Thalidomide Finds Redemption as a Cancer Foe
Nov. 17, 1999 (Seattle) -- Thalidomide, a drug best known for causing
horrific birth defects, is prolonging the lives of people with a rare form of
cancer, according to a study in TheNew England Journal of
The study, conducted by a team at the University of Arkansas for Medical
Sciences in Little Rock, found that thalidomide helped a third of people with
advanced multiple myeloma, an incurable cancer of the bone marrow. Most of the
people in the Arkansas study had exhausted all other treatment options before
they got the drug.
One of the researchers, Bart Barlogie, MD, PhD, tells WebMD that the results
represent "a milestone in therapy for myeloma." Barlogie, an
internationally known expert on the disease, says the prospect of a new weapon
to fight it is exciting because doctors have been using the same two drugs for
about 40 years.
Multiple myeloma strikes nearly 14,000 people a year in the U.S. More than
70% of them die within five years. Thalidomide is a drug that was widely used
in Europe to prevent morning sickness in pregnant women. It was pulled off the
market after reports that it led to severe birth defects, including children
with flipper-like arms and legs. In recent years, though, thalidomide has made
a comeback as a treatment for leprosy.
In the new study, researchers gave increasing doses of thalidomide to 84
people with multiple myeloma who were failing other treatments. Most were
getting sicker despite having received the highest possible doses of
chemotherapy -- usually the final treatment option.
But despite the advanced stage of their disease, about one-third of the
patients got better with thalidomide. Tests that indicate the presence of
cancerous cells in the bone marrow showed dramatic reductions for about 10% of
the patients, including two who had a complete remission. Barlogie says it's
not clear yet how long the benefits last.
In an editorial about the study, two cancer researchers from Harvard Medical
School called the results "remarkable." One of the researchers, Kenneth
Anderson, MD, tells WebMD that thalidomide is generating huge excitement in the
medical community because multiple myeloma has proven so difficult to
Anderson says the results of this study are particularly striking because
the study included people who were no longer responding to any other treatment.
"It's likely to be even more effective with people in an earlier stage of
the disease," he says.
But scientists say that they still need to determine the ideal dose of
thalidomide, which in large amounts can cause side effects including
constipation, sleepiness, and fatigue. They also hope to learn how the drug
works against myeloma.
"We are in the midst of examining several different mechanisms,"
says Barlogie. One involves the drug's ability to prevent the formation of new
blood vessels, he says, which help cancers grow. Barlogie says it's also
possible that thalidomide helps the body's immune system attack myeloma
- Multiple myeloma is a rare, incurable cancer of the bone marrow, striking
14,000 Americans each year.
- In a study of people in advanced stages of the disease who were not
responding to traditional therapy, the drug thalidomide improved the condition
of one-third of the patients.
- Researchers suspect the drug will be even more effective for patients in
early stages of the disease, but further research is necessary.