Studies highlight new drug targets or compounds for acute myeloid leukemia

December 07, 2015

ORLANDO, FL (Dec. 7, 2015) - Preclinical data unveiled across four studies presented at the 57th annual meeting of the American Society of Hematology highlight four potential treatment opportunities for acute myeloid leukemia (AML), a blood cancer accounting for approximately 20 percent of all childhood leukemias and 32 percent of adult leukemias. The four studies, led by investigators from Dana-Farber/Boston Children's Cancer and Blood Disorders Center, represent significant progress in seeking out and targeting multiple vulnerabilities within AML cells, including aspects of the cells' metabolism, internal communications and ability to transport proteins between different compartments.

The overall survival rate for pediatric AML is between 60 and 70 percent -- above 80 percent for some specific subtypes. For adult AML, the numbers are far worse; overall survival is 45 percent, and goes down dramatically with age. Both pediatric and adult AML frequently relapse, generally in a treatment-resistant form. Thus, there is a significant need for treatment options that can improve the survival rate and can effectively treat patients with relapses when they do occur.

"When you look at overall survival and how we treat most patients with AML, things have changed remarkably little in the last 20 years," said Kimberly Stegmaier, MD, a pediatric hematologist/oncologist at Dana-Farber/Boston Children's and senior investigator on three of the four studies. "We still use older chemotherapy drugs in both adults and children that are very toxic, and we have quite a way to go to cure all patients with this disease.

"In the last few years, target and molecule discovery have really accelerated," Stegmaier continued, "which for continued progress in treating patients with AML is essential. "To make headway against those forms of AML resistant to current therapy, we will need treatments that are fundamentally different from those in our current armamentarium."

The four Dana-Farber/Boston Children's AML studies, each of which focuses on a different aspect of AML biology, are:

The targets and molecules being presented represent just a few of the opportunities created by the last decade's efforts to tease apart the complicated genomics of cancer in general, and AML in particular.

"AML led the genomic sequencing revolution in cancer. The first cancer genome sequenced was from a patient with AML," Stegmaier explained. "We have learned a great deal since then. There are a number of new treatment approaches being tested in the clinic that are very exciting. These abstracts reflect just a few of the emerging targets of great interest."
About Dana-Farber/Boston Children's Cancer and Blood Disorders Center

The Dana-Farber/Boston Children's Cancer and Blood Disorders Center -- the nation's #1 pediatric cancer program, according to US News & World Report 2015-16 -- brings together two internationally known research and teaching institutions that have provided comprehensive care for pediatric oncology and hematology patients since 1947. The Harvard Medical School affiliates share a clinical staff that delivers inpatient care and surgery at Boston Children's Hospital, outpatient oncology care at Dana-Farber Cancer Institute and outpatient blood disorders care at Boston Children's.

Dana-Farber Cancer Institute

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