Study Involving CHOP Researchers Could Lead to More Precise Treatment for Acute Myeloid Leukemia

Published on in CHOP News

A new study involving researchers from Children’s Hospital of Philadelphia has identified immune biomarkers in patients with acute myeloid leukemia (AML) that can help predict which patients will be resistant to chemotherapy and which might benefit from a new immunotherapy. The findings were published today in Science Translational Medicine.

AML is a quickly-progressing, aggressive cancer in both children and adults with a cure rate of only 35 to 40% in patients under 60 years of age and 5 to 15% in patients over 60. Chemotherapy resistance is common, and both children and adults with AML have high risk of relapse, which accounts for the majority of patient deaths.

Although immunotherapy has potential for use in patients with AML, currently there is no way to predict which groups of patients or forms of leukemia will respond to immune targeting therapy. To help close that knowledge gap, the research team used targeted gene expression profiles and other sophisticated technologies to analyze the bone marrow of large cohorts of both adult and pediatric AML patients. Their analysis revealed distinct immune subtypes, which could help deliver predictions of chemotherapy resistance, survival, and immunotherapy response and allow physicians to tailor treatment based on a patient’s immune profile.

In particular, the research revealed immune biomarkers that indicate whether a patient might respond well to flotetuzumab, a new bispecific antibody therapy that both recognizes tumor cells and activates T cell production, bringing the T cells close to the tumor to destroy the cancer cells.

“These collaborative efforts and research findings provide real hope for successful actualization of new immunotherapies to overcome traditional chemotherapy resistance and increase cure rates in patients with AML. We are also excited to be studying flotetuzumab now specifically in children with relapsed/refractory AML via our recently-opened PEPN1812 phase 1 clinical trial,” said study co-author Sarah K. Tasian, MD, an Attending Physician and Assistant Professor of Pediatrics in CHOP’s Division of Oncology and Center for Childhood Cancer Research.

You can read more about the study, which was led by researchers at Nottingham Trent University in the United Kingdom, here.

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