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Beat AML proved that a newly diagnosed leukaemia can be fully genetically typed within a week and the patient started on a drug aimed at their particular mutation, instead of the same chemotherapy for everyone. Older patients treated this way lived longer than those given standard care.
The Beat AML Master Trial opened in November 2016, sponsored by the Leukemia & Lymphoma Society through a dedicated company and led by John Byrd with Brian Druker's Beat AML research programme and Ross Levine among its scientific leaders. Patients aged 60 or older with suspected new acute myeloid leukaemia consent before diagnosis is confirmed; bone marrow is sent for cytogenetics and next-generation sequencing, and a treatment assignment is returned within seven days according to a hierarchy of mutations (FLT3, IDH1, IDH2, TP53, core-binding factor, NPM1 and others), each with its own phase 1b or 2 sub-study of a targeted drug such as gilteritinib, ivosidenib or enasidenib, often combined with azacitidine or later venetoclax.
The feasibility and early efficacy report in Nature Medicine in 2020 covered the first 487 patients: results came back within seven days for nearly all, most eligible patients could be assigned to a sub-study, and those treated on Beat AML arms had a median overall survival of 12.8 months compared with 3.9 months among patients who chose standard care, with the caveat that the comparison was not randomised. Sub-studies have since reported individually, including combinations for TP53-mutant and complex-karyotype disease, and the trial has expanded to include younger patients and new drug classes such as menin inhibitors.
Beat AML changed the timetable of leukaemia care: it showed that waiting a week for genomics is safe in most older patients and that assignment by mutation at diagnosis is workable across many centres, which is the premise of the NCI's myeloMATCH. Whether the individual sub-study drugs improve survival against modern standards such as azacitidine with venetoclax is being answered arm by arm.
Numbers are from the trial as recorded here; see the source links in the table below. This is orientation, not medical advice: ask your team how closely the trial population matches you.
Shares Gilteritinib, BCL-2 inhibitors, Azacitidine, Venetoclax.
Shares IDH inhibitors, Ivosidenib, Azacitidine, Acute myeloid leukaemia.
Shares BCL-2 inhibitors, Azacitidine, Venetoclax, Acute myeloid leukaemia.
Shares Master protocol (platform, basket and umbrella trials), Basket, umbrella, and platform trials, Trial design, endpoints and cost, Comprehensive genomic profiling.
Shares IDH inhibitors, Ivosidenib, Azacitidine, Acute myeloid leukaemia.
Shares BCL-2 inhibitors, Azacitidine, Venetoclax, Acute myeloid leukaemia.
Shares BCL-2 inhibitors, Azacitidine, Venetoclax, Acute myeloid leukaemia.
Shares BCL-2 inhibitors, Azacitidine, Venetoclax, Acute myeloid leukaemia.