don't know the difference between embryonic stem cells and adult cells -and because of the questionable safety or efficacy record of stem cells that has little or nothing to do with the stem cells, and everything to do with the dangerous and devastating immunosuppressive therapies hospitals and researchers do prior to administering the stem cells.

That said, genetically engineered umbilical cord stem cells are being used in Mexico for MS and ALS and have been able to help patients slow the degenerative progression. See www.stemcelltherapies.org