Diabetes Drug Kills Leukemia, Regenerates Healthy Blood Cells

PPARγ agonists may improve outcomes for patients with acute myeloid leukemia.

Traditional cancer treatment affects both cancerous cells and healthy cells, which can cause toxicity. Treatments such as immunotherapy have come to the market over the past few years that seek to preserve healthy cells and drive optimal patient outcomes. A new approach could kill acute myeloid leukemia (AML) cells, while regenerating healthy blood cells as well, according to a study published by Nature Cell Biology.

The investigational approach boosts adipocytes located in the bone marrow, which can lead to anti-cancer activity and restore healthy cells, according to the study authors.

Healthy red blood cells are crucial for patients with AML, but current therapies focus only on killing cancer cells. These patients typically develop anemia and infection due to a lack of proper blood cell production, which leads to hospitalization and mortality, according to the authors.

“Our approach represents a different way of looking at leukemia and considers the entire bone marrow as an ecosystem, rather than the traditional approach of studying and trying to directly kill the diseased cells themselves,” said first author Allison Boyd, PhD. “These traditional approaches have not delivered enough new therapeutic options for patients. The standard-of-care for this disease hasn’t changed in several decades.”

The authors collected bone marrow samples from large cohorts of patients with AML. The investigators studied and imaged leukemia cells and compared it with healthy cells in the bone marrow, which showed the effects of targeting fat cells.

A common class of diabetes drugs—PPARγ agonists—were found to increase fat cell production in the bone marrow, which increased healthy red blood cell production and suppressed cancer cells, according to the study.

“The focus of chemotherapy and existing standard-of-care is on killing cancer cells but instead we took a completely different approach which changes the environment the cancer cells live in,” said lead researcher Mick Bhatia, PhD. “This not only suppressed the ‘bad’ cancer cells, but also bolstered the ‘good’ healthy cells allowing them to regenerate in the new drug-induced environment.”

With many individuals diagnosed with AML each year, more effective therapies are needed. These results suggest that by normalizing the production of healthy blood cells, patients may have better outcomes.

“We can envision this becoming a potential new therapeutic approach that can either be added to existing treatments or even replace others in the near future,” Dr Bhatia said. “The fact that this drug activates blood regeneration may provide benefits for those waiting for bone marrow transplants by activating their own healthy cells.”