Precision medicine in the treatment stratification of AML patients: challenges and progress

// Ines Lohse 1, 2, 3 , Kurt Statz-Geary 2 , Shaun P. Brothers 1, 2, 3 and Claes Wahlestedt 1, 2 1 Center for Therapeutic Innovation, Miller School of Medicine, University of Miami, Miami, FL, USA 2 Department of Psychiatry and Behavioral Sciences, Miller School of Medicine, University of Miami, Miami, FL, USA 3 Molecular Therapeutics Shared Resource, Sylvester Comprehensive Cancer Center, University of Miami, FL, USA Correspondence to: Ines Lohse, email: ixl180@med.miami.edu Claes Wahlestedt, email: cwahlestedt@med.miami.edu Keywords: AML; ex vivo drug sensitivity screens; omics-based screens; phenotypic screens; precision medicine Received: September 25, 2018      Accepted: December 10, 2018      Published: December 28, 2018 ABSTRACT Recent advances in high throughput technologies have led to the generation of vast amounts of clinical data and the development of personalized medicine approaches in acute myeloid leukemia (AML). The ability to treat cancer patients based upon their individual molecular characteristics or drug sensitivity profiles is expected to significantly advance cancer treatment and improve the long-term survival of patients with refractory AML, for whom current treatment options are restricted to palliative approaches. The clinical development of omics-based and phenotypic screens, however, is limited by a number of bottlenecks including the generation of cost-effective high-throughput data, data interpretation and integration of multiple approaches, sample availability, clinically relevant timelines, and the development and education of multidisciplinary teams. Recently, a number of small clinical trials have shown survival benefits in patients treated based on personalized medicine approaches. While these preliminary studies are encouraging, larger trials are needed to evaluate the utility of these technologies in routine clinical settings.