Acute myeloid leukemia, often called AML, is one of the most aggressive blood cancers. Every year, more than 20,000 people in the United States are diagnosed with this disease.
AML grows quickly and mainly affects older adults, although it can occur at any age.
Despite advances in treatment, long-term survival rates remain low, and many patients face relapse after an initial response to therapy. This makes AML one of the most challenging cancers for doctors and patients alike.
In recent years, a drug called venetoclax has become a key part of treatment for AML. Approved in 2019, venetoclax is often given together with another drug, azacitidine. This combination helps many patients achieve remission and improves quality of life, especially for those who cannot tolerate strong chemotherapy.
However, there is a major problem. Almost all patients eventually stop responding to venetoclax. The cancer finds ways to survive, and once resistance develops, treatment options become limited.
Now, scientists at Oregon Health & Science University may have found a way to break through this resistance. In a study published in Cell Reports Medicine, the research team tested hundreds of patient samples and discovered that combining venetoclax with another drug, palbociclib, produced much stronger and longer-lasting effects than venetoclax alone.
Palbociclib is already approved to treat breast cancer, which makes this finding especially exciting because it opens the door to faster clinical testing.
The researchers examined more than 300 AML patient samples and tested 25 different drug combinations. Among all the options, the pairing of venetoclax and palbociclib stood out clearly.
It reduced leukemia cell survival far more effectively and worked even in cases where venetoclax alone failed. These results were confirmed not only in patient-derived cells but also in mice that had been implanted with human leukemia cells.
To understand why this combination works so well, the scientists looked closely at how leukemia cells react to treatment. When AML cells are treated with venetoclax alone, they try to adapt. One of their survival tricks is to increase protein production inside the cell. This helps the cancer cells stay alive even under drug pressure.
Palbociclib blocks this escape route. It interferes with the cell cycle and slows down the machinery that makes proteins, leaving the leukemia cells with fewer ways to survive.
The team found clear genetic evidence to support this idea. Cells that responded well to the drug combination showed lower activity in genes linked to protein production.
Additional experiments using advanced genetic tools confirmed that the two drugs work together to shut down several survival systems at the same time. This makes it much harder for the cancer to adapt.
The most striking results came from animal studies designed to mimic real-world drug resistance. In mice carrying human AML cells that were known to resist venetoclax, treatment with venetoclax alone did not extend life at all.
But when palbociclib was added, most of the mice lived for nearly a year. One mouse was still alive when the study ended, which is extremely rare in this aggressive disease model.
This research also carries a deeply personal meaning for the lead author, who is herself a cancer survivor. Her experience as a patient helped fuel her motivation to follow the data and push the research forward. The study is also part of a larger national effort to improve AML treatment by using real patient data to guide drug discovery.
The findings highlight an important lesson in cancer research. Drugs developed for one type of cancer can sometimes work in very different diseases because cancer cells often share common survival strategies. Palbociclib was originally designed for breast cancer, but its ability to block cell growth makes it useful in other settings as well.
When reviewing and analyzing these results, several points stand out. First, the study is strong because it used a large number of patient samples and multiple testing systems, including animal models.
Second, the drug involved is already approved for human use, which lowers safety barriers and shortens the path to clinical trials. Third, the combination directly targets known resistance mechanisms, addressing one of the biggest problems in current AML treatment.
At the same time, caution is still needed. The combination has not yet been tested in people with AML, and human trials will be necessary to confirm safety, dosing, and real-world effectiveness. Side effects may also differ when the drugs are used together. Still, based on the evidence so far, this research represents a meaningful step forward.
If future trials confirm these findings, this drug pair could significantly improve outcomes for patients who currently have very limited options. For a disease where progress has been slow and survival rates remain low, this discovery offers genuine hope that smarter, data-driven combinations can finally change the course of AML treatment.
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