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Monday, May 16, 2005

Drug tested on blood disorder might fix genes
Patients in study required fewer transfusions, and underlying cause of
disease seemed to disappear.

By MARILYNN MARCHIONE
The Associated Press

ORLANDO, FLA. - Doctors were hoping to treat just the symptoms of a deadly
blood disorder when they gave people a drug to lower the need for
transfusions.

To their astonishment, signs of the disease itself disappeared in nearly
half of them.

Specialists said the experimental drug, Revlimid, looks like the first
effective treatment for many people with myelodysplastic syndrome, or MDS,
which is 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 specialist at
Vanderbilt-Ingram Cancer Center who is familiar with but had no role in the
research.

Researchers don't know how Revlimid works other than that it boosts the
immune system.

In small studies, it showed promise with far fewer side effects. In a new
study, doctors tested it on 115 people with MDS who have the most common
chromosome abnormality that causes the disease.

After about six months on the drug, 66 percent no longer needed blood
transfusions, said the study's leader, Dr. Alan List of the H. Lee Moffitt
Cancer Center in Tampa, Fla. A year later, three-fourths of them still don't
need transfusions.

But the big surprise was that signs of the genetic mutation fueling the
disease diminished in 81 patients and vanished in 51.

"The chromosome abnormality completely disappeared - something we've never
seen before" from a drug aimed just at boosting red blood cells, List said.

About one-third of people on Revlimid had temporary drops in other blood
cells and clotting components, fixed by briefly interrupting treatment or
lowering the dose.

The study was sponsored by Revlimid manufacturer Celgene Corp. List is a
consultant for the company and reported results Sunday at a meeting of the
American Society of Clinical Oncology in Orlando.


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