Patients Gain Strength, Quality of Life From Experimental Drug

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Robert Lipnick Patients taking a study drug in an MD Anderson Cancer Center clinical trial put on the pounds -- lots of them. Up to 50 pounds. And no one is complaining.

In fact, it's a welcome sign that their treatment for myelofibrosis -- a debilitating and lethal bone marrow disorder that causes anemia and malnutrition -- is working.

"This drug saved my life, basically," says Robert Lipnick of Alexandria, Va. He was wheelchair-bound when he arrived at MD Anderson in 2008 to start the clinical trial for INCB018424, a pill taken daily. His weight had fallen from 155 pounds to 125, chronic pain required daily narcotics, and he suffered from night sweats and pruritis, an unpleasant, consistent itch.

Today, the retired scientist can walk for miles along the beach boardwalk using only a cane and is completely off pain medication. No night sweats, no itching. His appetite is back and he's up to 170 pounds. "How good it is to go to a restaurant and actually eat what you order."

There are no approved therapies for myelofibrosis, says Srdan Verstovsek, M.D., Ph.D., associate professor, Leukemia, and principal investigator and lead author of a study out today in the New England Journal of Medicine. Average life expectancy for people with this disease is five to seven years. End-stage patients resemble the severely malnourished, with bloated abdomens and thin limbs.

Verstovsek is careful to note that the study drug, INCB018424, is not a cure, but it is the first drug to target an underlying cause of the disease. It deals with two of the three main symptoms:

  • Anemia
  • Greatly enlarged spleens, which cause severe malnutrition and weight loss
  • Poor quality of life

The drug, developed by Incyte Corporation and co-marketed with Novartis Pharmaceuticals, shrinks the spleen and vastly improves quality of life, Verstovsek notes.

Robert Lipnick About those spleens. Myelofibrosis is caused by the build-up of malignant bone marrow cells that trigger an inflammatory response, scarring the bone marrow and limiting its ability to produce blood, leading to anemia. Renegade cells pile up in the spleen, which also attempts to make up for the anemia by producing bone marrow inside of itself and making new blood cells. 

"The growing spleen causes significant problems for the patient, and not just because it's painful. It compresses the stomach and bowels, so patients suffer malnutrition and lose weight," Verstovsek says. "The ability to walk and to bend is affected, and the body deteriorates overall."

The study drug cut spleen size by more than 50% in about half of patients. Shrinkage for most occurred within the first two months and endured, with 75% of the 153 participants still on the study.

Virginia Murrell of Spring City, Tenn., felt "immediate relief," when her spleen noticeably shrank within two weeks of starting therapy.

She has retrieved 40  lost pounds, baby-sits her grandchildren, works a few days a week in her son's garden center business and is highly active again in her church. "I can do everything I want to do," she notes.

Phase III clinical trials of INCB018424 are wrapping up. If the early findings hold, myelofibrosis will have its first approved treatment in a year or two. Next, finding a drug that boosts blood cell counts to combine with INCB018424 will provide the key to control and perhaps cure of myelofibrosis.

Safety and Efficacy of INCB018424, a JAK1 and JAK2 Inhibitor, in Myelofibrosis

From Palliation to Targeted Therapy in Myelofibrosis

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