Acute myeloid leukemia

Rapid genetic screening shows feasibility of precision medicine

Jatros Digital, 03.12.2018
Onkologie | Hämatologie

A new study demonstrates it is feasible – and helpful – for doctors to rapidly determine which molecular subtype of acute myeloid leukemia a patient has before beginning treatment and to use this information to pick an approach that best matches the individual.

Because acute myeloid leukemia (AML) is a rapidly progressing cancer, treatment is typically started on the day of diagnosis and physicians have been reluctant to wait the two to three weeks that it typically takes for genomic analysis. This leaves doctors with little time to learn which AML subtype the patient has, so in current practice, all patients are given the same treatment regimen.

However, in this study, researchers demonstrate the ability to determine AML subtype based on genetic analysis of blood samples in seven days or less. The findings suggest rapid genetic screening could soon be an integral part of AML diagnostics in order to match each patient with the therapy best suited for his specific disease.

The results represent the first findings from the Beat AML study, a multi-arm, multi-site collaborative trial designed to test precision medicine approaches for improving the generally poor prognosis among patients with AML. In recent years there have been renewed efforts to improve the standard treatment for AML, which has not significantly advanced in about four decades. Many experimental new therapies target specific AML subtypes, so the ability to determine a patient’s AML subtype is crucial to realizing the full benefits of these therapies, said lead study author Amy Burd, PhD, vice president for research strategy at The Leukemia & Lymphoma Society (LLS).

The reported findings include data from 365 patients over age 60 with suspected or confirmed AML who enrolled in the study from November 2016–2018. The researchers applied three genetic analysis techniques – cytogenetics, polymerase chain reaction (PCR), and next-generation sequencing (NGS) – to patient samples to create a genetic profile of each patient’s disease. 66 patients were removed from the study because they turned out to not have AML upon laboratory analysis.

To date, 146 patients have continued on to the study’s second phase, in which they were treated on a clinical trial for experimental AML therapies targeting their disease subtype. The remaining patients did not continue on to the study’s second phase for a variety of reasons, including choosing standard AML therapy after molecular profiling, enrolling in a different trial, or deciding not to pursue therapy. Waiting for a few days to start therapy after receiving a diagnosis also gave patients time to make informed decisions about their therapy, something the former approach did not allow. „This supports a patient-centric approach,‟ said Dr. Burd.

The study is designed to incorporate new experimental therapies as they are developed. Having started in 2016 with three treatment arms, the study has now grown to include 11 arms testing therapies developed by seven different pharmaceutical companies. Initial results from some of these studies suggest patients benefit from therapies specifically chosen based on their individual disease subtype.

Burd A et al.: Initial report of the Beat AML Umbrella study for previously untreated AML: evidence of feasibility and early success in molecularly driven phase 1 and 2 studies. ASH Annual Meeting 2018, abstract #559