FDA approves new drug to treat a rare blood cancer

The U.S. Food and Drug Administration recently approved the first new medication in nearly a decade for patients with myelofibrosis, a rare blood cancer.

Ruben Mesa, M.D., FACP, director of the Mays Cancer Center, home to UT Health San Antonio MD Anderson Cancer Center, was part of the international research team that led development of the new drug.

And due to the drug’s effectiveness in treating myelofibrosis, fedratinib (brand name Inrebic), is being studied to see if it may also be beneficial for patients with other blood cancers, inflammatory diseases and possibly, one day, for diseases related to aging or that involve blood clots, such as heart attacks or stroke.

On Aug. 16, the FDA approved fedratinib for adults with intermediate-2 or high-risk primary or secondary myelofibrosis. Myelofibrosis is a blood cancer that begins in the bone marrow, where red blood cells, white blood cells and platelets are made. It is one of a related group of blood cancers and chronic leukemias called myeloproliferative neoplasms, or MPNs, that can lead to acute leukemia. Patients with myelofibrosis may experience severe anemia, night sweats, weight loss and significant and painful enlargement of the spleen, an organ involved with filtering the blood.

Ruben A. Mesa, M.D., FACP, is director of the Mays Cancer Center, home to UT Health San Antonio MD Anderson Cancer Center.

“Think about a healthy spleen as being about the size of your fist. With myelofibrosis, the spleen can swell to the size of a football or even a full-term pregnancy,” explained Dr. Mesa. He was an investigator on the fedratinib studies while he was a researcher, chair of the Division of Hematology and Medical Oncology, and deputy director of the Mayo Clinic Cancer Center in Scottsdale, Ariz. Dr. Mesa continued his research and advocacy for the approval of fedratinib after being recruited to UT Health San Antonio’s cancer center in August 2017

He has assembled a team of myelofibrosis experts at the Mays Cancer Center who provide care to patients from around the world. And now that the new drug has been approved, the team can now prescribe fedratinib to eligible patients.

“The pressure of the enlarged spleen can be very painful, pressing on nearby organs. Patients also may experience fatigue, night sweats or shortness of breath associated with anemia. Bone pain and weight loss are also common,” Dr. Mesa said. There is no cure for most myelofibrosis patients.

According to the MPN Research Foundation, about 18,000 individuals in the United States have myelofibrosis and approximately 300,000 live with one of the three MPN blood marrow disorders, which also include essential thrombocythemia and polycythemia vera.

Until fedratinib was approved, ruxolitinib was the only drug available to treat patients with myelofibrosis. “Now with fedratinib, physicians and patients have another treatment option, especially for patients who do not respond well to ruxolitinib,” said Dr. Mesa, who also was involved in the ruxolitinib research.

Dr. Mesa also led development of the National Comprehensive Cancer Network’s Guidelines for Myeloproliferative Neoplasms, published in 2017. In 2018 he was appointed to the national board of directors of the Leukemia & Lymphoma Society.

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