Researchers reported a number of important findings in leukemia treatment at the 2018 Annual Meeting of the American Society of Clinical Oncology:

Targeted therapy ivosidenib safe and effective in IDH1-mutated acute myeloid leukemia

A phase I trial showed the IDH1 inhibitor ivosidenib to be safe and effective in treating patients with advanced relapsed or refractory (not responding to treatment) acute myeloid leukemia (AML) that has a mutation in the IDH1 gene.

Ivosidenib, a targeted therapy, achieved durable (long-lasting) responses in some patients and reduced or eliminated patients’ dependence on transfusions, whether or not those patients achieved a durable response.

What Patients Need to Know

Ivosidenib is the second IDH inhibitor to show promise in the treatment of AML. Some people with AML have a mutation in the IDH2 gene that stops cells from maturing in the way they should. Approved by the FDA in August 2017, the IDH inhibitor enasidenib works by helping leukemia cells mature into normal cells.

Venetoclax combined with chemotherapy studied for untreated acute myeloid leukemia in elderly patients

Data from a phase Ib dose-escalation trial showed that the targeted therapy venetoclax, given in combination with the chemotherapy azacitidine, led to durable responses with a tolerable safety profile in elderly patients with untreated AML.

What Patients Need to Know

Many people with AML have a mutation of the BCL2 gene that prevents the death of cancer cells and is associated with a resistance to chemotherapy. Venetoclax, a BCL2 inhibitor, is designed to correct this mutation.

Phase III trial shows CPX-351 to be more effective than standard chemotherapy in treatment of acute myeloid leukemia

A common chemotherapy regimen for AML is called “7 + 3”; it consists of the chemotherapy cytarabine given daily for 7 days, followed by an anthracycline given daily for 3 days. This treatment is administered intravenously (into a vein) and in an in-patient hospital setting.

A phase III trial showed CPX-351, a combination of cytarabine and the anthracycline daunorubicin, to be more effective than “7 + 3” chemotherapy in the treatment of AML.

What Patients Need to Know

In August 2017, the FDA approved CPX-351 (brand name Vyxeos) for the treatment of newly-diagnosed therapy-related AML (t-AML). It was also approved for the treatment of AML with myelodysplasia-related changes, which cause the body to stop making enough healthy red blood cells and platelets.