FDA Approves New Treatment for Leukemia

The FDA yesterday approved Novartis Pharmaceuticals Corp.'s Gleevec, the "first in a new class of designer cancer drugs" that target specific molecules while leaving "normal cells" alone, the Los Angeles Times reports. The drug is a treatment for chronic myleongeous leukemia, a "life-threatening" cancer in which the body produces too many white blood cells. CML traditionally has been treated with chemotherapy and bone marrow transplants, but Gleevec will allow patients to "avoid the horrific chemotherapy that has characterized most cancer treatments until now," the Los Angeles Times reports. Taken orally four times per day, Gleevec disrupts a signal the body receives to produce an enzyme that helps white blood cells "proliferate." Dr. Richard Klausner, director of the National Cancer Institute, said that Gleevec "represents a breakthrough" because it is the first to attack a target that is "not just in a cancer, but a target responsible for the cancer." The drug has relatively minor side effects, including mild nausea, puffiness around the eyes and muscle cramps. "This new drug, we believe, is the picture of the future of cancer treatment," Klausner said.

The drug appears to work best in people who are in the early stages of the disease, and seems "less effective" when given to people in the disease's advanced stages. Because the drug has been studied in humans for less than three years, scientists have cautioned that they still are unclear about the medication's long-term effects and whether it actually cures CML or "simply hold[s] it in check" (Cimons, Los Angeles Times, 5/11). Nearly 8,000 people currently are receiving Gleevec, which is expected to cost between $2,000 and $2,400 per month. Novartis President Daniel Vasella said that the company intends to give the drug either free or at a reduced cost to uninsured patients with annual incomes below $100,000 per year (Okie, Washington Post, 5/11). Gleevec is expected to hit the market within two weeks (Los Angeles Times, 5/11). The drug also seems to work in gastrointestinal stromal tumor cancer, which is usually incurable if surgery fails, and could be effective against cancers in which a protein called "platelet derived growth factor" is active.

At a press conference announcing the approval yesterday, HHS Secretary Tommy Thompson called the FDA's decision on the drug "an all-time [speed] record for a cancer drug." Generally, the FDA takes six months to approve drugs submitted for priority review, but in this case, the agency took two and a half months. The quick approval "rests on [the drug's] immediate and striking results" in patients with CML, the New York Times reports. In a trial of 54 patients, Gleevec caused 90% of the patients' blood counts to return to normal and 13% of patients' cancerous cells to "disappear altogether" (Wade, New York Times, 5/11). Dr. Brian Druker of the Oregon Health Sciences University -- which participated in the drug testing -- said, "As we enter this new era ... you're going to see more and more clinical trials like this with high response rates with minimal side effects, and that will go hand in hand with a more rapid approval process" (Hamilton, "Morning Edition," NPR, 5/11). An audio file of the NPR report is available at http://www.npr.org/programs/morning/ later this afternoon. For RealAudio of Thompson's announcement, go to http://audio.pbs.org:8080/ramgen/newshour/expansion/2001/05/10/thompson.rm?altplay=thompson.rm.