Drug Under Investigation Shows Treatment Efficacy In Acute Myeloid Leukemia (AML)

Investigational drug when combined with chemotherapy shows clinical efficacy in relapsed/refractory acute myeloid leukemia (AML) patients.

Investigational drug along with chemotherapy may provide benefits in patients with relapsed/refractory acute myeloid leukemia (AML) as per a study, published in the journal Blood Cancer Discovery, a journal of the American Association for Cancer Research.

Adult acute myeloid leukemia (AML) is a type of blood and bone marrow cancer where the bone marrow produces abnormal myeloblasts (a type of white blood cell), red blood cells, or platelets. This condition may worsen quickly if untreated.

Acute Myeloid Leukemia-Causes-Symptoms-Diagnosis-Treatment-Prognosis-FAQs
Acute myeloid Leukemia, more popularly known by its abbreviated form AML, is a fast- evolving leukemia that affects both children and adults alike

‘Investigational drug – ficlatuzumab (a first-in-class monoclonal antibody) shows clinical efficacy in relapsed/refractory acute myeloid leukemia (AML) patients when combined with chemotherapy. This may help in treating the patients diagnosed with this very dangerous disease and provide new hope for life.’

Burden of AML It is reported that only about half of patients with AML achieve long-term disease control. Many others are treated with multi-agent chemotherapy as they fail to respond to initial therapy.

"Unfortunately, patients whose cancers relapse or don't respond to initial therapy face a poor outlook, as only 30 to 40 percent of these patients respond to subsequent multi-agent chemotherapy and even fewer develop long-term remissions. Most patients will eventually succumb to their disease," says senior author Charalambos Andreadis, MD, professor of clinical medicine at the University of California, San Francisco (UCSF).

Several therapies have been developed recently for targeting AML-specific genetic mutations. But the drugs prove effective for only a certain group of patients. Hence there is a need for widely applicable AML therapies.

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The Investigational Drug

The present study examined the efficacy of the investigational drug – Ficlatuzumab (a first-in-class monoclonal antibody) in 17 adult patients with refractory AML. They were administered four doses of Ficlatuzumab 14 days apart, along with the chemotherapeutic drug – cytarabine.

Combination Therapy May Help Treat Acute Myeloid Leukemia
Synergistic effect of two drugs that are potent against acute myeloid leukemia (AML) when combined, but only weakly effective when used as single-agent therapy.

It was shown that Ficlatuzumab shows clinical efficacy in 9 out of 17 relapsed/refractory AML patients (53%) who had a complete response when combined with chemotherapy. A progression-free survival was 31.2 months were reported in the patients.

The patients with refractory AML have higher levels of an extracellular circulating factor called hepatocyte growth factor (HGF). The drug is shown to bind with the HGF to prevent tumor growth rather than targeting a cancer-specific mutation.

Acute Myeloid Leukemia: Key Discovery Could Lead to New Treatments
Acute myeloid leukemia stem cells lose their leukemic properties once the transcription factor is knocked down or deleted, said researchers.

"Together, our findings suggest that targeting an extracellular factor in conjunction with existing cancer therapies could be an effective therapeutic strategy for AML treatment," says Andreadis.

Source-Medindia