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Huntsman Cancer Institute research discovery leads to new clinical trial for myelofibrosis


An experimental drug called selinexor may block myelofibrosis cells researchers at Huntsman Cancer Institute (HCI) at the University of Utah (U of U) discovered.

In a news announcement, laboratory studies published in Clinical Cancer Research, a journal of the American Association of Cancer Research, examined the new drug’s effectiveness in patients with myelofibrosis, which is a rare cancer.

Myelofibrosis prevents the bone marrow from making healthy blood cells. Life expectancy varies, but most patients will not live beyond five years after the diagnosis, the news release stated.

HCI treats about 25 newly diagnosed myelofibrosis patients a year. Srinivas Tantravahi, MBBS, MRCP, a physician-scientist at HCI and assistant professor of medicine at the U of U, said in a prepared statement:

Essentially, it’s a bone marrow failure. The patient will experience severe symptoms including enlargement of the spleen, anemia, pain in the belly, fatigue, and a very poor quality of life,” says Tantravahi, who was part of the study team. “A stem cell transplant can potentially cure the disease, but most patients diagnosed with myelofibrosis are older or not healthy enough to undergo the weeks-long procedure.

According to HCI, a drug called ruxolitinib is the current primary treatment for myelofibrosis patients who are not healthy enough to receive a stem cell transplant.

This drug can decrease spleen size, improve symptoms and improve quality of life, but it does not typically reduce the percentage of malignant cells, HCI said. The lack of available therapies for myelofibrosis patients motivated the research team at HCI to look for new avenues for treatment.

Dongqing Yan, PhD, a research associate in the Deininger/O’Hare Lab at HCI and lead author on the study, said in the news announcement:

Ruxolitinib is a fine drug, but its effects are mostly short-lived. And, until now, there were no other options for patients who aren’t healthy enough for a stem cell transplant.

Based on the study findings, the researchers designed a new clinical trial to study the effectiveness of selinexor in people with myelofibrosis. The clinical trial of selinexor is now open at HCI and aims to enroll at least 24 patients who will receive the treatment in a setting that will allow for rigorous examination.

If the early trial indicates positive outcomes for patients, more extensive studies involving additional sites and more patients will be planned, HCI said.

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