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FDA Approves New Drug For Rare Disease, San Antonio Researcher Helped Make It Happen

Myelofibrosis, Reticulin Stain Pathological and histological images courtesy of Ed Uthman at flickr.

The U.S. Food and Drug Administration has approved a new drug to treat a rare blood disorder. It’s the first new treatment for this disease in nearly a decade, and a San Antonio researcher is one of the people behind it.

The medicine is called fedratinib (generic version of INREBIC) and will be only the second medicine available to treat a leukemia called myelofibrosis.

Dr. Ruben Mesa, director of the Mays Cancer Center at UT Health San Antonio MD Anderson Cancer Center, said only about 20,000 people in the United States have myelofibrosis, but it’s part of a larger family of leukemias that impact around a half million people.

Mesa was one of the researchers who shepherded this medication through the FDA's approval process, and he says it works on a specific gene mutation — JAK2 — that causes bone marrow to make too many blood cells. It also causes inflammation. Fedratinib inhibits the enzyme activity caused by that mutation.

That means the medicine may, in the future, be used to treat many other conditions that are linked to JAK2 mutations, Mesa said.

"It may end up impacting people who have acute leukemia, and there are other chronic leukemias that overlap,” Mesa said. “The JAK2 mutation has implications in terms of aging. It has implications in terms of the development of blood clots or bleeding."

Mesa said getting FDA approval for fedratinib is good news for patients but stressed it's not a cure. He and his fellow researchers are still working on that.

“So next we will be doing combination studies to see how well it works in combination with other medications, or other strategies, we might potentially get closer to curing individuals that have this disease,” Mesa said. “Then looking at the potential role of this drug in other diseases, whether they be bone marrow diseases or others that we think are impacted by the JAK2 mutation.”

Bonnie Petrie can be reached at Bonnie@TPR.org and on Twitter at @kbonniepetrie.