Thirty-five years ago, as a fellow at the MD Anderson Cancer Center in Houston, Texas, Elihu Estey read dozens of protocols for clinical trials of drugs for acute myeloid leukaemia (AML). He was particularly drawn to their ‘rationale’ sections, which explain why the therapy is expected to work.
“They all sounded very compelling,” says Estey, now a haematologist at the University of Washington in Seattle. “But of course, very few of them worked.”
Since then, researchers have made significant advances in treating many types of leukaemia. However, AML — an aggressive blood cancer that causes white-blood-cell precursors called myeloid cells to proliferate uncontrollably — has remained a tough nut to crack.