Perhaps nothing known to modern science is as unlikely and outrageous as the daily work of our genes. From food, water, and air, they make a human being. Genes make proteins. Proteins are us.
And from this audacious process come humans, who are themselves audacious. Not content simply to sit back and marvel at this infinitesimal artistry, people seek to control and improve it. Medical manipulators aim to insert a normal gene in place of a defective one, or perhaps more boldly, to insert an “improved” gene in place of a normal one.
So far, the Food and Drug Administration has not approved any human gene-therapy product for sale, but the day is coming. In 2001 Philippe Leboulch of MIT and Harvard University used gene therapy on mice afflicted with sickle-cell anemia and wrought what may prove to be the technique’s first outright cure. Human trials are about one year away.
In the future, some hope, diseases will have nowhere to run; they will be flanked and overwhelmed by the traditional top-down approach of drugs and surgery and the bottom-up techniques of genetic therapy. Little wonder, then, that optimism reigns in medical schools. “Students used to say, ‘I want to understand basic biology,’” says Tom Curran, a former president of the American Association of Cancer Research. “Now they are saying, ‘I want to cure something.’”
Despite the progress, they won’t lack for challenges. The looming riddles of medical research are still overwhelming and will require extraordinary amounts of money, as well as brainpower, to solve. Here are eight unanswered questions that leading medical researchers say will command most of the attention and funding in the next decade.
