Effective Anti-Cancer Drug Combo Found for Acute Myeloid Leukemia

Preclinical studies of a combination therapy with birinapant and p38 inhibitors show promise treating AML.

Patients with acute myeloid leukemia (AML) typically respond poorly to treatment, with less than one-third surviving for 5 years after a diagnosis. However, new data suggests that combinational drugs show promise for treating AML patients.

A study published in Cancer Cell reported positive results during preclinical studies of a combination therapy with birinapant and p38 inhibitors. This was discovered after researchers from the Walter and Eliza Hall Institute were exploring new ways to enhance birinapant’s anti-cancer effects.

"We are hopeful that the combination of these agents could be an effective anti-cancer treatment," said researcher Najoua Lalaoui.

P38 inhibitors and birinapant monotherapy have been used safely in human clinical trials.

“Birinapant has been used in clinical trials for several types of cancers,” added researcher John Silke. “Our latest research is part of an exciting next step, fine tuning how birinapant can be used in the clinic to enhance its anti-cancer effects.”

Since the standard high-dose chemotherapy has numerous toxic side effects, researchers are hopeful for the potential of this therapy.

“Our findings have made us hopeful that a combination of birinapant and a p38 inhibitor may be more effective in treating AML than current therapies, and also have less toxicity for patients," Lalaoui said. "We tested forms of AML that are highly resistant to chemotherapy and found that birinapant and p38 inhibitors could even kill these cancer cells, which is great news."

“We have had a long term interest in how IAPs function in healthy and diseased cells," Silke addeed. "Our research into how IAPs work made an important contribution to the initial development of birinapant as a specific IAP inhibitor.”