Gene Therapy and Cell Therapy

Neergaard, Lauran. "New gene therapy halts 2 boys' rare brain disease - Yahoo! News." Yahoo! News. The Associated Press, 5 Nov. 2009. Web. 13 Nov. 2009. http://news.yahoo.com/s/ap/us_med_gene_therapy.

Stem cell therapies, while still limited by legal and ethical restrictions, have advanced by leaps and bounds since the field’s inception. One of the most exciting advances in the field occurred recently: the combination of gene and cell therapy.

To treat adrenoleukodystrophy (ALD), a genetic disease that destroys the myelin coating of nerve fibers in boys’ brains, French scientists disabled the HIV virus so that it could not cause AIDS and used it to transplant bone marrow. Bone marrow transplants stop ALD by allowing new stem cells that can form myelin to take root. The problem with curing ALD, prior to now, has been finding a vector with which to transport the marrow. HIV is a disease that permanently invades all cells, making it a perfect vector. Scientists were able to genetically correct the boys’ bone marrow stem cells and implant them in the body by infecting them with deactivated HIV. Two years later, the boys’ brain damage shows no signs of worsening and 15% of their cells now produce the beneficial proteins received in the stem cell transplant.

What does this mean for the future of medicine? If this treatment can be replicated, then many similar diseases to ALD could also be halted – or even cured. The technological ability to correct patients’ bone marrow stem cells and return them to the patient via deactivated HIV is incredibly promising for treatment of cancer, as well as various genetic diseases. While ALD is too rare for a cure to have much sway in the current healthcare debates, the possibility of a cure for some strains of cancer is tantalizing. A cure for cancer would save hundreds of millions of dollars in Medicare and Medicaid every year. And if there’s one thing that lawmakers today can agree on, it is that we need to find ways to save money in these hard economic times. There is still a huge divide between policymakers on the ethical ramifications of stem cell research, but financially, stem cell research makes sense.

- Annie Bonaccorso