A new pill not only can eliminate the need for transfusions in some people with a deadly blood disorder but also can wipe out all signs of the disease in some of them.
The experimental drug, Revlimid, is being tested in people with myelodysplastic syndrome, or MDS, a group of disorders caused by the bone marrow not making enough healthy, mature blood cells.
No one knows exactly how common MDS is, but most estimates are between 10,000 to 15,000 new cases diagnosed each year in the United States — and the number of cases is increasing.
MDS is rare in young adults. About 80 percent to 90 percent of all patients with MDS are older than 60.
Stem cell transplantation can be curative, but it's not an option for most of the patients due to their age. Most are treated with transfusions and growth factors to improve their blood counts and decrease symptoms.
But Revlimid could change all that, experts say.
"It's a successful treatment for a disease where options are now very limited," says J. Len Lichtenfeld, MD, deputy chief medical officer of the American Cancer Society. "Now we have something where there was not much before."
His comment echoed those of many cancer doctors who heard the news at the annual meeting of the American Society of Clinical Oncology (ASCO) this week.
Quick Approval Predicted
The findings are so promising, says ASCO President David Johnson, MD, that Revlimid will probably be quickly approved by the FDA.
Researcher Alan List, MD, of the H. Lee Moffitt Cancer Center in Tampa, Fla., calls the drug a "breakthrough."
He studied nearly 150 people with MDS who relied on blood transfusions to stay alive.
After just six months on the drug, two-thirds of them no longer needed the transfusions, he says. A year later, three-fourths of them were transfusion-free.
Even more exciting, he says, is that the drug eradicated all signs of a genetic defect that can cause the disease in about half of people who had the mutation.
"It looks like the drug is putting them into remission," Lichtenfeld says.
There were some side effects. Nearly four in 10 people experienced drops in blood cell counts, which resolved after the dose was lowered or people were given a drug holiday.
Revlimid is cousin to thalidomide, the infamous drug that was pulled from the market after it caused birth defects several ago but has since proven effective against cancers, including multiple myeloma.
The study was sponsored by Celgene Corp., which makes Revlimid.
SOURCES: 41st Annual Meeting of the American Society of Clinical Oncology, Orlando, Fla., May 13-17, 2005.
J. Len Lichtenfeld, MD, deputy chief medical officer, American Cancer Society, Atlanta. David Johnson, MD, president, American Society of Clinical Oncology; Vanderbilt-Ingram Cancer Center, Nashville, Tenn. Alan List, MD, H. Lee Moffitt Cancer Center, Tampa, Fla.