They were just hoping to relieve the symptoms of a deadly blood disorder — and ended up treating the disease itself. In nearly half of the people who took the experimental drug, the cancer became undetectable.
Specialists said Revlimid, which is made by Celgene Corp., now looks like a breakthrough and the first effective treatment for many people with myelodysplastic syndrome, or MDS, which is even more common than leukemia.
"It may be, if not eradicating the disease, putting it into what I would call deep remission," said Dr. David Johnson, a cancer specialist at Vanderbilt-Ingram Cancer Center who is familiar with but had no role in the research.
Revlimid "is not yet on the market but almost certainly will be" because of these findings, he said.
MDS refers to a group of disorders caused by the bone marrow not making enough healthy, mature blood cells. About 15,000 to 20,000 new cases are diagnosed each year in the United States, and as many as 50,000 Americans have it now. They usually suffer anemia and fatigue and need blood transfusions about every eight weeks to stay alive.
"It's a serious problem, it tends to occur in older people, and it's fatal for most," said Dr. Herman Kattlove, a blood disorder specialist at the American Cancer Society.
Revlimid is similar to thalidomide, a drug notorious for the birth defects it caused decades ago but that in recent years has proved effective against another blood cancer, multiple myeloma. Researchers don't really know how it works other than that it boosts the immune system in a number of ways.