Re-engineering Your Body (page 4 of 5)

Battling Genetic Disease

Personalized genetic diagnosis and therapy. Say you're young and healthy, and you go in for a routine physical. Your doctor takes a blood sample and has it shipped to a lab. There, a medical technologist places your serum sample on a glass chip the size of a postage stamp. That gene chip might contain up to 50,000 microscopic spots -- each with one of the genes in the human genome. When the doctor calls you with the results, she'll tell you which of thousands of human diseases you're at risk for. If you have a defective gene that's placing you at risk for disease, she might treat you with a healthy version of the gene to make up for it, keeping you out of harm's way.

Soon, such diagnoses and treatments could be routine, says Mark Kay, MD, a professor of genetics at Stanford University School of Medicine and president of the American Society of Gene Therapy. "In five years, you may be able to go to referral centers and get gene therapy," he says. Although gene therapists have talked like that for a while, and the field has tremendous promise, so far they have demonstrably cured humans of just one disease: severe combined immunodeficiency (SCID). Known as the bubble-boy disease, it decimates the immune system and causes children to die young from infections. While the treatment looks promising, the virus used to deliver the gene in one trial may have activated a gene that causes cancer.

Is it safe? Such safety issues have dogged gene therapy. But gene therapists are pressing on. More than a dozen advanced clinical trials are underway that use genes to treat a variety of cancers, and other trials are ongoing for multiple sclerosis, AIDS and cystic fibrosis. Dr. Losordo has also begun a large trial of a gene therapy that seems to help patients regrow blood vessels that supply the heart -- "grow your own bypass, if you will," he says. "It's a very exciting time."

Nanotechnology. To get around the safety problems in gene therapy -- as well as the side effects of conventional drug therapies -- scientists have begun tapping nanotechnology for solutions. Most nanotechnology-based therapies being studied now remain at least five years from the clinic, but scientists have begun using them to tackle cancer in mice. Robert Langer, ScD, a professor of chemical engineering at MIT, and Daniel Anderson, PhD, a research associate, have taken a plastic-like molecule called a polymer, much smaller than a cell, wired it with a gene for a bacterial toxin, and sent it, like a suicide bomber bent on destruction, into a nasty human prostate tumor growing in a mouse. When all was said and done, the suicide polymers had shrunk 40 percent of the tumors and slowed or stopped the rest.

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