U. home to genetics advance

Today, most medicines are made by big pharmaceutical companies with large staffs and vast resources. But one day soon, according to experts, that same medicine might be produced in the human body using its own genetic make-up. A study published in this month's edition of the journal Science by researchers from the University of Pennsylvania Health System is moving that day closer. The study's research team, led by Molecular and Cellular Engineering Department Chairperson James Wilson, found a way to regulate the amount of medicine created by the body. Once the technique of gene therapy is perfected, doctors will be able to inject a gene into someone's body, which would then create the medicine at will. Until now, scientists have been unable to turn the medicine-production mechanisms "off" after inserting the gene. And too much medicine can be fatal. Wilson's experiment turned the switch off in rhesus monkeys -- raising hope that it could also be used in humans after scientists complete work on the solutions to a few problems. Such an advance would be advantageous for patients with chronic illnesses like diabetes who must routinely inject medicine into themselves. Using Wilson's technique, a patient receives a gene injection, which remains inactive until he takes a special pill. Another important advantage is that gene therapy allows the user to maintain a consistent level of medication in the body, unlike conventional medicine, which starts fast but then slows down. The new technique is a significant breakthrough, experts say. But despite the optimism, scientists must still find a way to make the technique work on humans. "The major next step is running clinical trials. That should take several years; this is all four or five years away from being completed," said Harvey Berger, the chief executive officer of the Cambridge, Mass.-based ARIAD Pharmaceuticals, which was involved in the study and hopes to market and sell the technique. Wivel, though, cautioned that the pill used in the experiments is harmful to humans, since it "suppresses immune response." But Wilson, who is also the director of the Institute for Human Gene Therapy at the Wistar Institute, maintained that he would be able to get around that problem by using a smaller dose or different kind of pill, on which scientists are currently working.

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