Spina Bifida in the news.

Spina Bifida in the news...

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Researchers Produce Nerve Cells

Scientists have been able to produce nerve cells in the lab by using stem cells drawn from bone marrow, a breakthrough that could help people with Alzheimer's disease, Parkinson's disease or spinal-cord injuries.

If the findings are borne out, they might one day enable doctors to take cells from a patient's bone marrow, turn them into nerve cells and then inject them into patients' brains and spinal cords, replacing injured cells.

The research, conducted at the University of Medicine and Dentistry of New Jersey and MCP Hahnemann University in Philadelphia, was funded in part by the Christopher Reeve Paralysis Foundation. An account of the research was published Monday in the Journal of Neuroscience Research, based in Los Angeles.

It could be years before the technique is tested in humans, however, since studies and tests in animals will be needed first. Still, other researchers were excited by the possibilities.

``Clearly it is important to generate neurons from other tissues,'' neuroscientist Ronald McKay told The Philadelphia Inquirer in Tuesday's editions.

``We have to demonstrate that these cells will perform ... specific functions'' in the brain and spinal cord, and not behave like stem cells only in the lab, said McKay, of the National Institutes of Health.

``It's really beautiful,'' Dr. William Greenough, a neuroscientist at the University of Illinois in Urbana-Champaign, told The New York Times. ``I see this as a truly significant step along the way toward developing new technologies for the repair of the damaged brain.''

Stem cells are stored in various organs and grow naturally into the specific types of cells needed by those organs. Stem cells from fetal tissue can grow into any type of cell; the latest study suggests that the same might be true for adult stem cells.

Dr. Ira Black of the University of Medicine and Dentistry of New Jersey reported the stem cells, treated with growth factors and antioxidants in tissue culture experiments, quickly divided into two cells - another stem cell and a nerve cell.

By tinkering with the chemical signals he used to stimulate the conversion, Black and his colleagues were able to turn 80 percent of the bone marrow cells taken from rats and humans into nerve cells. He said he transplanted rat nerve cells to the brains and spinal cords of rats and found that they formed connections with other neurons and survived.

Stem cells could be drawn from the patient being treated, eliminating the problem of adding tissue the immune system might attack, said Darwin Prockop, of Tulane University's medical school, who co-authored the research.

Susan Howley, director of research at the Christopher Reeve Paralysis Foundation, cautioned that many questions still surround the new technique.

Spina Bifida Procedure Publicized

NASHVILLE, Tenn. (AP) - Drs. Joseph Bruner and Noel Tulipan have received accolades for their pioneering method of treating spina bifida in the womb but have gotten chilly reviews in some medical circles for their method of publicizing it.

Rather than waiting months or years for their procedure to be reviewed by peers and published in a medical journal, the Vanderbilt University Medical Center doctors took their story straight to the media. Soon, stories about them, their procedure and their patients started showing up in newspapers and magazines, and on television shows around the country.

Then came e-mails and phone calls from doctors criticizing them as media hounds more interested in self-promotion than sound science.

``We felt it was important for the public to know we were performing a new and innovative procedure on unborn babies,'' Bruner said. ``The public has a right to know about that, and we wanted to work with news organizations to let the public know as much as we could.''

Though the procedure is yielding good initial results, some physicians are uncomfortable with the doctors going public before their work was reviewed by peers.

Among them is Dr. N. Scott Adzick, director of the Center for Fetal Diagnosis at Children's Hospital in Philadelphia and the only other doctor using a similar technique to treat spina bifida. ``Folks can make claims about everything, and I don't think the lay process is the place to ferret that out,'' he said.

``My approach would not be to have reporters follow families through the process or have TV crews following families through the process because these are very vulnerable families now.''

A bitter rivalry developed between Adzick and the Vanderbilt doctors last November when he published the results of his work in The Lancet, a British medical journal, and called his procedure the first of its kind.

The Vanderbilt doctors sent an angry letter to the journal saying they were the first, and Adzick responded by criticizing the doctors for using the mass media to publicize their work.

The acrimony has ended, both sides say, and the doctors now refer patients to each other.

Spina bifida is the nation's most common permanently disabling birth defect, affecting about one of every 1,000 births. It occurs when the skin fails to properly close over the spinal cord early in the pregnancy and may result in excess fluid on the brain, bowel and bladder problems, clubbed feet, partial paralysis and learning difficulties.

Traditionally, doctors have tried to limit its effects by closing the spinal cord in the first 24 hours after birth. In the procedure done by Bruner and Tulipan, doctors operate while the baby is in the womb, between 20 weeks and 28 weeks after conception.

The surgeons place the uterus on the mother's belly, pull the fetus out through an incision, locate the spinal cord and close tissue over the exposed area. The fetus is then returned to the womb and allowed to develop until the mother gives birth.

The doctors have performed about 60 operations. In about half, their patients have avoided the shunts almost all spina bifida patients need to drain fluid from the brain for the rest of their lives. Though it is too early to tell, Bruner and Tulipan are hopeful the surgery will improve brain function, too.

When Bruner and Tulipan began doing the procedure in April 1997, they planned to wait and see the results of 10 cases before they went public with their findings.

But their initial patients were so pleased they contacted media in their hometowns to try to raise money to help other parents get the procedure, which at the time was not covered by insurance. The doctors cooperated with the reporters.

``I don't think that we really had a concept at the time how this would really capture the public imagination and how tremendous of an advance the public would perceive this as,'' Bruner said.

While the public wanted to learn about the procedure, medical journals were not eager to provide a forum. The New England Journal of Medicine rejected a paper on their technique because the results already had appeared on the hospital Web site.

Medical journals want doctors to follow tradition, presenting their work to their colleagues for review before any word of it is published.

When doctors go to the public without having their work verified and scrutinized by other doctors, ``the risk of harm (to the public) may actually outweigh the benefits,'' said Dr. Richard Glass, an interim co-editor of the Journal of the American Medical Association.

Since their paper was rejected, Bruner said, he and Tulipan have become more reluctant about sharing their results to prevent themselves from being barred by other publications. That puts them in a bind. As they perform more surgeries, they have more results to share.

``We now know, for example, that there are some babies that have better outcomes than others. There are some babies that will benefit tremendously from this and there are others that won't benefit so much,'' Bruner said.

``We think this is important information to get out to the public. Unfortunately, the turnaround time for journals is somewhere between one to two years. By the time the information that we've already submitted to journals gets published, it's going to be out of date. And by the time we send them new information, it'll be another year or two years to get it published.

``It's very frustrating to have information that we know is important for the public and that we know patients need in order to make reasonable decisions.''

The need for speed is not lost on medical journals. The Journal of the American Medical Society announced in May a new ``JAMA Express'' process to rush information deemed critical into publication within six weeks, up from an average of five months.

Extremely critical articles can be published much faster on the journal's Web site. In June 1998, JAMA posted a report about a potentially fatal pairing of two drugs used to treat high blood pressure. The report hit the Web three days after it was accepted.

``Those things have to be considered very carefully,'' Glass said. ``Everybody would like things to go fast, and it's just not possible for everything to go that fast.''

Adzick said while things move faster now, it's important for the peer review process to continue to ensure credibility. Tulipan agreed.

``I don't think the lay press or the public is qualified to decide whether a procedure is effective or not,'' Tulipan said. Peer review is ``still the gold standard. It's still the benchmark.''

Still, Bruner has few regrets about the methods he and Tulipan used.

``Looking back over the past few years, I can't see it happening any differently,'' he said. ``The only way it could have been different is if we refused to talk to the media altogether, and that would have been unfair.

``Back in the '50s and '60s, a lot of human experiments were done that were kept secret from the public and when the public found out, they were always upset. So we weren't going to hide what we were doing.''

New Hope Against Spina Bifida

NASHVILLE, Tenn. (AP) - Seventeen-month-old David Gregory is beginning to form words and already is exploring the world with his walker with wheels. One day, his parents hope, he will walk on his own.

Spina bifida will keep David from living an ordinary life, but his parents believe a pioneering surgical procedure performed on him while he still was in the womb will dramatically improve his quality of life. ``I feel like David got the best outcome that he could have hoped for mobility-wise, and I feel that's probably attributable to the surgery,'' his mother, Becky, said from her home in Sparr, Fla.

Spina bifida occurs when the spinal cord is exposed early in the pregnancy and may result in excess fluid on the brain, bowel and bladder problems, clubbed feet, partial paralysis and learning difficulties. The defect affects about one of every 1,000 newborns each year. Doctors do not know what causes the condition, but they have found that enough folic acid in the mother's diet early in the pregnancy - often before she even knows she is pregnant - can prevent it.

About 2,000 U.S. infants are born with the condition each year. Roughly the same number of fetuses are estimated to be aborted after the condition is diagnosed.

To treat spina bifida, Vanderbilt University Medical Center Drs. Joseph Bruner and Noel Tulipan operate while the baby is in the womb.

They remove the mother's uterus and place it on her belly. After pulling the fetus through an incision, they locate the spinal cord and close tissue over the exposed area. Then they return the fetus to the womb and allow it to develop until the mother gives birth.

It is too early to know how effective Bruner and Tulipan's procedure is, but parents are enthusiastic, and the doctors are optimistic. Only half of the babies they operate on need a shunt to drain excess brain fluid, as compared to 95 percent in other babies with spina bifida.

For some parents, eliminating the unwieldy shunt and possibly countless replacement surgeries is reason enough for the surgery. The doctors hope the procedure will also improve brain function. Brain scans suggest they may.

``Our oldest patient is 2 1/2 years old,'' Tulipan said. ``So he's got another 2 1/2 years to live before he even gets to kindergarten. We don't know whether they're going to have learning disabilities or not. But there is the potential possibility that they will do better overall in terms of brain function.''

The technique is only performed at Vanderbilt and at Children's Hospital in Philadelphia, but that may change. Bruner said Vanderbilt plans to start a training program for doctors from other hospitals. The first new hospital is scheduled for this year, Bruner said. He declined to identify it.

Bruner, 46, graduated and received his medical degree from the University of Nebraska. He has been at Vanderbilt since 1990. Tulipan, 48, received his bachelor's and medical degrees from The Johns Hopkins University in Baltimore and joined the Vanderbilt faculty in 1986.

They met in a hallway, Bruner said, and began talking about some promising spina bifida research on lab rats. They worked out a procedure to operate in utero on sheep, then adapted it to humans.

The first attempts were dismal failures. Using an endoscope to peer inside the womb, they maneuvered their instruments like chopsticks, leaving the womb in place while they operated on the fetus. Two fetuses died. One was delivered prematurely.

They abandoned that technique for their current one. The success of the method has surprised them.

Only one baby was stillborn, which Bruner thinks could have been prevented by better care at her home hospital. Another baby had to be delivered during the procedure, but has gone on to do well.

All babies that undergo fetal surgery are delivered prematurely. Of their 47 patients born so far, all but five were born after at least 30 weeks, and none has had serious complications.

``It's almost too good to be true,'' Bruner said. ``But we attribute this to the fact that what we have begun doing is basically elective surgery for the treatment of the fetus. ... Not only are our patients fairly healthy, but we can pick the time, we can pick the place and we can pick the patients, and as a result, our outcomes are very good.''

In the case of David Gregory, he has not needed a shunt to drain brain fluid. He is beginning to talk but has trouble eating. He can walk with help, but lacks bladder and bowel control. He may never have sexual function, and, his mother said, it is too early to tell if he will have trouble learning, but she is hopeful.

``Even though these babies are high maintenance, they're really a joy and they really add a dimension to your life that you wouldn't have experienced otherwise,'' she said.

``Even with the spina bifida, that partly makes (David) who he is - the fact that he is going to have to overcome pretty significant obstacles. He's not going to be less because he has spina bifida. I can already see, he's going to be more.''

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