Gleevec, a once-a-day pill turns chronic myelogenous leukemia, or CML, from a certain death sentence into a manageable disease. Data shows that it has helped 83 percent of patients live 10 years or longer, even with side-effects that include a characteristic rash, fatigue and nausea. Before Gleevec was introduced to the market, CML patients had three options: to receive treatment with toxic chemotherapy, a bone marrow transplant, or just dying. Even with treatment, patients rarely lived longer than three years. “It was really a death warrant,” said Dr. Richard Silver, a hematologist and oncologist at New York Presbyterian-Weill Cornell Medical Center who helped first test the drug in patients.
“It’s the first targeted personalized medicine that had ever been used. It was also the most successful,” said “This has been the thrill of my life,” Silver told NBC News.
On Thursday, the original team of researchers reported in the New England Journal of Medicine that some patients may be able to stop taking the pills altogether, even though they are not cured. Gleevec worked so well and so quickly that the trial testing the drug against the older chemo regimen was stopped so everyone could get the pill. The FDA’s approval was swift and now the Leukemia & Lymphoma Society estimates 36,000 to 100,000 Americans are CML survivors.
The researchers, led by Dr. Brian Druker of Oregon Health & Science University, published their final report Thursday on the original Gleevec trial, which had 1,100 patients.
The drug was the first to be designed to match the particular genetic mutation that causes CML. Up to then, most chemotherapy went after rapidly dividing cells — a hallmark of cancer, but an approach that also damages hair follicles, the inside of the mouth, the lining of the intestines and other healthy tissue.
Gleevec is targeted to a mutation specific to CML. Now targeted therapies are common and can have remarkable results in a small group of patients with specific genetic mutations in their tumors. With CML, the same genetic changes affected almost every patient.
“It’s a 10-year survival of 83 percent, which is extraordinary,” Silver said. “It has led to what we call biologic cures.” Patients still have leukemia, but it’s not affecting their blood cell counts.
In Europe, doctors are beginning to recommend that patients who are doing well on the drug for a year or longer to try stopping. Dr. Druker and colleagues believe that around 10 percent of all Gleevec patients will be able to safely stop taking the drug, and 40 percent or more of those who show a quick response to the drug.