ROANOKE TIMES
Copyright (c) 1996, Roanoke Times
DATE: Thursday, August 8, 1996 TAG: 9608080051
SECTION: NATIONAL/INTERNATIONAL PAGE: A-1 EDITION: METRO
DATELINE: BOSTON
SOURCE: Associated Press
NOTE: Below
Bone marrow transplants have been found to cure sickle cell anemia, a disease that afflicts 80,000 Americans, most of them black. But the risky treatment will probably be reserved for only the worst cases.
A study published today found that while three-quarters of victims are cured, 10 percent die from the treatment itself.
The approach is radical. Doctors use chemotherapy drugs to destroy the victim's own blood-producing bone marrow, then substitute marrow taken from a genetically similar brother or sister. Evidence has been building for more than a decade that this can reverse the disease, and the new study offers proof.
However, two major shortcomings remain: Only an estimated 18 percent of sickle cell patients have a suitable marrow donor. And even when a donor is available, doctors will be reluctant to risk the procedure unless they are certain patients will have severe forms of the disease.
Nevertheless, experts say finding a way to cure - not just treat - even a small percentage of sickle cell cases is an important milestone.
The word ``cure'' is being used ``with a great degree of consideration and advisement,'' said Dr. Keith M. Sullivan, an author of the study. ``But these patients have been observed for a long period, and they have no evidence of sickle cell disease.''
Sickle cell disease results from an inherited genetic flaw that causes oxygen-carrying red blood cells to take on a sickle shape. They get stuck and clog tiny blood vessels, cutting off the blood supply. Organ damage and bouts of severe pain may result.
About one in every 400 American blacks is born with sickle cell anemia. On average, the disease shortens their lives by about 30 years.
In their study, published in the New England Journal of Medicine, doctors from more than 30 hospitals in the United States and abroad gave bone marrow transplants to 22 children with severe symptoms of sickle cell anemia. Sixteen were cured, two died, and the treatment failed in four.
However, the transplanted tissue can attack the patient in what is known as graft vs. host disease, a common transplant complication that can kill. Even if the patient survives the transplant, he or she faces an increased risk of sterility and cancer later in life as a result of the treatment.
Marrow transplants already are routine for treating some forms of cancer. In a U.S. case in 1984, doctors gave a transplant to a young leukemia victim who also happened to have sickle cell anemia. The treatment cured both diseases.
The transplants cost $150,000 to $180,000. But the medical bills for three to five years of treatment without transplants could add up to this much.
Because the severity of the disease varies substantially, doctors usually cannot tell how bad it will be until serious complications have already occurred.
Sullivan, a doctor at the Fred Hutchinson Cancer Research Center in Seattle, said that only about 6 percent of sickle cell victims are as clearly ill as those enrolled in the study.
The patients ranged in age from 3 to 13. Half had already suffered strokes because of their disease. Others had lung problems or frequent pain.
``We think this is an important first step in looking at potential cures, but without the ability to identify patients who are at significant risk for complications, a lot more work needs to be done,'' said Dr. Helena Mishoe of the National Heart, Lung and Blood Institute.
Sullivan, a doctor at the Fred Hutchinson Cancer Research Center in Seattle, said doctors are working on brain scans intended to spot small areas of brain damage that are an ominous signs of strokes to come. Perhaps 20 percent of young sickle cell victims have this early damage, and they might be considered for marrow transplants if the scans prove useful.
Dr. Ronald Nagel of Albert Einstein College of Medicine in New York City worried ``the bottom line is this is a significant advance, but patients should not think this is a cure for all,'' he said.
LENGTH: Medium: 77 linesby CNB