Chemotherapy Add-On Drug Removed From Market Shows New Promise: Study

A chemotherapy add-on drug taken off the market last year has been found to be effective in older adults with leukemia, according to study results presented at a conference this week.

"This new study is really quite tantalizing," Dr. Charles Abrams, American Society of Hematology secretary and associate chief of hematology/oncology at the University of Pennsylvania, said of Pfizer's Mylotarg. "This is a drug that wasn't studied fully enough." 

Pfizer Inc voluntarily stopped selling Mylotarg, known generically as gemtuzumab, after a study showed adding it to chemotherapy did not prolong survival for patients with acute myeloid leukemia (AML), and showed more deaths in the first few months of treatment with the drug. The drug contains an antibody targeting a protein found on the surface of AML cells and a cancer-killing toxin. AML causes abnormal cells to form in bone marrow. About 13,000 Americans get it each year.

A French study looked at newly-diagnosed patients from ages 50 to 70 given Mylotarg and chemotherapy. After two years, 41.4 percent were still alive, versus only 15.6 percent of patients who got chemotherapy alone.

Mylotarg patients survived 34 months overall, and patients who got chemotherapy alone survived 19 months.

Also among Mylotarg patients, the rate of fatalities was 8.7 percent, verus 6.7 percent for chemotherapy patients. Mylotarg patients had higher rates of low platelet levels and liver vein blockage. The trial "uses Mylotarg in a somewhat different fashion ... split over several doses," Dr. Mace Rothenberg, head of clinical development and medical affairs at Pfizer, told Reuters. "By using it in a slightly different way ... this drug is showing promise." Pfizer is talking with researchers about how to move forward with the drug, but is not yet talking to regulators, according to Reuters. Researchers announced the study this week in San Diego at a meeting of the American Society of Hematology.  
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