Scientists Find New Way to Fight Rare Blood Cancer

Scientists just figured out why a devastating blood cancer keeps fighting back, and they found a way to stop it.

Researchers at MetroHealth and Case Western Reserve University discovered that myelofibrosis, a rare bone marrow cancer, has a hidden backup system that keeps it alive even when patients take their prescribed drugs. The team identified two existing medications that could shut down this survival mechanism.

In myelofibrosis, scar tissue builds up in the bone marrow, causing the spleen to swell and making it hard for the body to produce healthy blood cells. Most patients take JAK2 inhibitor drugs that help them feel better, but these medications don't cure the disease and often lose their effectiveness over time.

The research team, led by Dr. Huiqin Bian and Dr. Shujun Liu, discovered why these drugs fail. Cancer cells have a secret communication pathway that acts like a backup generator, keeping the disease growing even when standard treatments are working.

The scientists tested two drugs already approved for other conditions: ixazomib, used for different blood cancers, and emetine. Both successfully blocked the cancer's backup system in lab studies.

"We were surprised to find that the disease's survival signals can stay active even if patients take JAK2 inhibitors for a short time or on and off," said Dr. Bian. The discovery means the cancer can resist treatment regardless of whether patients have the most common genetic mutation.

The Ripple Effect

This breakthrough changes how doctors understand myelofibrosis resistance. Because both drugs are already approved for other uses, clinical trials could start faster than if researchers had to develop entirely new medications from scratch.

The findings, published in Blood Cancer Journal and funded by the National Cancer Institute, offer hope beyond just this one cancer. Understanding how cancer cells create backup survival systems could help researchers tackle drug resistance in other diseases.

Dr. Liu says these drugs might work alone or combined with current treatments to overcome today's limitations. The team plans to test their findings in larger patient groups and begin clinical trials soon.

For patients struggling with myelofibrosis that no longer responds to standard care, this research opens a door that seemed closed.