Karolinska initiating a study with generic medication that can provide more effective treatment of acute myeloid leukemia

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For many patients with acute myeloid leukemia (AML), chemotherapy doesn’t work because of resistance. Overcoming the resistance could be game-changing for this patient group. In Sweden, about 350 people a year are diagnosed with AML, and 1 of 4 have less than five year’s survival.

A research team has been testing a large number of substances to find something that can overcome drug resistance. They tested 33,000 molecules, including about 1,200 approved pharmaceuticals, before they found hydroxyurea, which is currently used in treating sickle-cell anemia.

"In the spring we will be moving on with a clinical trial, and it helps that the medicine isn't new, is relatively inexpensive and has multiple manufacturers," says Nikolas Herold, researcher and resident physician at the Astrid Lindgren Children's Hospital.

"We are going to monitor the patients for five years to see how their survival is affected. The preliminary studies have shown good outcomes and I hope we can cure more patients."

The study will include 69 patients who were recently diagnosed with AML. They will be given hydroxyurea along with their chemotherapy. Bone marrow samples will be taken before and after the treatment to measure the number of leukemia cells and determine the efficacy of the treatment.

This is a national study organized by Karolinska University Hospital and Region Stockholm, in close collaboration with the Svenska AML-gruppen.

Nikolas Herold has previously shown that resistance to chemotherapy can be caused by leukemia cells expressing a specific protein (SAMHD1).

Now he and his research team announce in an article in EMBO Molecular Medicine that they have identified the generic pharmaceutical hydroxyurea as a possible treatment to overcome resistance to chemotherapy. The article also describes how the drug works to overcome resistance.

Read the article in EMBO here

Facts

Acute myeloid leukemia (AML), also called myeloblasts or myeloid leukemia, is one of the most common types of leukemia in adults and children.

In Sweden, about 350 people a year are diagnosed with AML.

The risk of developing it increases with age after 40.

The diagnosis is based on finds of abnormal cells in the blood and bone marrow at the same time as a decrease in the number of normal red and white blood cells and platelets.

It is important that treatment is started more or less immediately after diagnosis. The primary symptoms are tiredness, a general feeling of unwellness and general symptoms.

Although the gender distribution is even, in the elderly more men than women develop the disease.