Roanoke Times
Copyright (c) 1995, Landmark Communications, Inc.
DATE: SUNDAY, June 3, 1990 TAG: 9006030097
SECTION: NATIONAL/INTERNATIONAL PAGE: A1 EDITION: METRO
SOURCE: The New York Times
DATELINE: LENGTH: Long
The therapy involves transplanting healthy muscle cells from another person into the muscle of a patient with muscular dystrophy.
The healthy cells then begin making the protein that is lacking in patients with the disease.
The work does not mean a treatment is imminent for muscular dystrophy, a genetic disease that causes progressive and irreversible muscle deterioration that almost always leads to death by the age of 30.
Among other things, scientists must determine whether the transplants will work on large muscle groups and whether patients will reject the transplanted tissue.
Nevertheless, experts who cited the new results and previous work in animals said the finding held out a real ray of hope, not only for muscular dystrophy but also for other degenerative muscle diseases.
The results were announced Saturday by Dr. Peter Law, a professor of neurology at the University of Tennessee in Memphis, at a meeting of the Muscular Dystrophy Association in Tucson, Ariz.
Law's finding involved a single patient, Sam Looper of Pickens, S.C., a 9-year-old boy with Duchenne muscular dystrophy, the most common form of the disease.
To avoid possible damage to important muscles, the researchers tested the treatment in one of his big toes.
At least seven other patients are undergoing the experimental treatment, Law said in an interview Saturday, but those results are not yet available.
Dr. Leon Charash, chairman of the national medical advisory board of the Muscular Dystrophy Association, said he believed another group of researchers, at the Montreal Neurological Institute, had replicated the feat.
Charash said he expected the group, which like Law is being financed by the association, to announce the findings Monday.
Two other groups also are pursuing the treatment and are supported in part by the Muscular Dystrophy Association, which encouraged the concurrent studies to have a "horse race," Charash said.
Charash added that the finding is promising enough that the researchers now will be moving quickly to try the muscle cell injections in larger muscles of the leg that help control balance.
In addition, Law said, the method could be applied to other forms of muscular dystrophy or to any disease of muscular degeneration, whether or not the exact genetic defect causing the degeneration is known.
"I think it is of great importance," Charash said.
"This represents the first time a treatment has been offered. It is a moment of great scientific significance."
If the finding is confirmed, said Dr. W. French Anderson, a branch chief at the National Institutes of Health who has devoted his entire 22-year scientific career to designing human gene therapies, "this would be a landmark."
But investigators also sounded a note of caution.
Dr. Francis Collins, a gene-therapy researcher at the University of Michigan in Ann Arbor, said, "It is encouraging to see that there are some real possibilities here, but there are a lot of hurdles ahead," before there can be an effective treatment.
Collins said the research "is a promising lead, but we ought to have our eyes open. Patients with muscular dystrophy are in a desperate situation, and I think we should be very careful not to give them false hope."
Collins said it is not known how effective the treatment will be in larger muscles or whether the body will reject transplants of massive numbers of muscle cells.
It is also not known how long the treatment will last and, although it seems to correct muscle weakness and greatly prolong the lives of mice with muscular dystrophy, mice are not humans.
Law and others have long thought that muscle cell injections might offer real hope for patients with muscular dystrophy and other muscle-wasting diseases because of the special way that muscle cells form.
Unlike other cells, which exist as separate entities in organs, touching each other like lilies in a pond, muscle cells fuse together to form one giant cell with many nuclei.
In his experiments with eight patients, all boys, he took the healthy cells from the boys' fathers and injected them into a muscle of the foot that controls the movement of each boy's large toe.
The muscle of the other foot was given an injection with a dummy substance and neither the parents nor the researchers knew which foot got which treatment.
The boys took the anti-rejection drug cyclosporine to prevent their immune systems from attacking the transplanted cells.
Three months later, the muscle cells were biopsied to see if the transplant was effective.
Law also tested muscle strength and in the case of Sam Looper, which was reported Friday, strength had increased 20 percent, he said.
Lynn Looper, Sam's mother, said that while she was awaiting the results, she kept asking her son to move his toes, looking for signs that the toe on one foot was stronger.
"After maybe a month, Sam said, `This one's stronger,' " she said Saturday.
"And he was right."
When Law first saw the results two weeks ago, he had another family in his office, Marty and John Donnelly of Cookeville, Tenn.
Marty Donnelly recalled that Law very excitedly showed them the evidence that the treatment was working and swore them to secrecy.
"He was crying as he showed us the pictures," Donnelly said.
by CNB