Posted on May 10, 2001 at 4:20 p.m.
BIRMINGHAM, AL — The U.S. Food and Drug Administration (FDA) is expected to grant approval soon for the marketing of a new cancer drug whose design is a “peek into the future” of drug development, according to UAB’s Dr. Peter Emanuel.
Emanuel, a cancer doctor, has entered some of his patients into a large clinical trial of Gleevec, made by Novartis Pharmaceuticals. Gleevec targets the genetic mutation responsible for chronic myelogenous leukemia (CML), one of four main types of leukemia. “The drug has achieved early success with CML, and it has potential for a limited application to other cancers,” he said. “It appears to at least buy these patients some time and unlike other options causes virtually no side effects.”
Gleevec’s progression from clinical trial to the marketplace would be one of the fastest ever, Emanuel noted. It was discovered in 1993 with clinical trials starting only in June 1998. “Its greatest importance, however, is that it would be a model for drugs designed from use of data mined from the Human Genome Project.”
The genetic error thought to cause CML was discovered long before the human genome was mapped, but is an example of how the genome project is expected to be used, he said. Drugs such as Gleevec offer precision treatment based on data learned from three decades of research into the basic biology of cancer. It targets an intracellular signaling molecule that has gone awry.
Although doctors have called results “striking” and “remarkable”, Gleevec has not been used long enough to provide direct evidence that it prolongs life. An editorial in last month’s New England Journal of Medicine cautioned that “important clinical problems could still emerge.” Still, the prestigious journal was otherwise optimistic in its evaluation of the new drug.
Development of Gleevec is noteworthy for another reason, Emanuel said. “Amazingly, nearly 6,000 patients worldwide have been entered into clinical trials of this product in only 18 months.”
The drug company initially was hesitant to conduct expensive, large-scale studies based on positive results from an initial trial of 24 patients, he noted. But in December 1999 the company reported that 23 of the participants had achieved complete remission after the treatment. Partially because of pressure from patients demanding the drug, Novartis increased the supply and the FDA put it on the fast-track process leading to its expected approval in the next few weeks.