For Mike Harris, life is good.
"I'm alive today because of a clinical trial," he says of a Phase I study he entered at MD Anderson four years ago for his myelofibrosis, a rare blood cancer that had no standard treatment at the time.
Last year, he and wife Sandy celebrated their 45th wedding anniversary. They live in Kingwood, north of Houston, near their son's family, and enjoy strong connections to their two grandchildren.
First sign of hope
The drug Harris received on that earliest-stage study -- and has taken ever since -- went on to become in November 2011 the first drug ever approved by the U.S. Food and Drug Administration to treat myelofibrosis.
Srdan Verstovsek, M.D., Ph.D., associate professor in MD Anderson's Department of Leukemia, led that Phase I study and was principal investigator on every U.S. clinical trial for ruxolitinib, developed by Incyte Corporation. It is known commercially as Jakafi™.
Myelofibrosis strikes about 3,000 Americans annually. A build-up of malignant cells in the bone marrow triggers inflammation that scars the bone marrow, eventually causing severe anemia, swelling of the spleen and liver, extreme weight loss and, ultimately, death. The debilitating symptoms contribute to a poor quality of life.
"We previously had no therapies to attack the molecular abnormalities that cause myelofibrosis. The emergence of ruxolitinib has been a major improvement for our patients. They feel better and live longer," Verstovsek says.
"The clinical trials showed ruxolitinib provided very good control of the symptoms of myelofibrosis, including spleen shrinkage, liver reduction, and improvements in the patients' ability to walk, eat and gain weight. A Phase III trial showed a survival benefit, too."
With no approved therapies, oncologists tried chemotherapies approved for other malignancies combined with supportive care, mainly blood transfusions. About 10% of patients are eligible for bone marrow transplants.
When these off-label drugs work at all, the effect usually lasts only 6 to 12 months, Verstovsek says.
Heavy symptom burden
In Harris' case, a variety of drugs kept the disease at bay for two years before his oncologist referred him to Verstovsek. "My doctor had heard Dr. Verstovsek speak about this new drug at a meeting."
Myelofibrosis comes with a formidable set of symptoms, including:
- Abdominal pain
- Steep weight loss
- Night sweats
- A powerful and persistent itching sensation
- Bone pain
- Expansion of the spleen to the extent that it suppresses appetite, affects breathing and makes it difficult to bend or walk
End-stage patients resemble those dying of starvation, with huge abdomens and emaciated limbs.
Harris had the swollen spleen and vividly recalls being too tired to leave the house. "I couldn't walk up a single flight of stairs without resting."
Some patients, Verstovsek notes, have lost 30 to 50 pounds as their disease progressed. Many regain it all and more when taking the drug. "The main long-term side effect of this drug appears to be weight gain," he says.
He estimates 80% of myelofibrosis patients receive some clinical benefit from ruxolitinib.