Over its lifespan, the drug has starred in a drama with enough plot twists to rival a soap opera.
When gemtuzumab ozogamicin (Mylotarg) arrived on the scene in 2000, it was the first so-called “magic bullet” drug on the market, a targeted therapy that steers a cell-killing toxin to leukemia cells.
Then, 10 years later, it racked up another, more ignominious first. After a more extensive FDA-required clinical trial showed no clear evidence of the drug’s benefit to patient survival, it was the first medication to be withdrawn from the market after having been authorized through the FDA’s accelerated approval program, which is designed to speed urgently needed therapies to patients.
Now, new evidence may be bringing it back to life. Researchers are zooming in on a specific group of patients who could benefit from it in the latest example of the promise of precision medicine in cancer care. And its story offers important lessons about how we study new therapies, said one of the scientists whose research has helped lay the foundation for its reemergence.
“Our search for one drug that cures everything is really misplaced,” said pediatric leukemia specialist Dr. Soheil Meshinchi of Fred Hutchinson Cancer Research Center, where the drug was first developed more than two decades ago. “The drugs that failed in the past, [it] may not have been the drug’s failure but our failure to match them to the appropriate target.”