A new study published in the journal Cancer Research suggests that a drug being used to treat some advanced cancers may offer a new treatment option for myelofibrosis.

Myelofibrosis is a rare but serious form of leukaemia. It affects approximately 1 to 1.5 people in every 100,000, most of whom are middle-aged or older. Patients with intermediate or high-risk cases typically have a survival rate of only 16–35 months.

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Existing myelofibrosis treatments do not address the disease’s hallmark bone marrow scarring. While ruxolitinib is currently used to alleviate patients’ symptoms, Mohi’s new research suggests that pairing it with palbociclib may result in a far superior treatment.

Palbociclib may be able to prevent bone marrow scarring in ways that current treatments for myelofibrosis cannot. This scarring impairs the marrow’s ability to produce blood cells, resulting in severe anaemia that leaves patients weak and exhausted.

Additionally, scarring decreases the number of platelets in the blood, which makes clotting more difficult, and frequently results in an enlarged spleen.

Palbociclib reduced bone marrow scarring in two myelofibrosis mouse models. It also reduced the number of white blood cells in myelofibrosis mice and shrank their enlarged spleens.

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Adding ruxolitinib to the mix improved the mice’s bone marrow and white blood cell counts while shrinking their enlarged spleens.

Additional research is required to ascertain whether the findings are applicable to human patients. However, Mohi and his team remain hopeful. They note that palbociclib has been shown to suppress bone marrow activity in patients with metastatic breast cancer (cancer that has spread to other parts of the body), and they hope that this will also be true for patients with myelofibrosis.

“A combinatorial therapeutic approach involving palbociclib and ruxolitinib will enable lowering the doses of each of the inhibitors and thus reducing toxicities while enhancing the therapeutic efficacy,” they write in a new scientific paper outlining their findings.

New treatments for myelofibrosis are particularly needed, the researchers note, because ruxolitinib does not significantly reduce bone marrow fibrosis and frequently loses its efficacy with prolonged use.

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“The findings from this study are very exciting, and they support the clinical investigation of palbociclib and ruxolitinib combination in patients with myelofibrosis,” Mohi said.

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