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Cerebral Palsy - Back to News Menu
A cure for Adam?
Parents believe umbilical cord blood injections help their son with cerebral palsy, but doctors warn of ethics violations, faulty science
By Terri Somers
UNION-TRIBUNE STAFF WRITER
June 13, 2005 - Brandon Susser was born perfect. His twin brother, Adam, was born dark blue.
The umbilical cord had wrapped around Adam's neck, suffocating him for who knows how long. The oxygen deprivation caused severe brain damage, leaving him unable to see or control his motor functions.
Gary Susser, father of twins Adam (center) and Brandon, stayed in San Diego last week and visited a doctor in Tijuana, where Adam received an injection of stem cells to treat his cerebral palsy.
Adam, now 4, has been diagnosed with cerebral palsy, a condition of impaired neurological function. Most of the medical establishment says there is no cure. If there is good news about cerebral palsy, it is that it doesn't get any worse.
That prognosis is unacceptable to Adam's parents Gary, a lawyer, and Judy, a paralegal, who live in Boca Raton, Fla. So they decided to take a gamble on what's been hyped as one of the most promising areas of medical research in decades: stem cell therapy.
On June 6, the Sussers made their third trip to Tijuana, where a doctor injected a syringe full of what he said was 1.8 million stem cells from a stranger's umbilical cord blood into Adam's stomach. According to the Mexican doctor and the U.S. doctor who referred the Sussers to him, the injections help Adam's brain replace damaged cells or repair broken circuitry. The parents are convinced the treatment is already working miracles. But some respected physicians and scientists said the family is buying pure snake oil.
They said the Sussers have been misled by hucksters who are capitalizing on the family's emotions and on the hoopla that has built around the potential for stem cell treatments to someday be used for such things as repairing damaged spinal cords or neurons in the brains of people with Alzheimer's disease.
"I just feel terribly sorry for the family," said Dr. Lawrence Schneiderman, a professor of family and preventive medicine at the University of California San Diego. "I think the doctors and all the health care providers who are promoting this are violating if not the law, certainly professional ethics."
Brandon Susser has learned to talk, walk, run, use the bathroom and name all his colors.
Adam has not mastered any of those skills. But tell him something funny, and he'll respond with a contagious belly laugh. And when he concentrates really hard, he can call out the name of his nanny.
Since receiving the stem cell therapy, the Sussers said Adam has been able to do something a Miami doctor deemed impossible – see.
SEAN M. HAFFEY / Union-Tribune
Gary Susser's son, Adam, who has been diagnosed with cerebral palsy, is able to respond to something funny with a belly laugh.
Three months and one week after Adam received his first shot, the Sussers said, they were sitting at home with the children when Brandon rolled a ball across the floor and Adam tracked it with his eyes.
"He'd never done that before," said Gary Susser, his voice getting loud with enthusiasm.
His parents took him to a second eye specialist, this one outside Fort Lauderdale. She said Adam could see, though his vision was very poor, Susser said.
"He needs glasses. . . . But now he can see me come into the room. He can recognize me without my saying a word.
"Isn't that worth the money?"
Each treatment, which the Sussers said is about as quick and easy to administer as a flu shot, costs $6,000. They don't know how many they will need. To pay, they tap a $700,000 medical malpractice settlement they secured from the doctors who delivered Adam.
"We consider that blood money," Gary Susser said. "We're not about to buy a sports car with it. We are using it to try to improve Adam's life."
It has paid for full-time nannies, a state-of-the-art wheelchair and physical, occupational and speech therapies. It also provided seed money for the Adam Susser Foundation, which has given $500 scholarships to physical therapy students and bought equipment for other disabled children.
But stem cell researchers said the family is wasting the money it is spending on Adam's injections.
Because cerebral palsy doesn't get worse, it can appear the child is getting better as he or she grows up, developing motor and language skills, said Dr. Evan Snyder, a pediatrician who runs the stem cell research program at the Burnham Institute in La Jolla. The physical therapy the boy is receiving could also be having a beneficial effect, he said.
Gary Susser disagreed.
"That doesn't explain how he can suddenly see," Susser said. "He hasn't had therapy on his eyes."
The father believes it could also be a combination of things that prompted the change.
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"It could be the (physical) therapy. It could be prayer. It could be the stem cell therapy. It could be the special diet," Susser said. "So I'm not about to change any of it."
Snyder, though, said he is certain it is not the umbilical cord stem cell treatment.
Both Snyder and Lawrence Goldstein, a stem cell researcher with the Howard Hughes Institute at UCSD, said there is no reliable data that shows stem cells in umbilical cord blood become the nerve cells needed to replace those damaged in Adam's brain. Cord blood stem cells become blood cells, Snyder said.
Further, Snyder said it is impossible to imagine that stem cells in umbilical cord blood would randomly find their way from the boy's abdomen into his bloodstream and then make their way across the hard-to-penetrate barrier that would allow them to get into his brain.
The cord blood stem cells are different from the embryonic stem cells that have generated scientific excitement along with controversy. Umbilical cord stem cells are "adult" stem cells, meaning they have already been biologically programmed as to what type of cell they can become, while embryonic stem cells can develop into any cell in the body.
Embryonic stem cells have been the focus of an intense debate since President Bush signed an executive order in 2001 that severely limited federal funding for the science. He objected to the work because it requires the destruction of embryos.
The science has continued to generate headlines as California passed a $3 billion initiative to fund the research and as debate continued in Congress over whether to restore some of the funding.
Michael Kalichman, a UCSD ethicist, said the publicity has given some people unrealistically high hopes.
"I can't say these doctors are intentionally capitalizing on it, but I can say that their timing is very good," he said. "There's so much about this in the news that people attach hope to the words stem cell and think these are magical cures for many things."
Last week, Kalichman participated in a stem cell ethics conference attended by top researchers. It seemed that everyone there agreed that stem cell therapies were years away, Kalichman said.
"And the idea of injecting them into a boy to repair disease is something no one is considering," he said.
But that is exactly the idea that Dr. David Steenblock, a doctor of osteopathy, is promoting on a Web site that Gary Susser devoured. Susser, a consumer advocate trial attorney, said he heard about the Steenblock Research Institute in San Clemente through a Florida acquaintance whose child has a brain injury.
The Steenblock Research Institute is collecting data from doctors outside the United States who are using stem cell therapies and claiming great success, said Anthony Payne, who runs the institute.
The institute, and the doctors it works with, said they are not sure what it is in the injections that is causing change, Payne said. It could be that something in the shots stimulates the patients' bodies to produce growth factors that stimulate new nerves and connections, he said.
The stem cell therapy provided to Adam Susser is not allowed in the United States because it has not been approved by the Food and Drug Administration, said Payne, a Ph.D. who identifies himself as "doctor."
Payne said he has referred several families of children with cerebral palsy, including the Sussers, to Dr. Fernando Ramirez DelRio in Tijuana.
"We've been following 13 children with cerebral palsy treated by Dr. Ramirez, and 85 percent have shown clinically significant responses in seven areas of motor function and cognitive skills," Payne said.
Three children who were blind can see, and a child who couldn't walk was able to walk down the aisle at a family wedding, Payne said.
Susser said he was skeptical when he first heard the claims. But he was also intrigued.
"I thought about my son 15 years from now saying, 'Dad, you had the money and this was available. Why didn't you do everything you could to help me?' "
Susser and his wife talked about the therapy. And they talked to parents of children who were treated by Ramirez.
"There wasn't a lot of room for him to get worse," Susser explained last week, as his family enjoyed a cruise around San Diego harbor with dozens of tourists. "And there was a chance he could get better. How could I not give that to my son?"
Scientists and ethicists cringed when told about the Sussers' decision making. Although Steenblock calls his organization a research institute, they said it is much different from institutes such as San Diego's Salk and Burnham institutes that are highly regarded in the scientific community.
Those institutes support themselves largely through research grants from the National Institutes of Health and other philanthropic organizations. They employ members of the National Academies of Science.
Steenblock's institute receives no grant money and employs no one with National Academies credentials, Payne said.
Steenblock also has had problems in the past. In 1994, he was fined and placed on five years of probation by the state Board of Osteopathic Examiners in connection with a complaint filed after the doctor examined a 13-year-old girl for chronic acute diarrhea. After visiting the doctor several times, she was taken to a hospital where she had surgeries and later died, according to documents obtained from the licensing board.
In the documents state attorneys said Steenblock failed to recognize the girl's advanced illness, failed to refer her to the proper specialist, failed to treat her in a timely fashion and failed to explain the side effects of an iron shot he gave her.
Steenblock did not admit or deny any of the charges. Instead he agreed to settle the matter and avoided a hearing by accepting downgraded charges and probation and a 90-day license revocation, which was stayed.
Steenblock did not respond to requests for interviews.
Asked about the matter, Payne referred a reporter to the Internet, where he responded to questions about the licensing board's actions posed by the consumer Web site Quackwatch.
The state Department of Consumer Affairs initially investigated the complaints, Payne wrote. Investigators visited Dr. Steenblock, examined his charts and declared that he was in the clear.
A year later, the licensing board reviewed Steenblock's records, including copies of documents so poorly reproduced they were illegible, he said. Steenblock stipulated to the downgraded charges to avoid a lengthy and expensive legal battle, Payne wrote.
Scientists and ethicists said Steenblock's current work also raises procedural and ethical concerns.
Steenblock's institute receives fees from the doctors to which it makes referrals to cover the cost of collecting patient data and for special dietary plans developed by Steenblock, according to Payne. They are tracking 75 patients who have received this treatment since 2003, some of them returning for second and third shots, Payne said. Steenblock and Payne are also writing a book about the treatment.
Experts said their financial interest in having positive results creates a conflict of interest.
Likewise, experts said the institute's studies of the stem cell treatment fall short of Food and Drug Administration standards that foster objectivity.
For instance, the institute measures improvements in the children treated with stem cells by collecting reports from parents, therapists and caregivers, according to Payne.
But Goldstein, the UCSD researcher, pointed out that the tests are not "controlled" as they are in most widely accepted studies, meaning some people get the treatment and others get a placebo.
The FDA also generally requires researchers first to test therapies on animals to look for side effects and then to slowly increase dosages in humans.
But there's no animal or safety data backing up Adam Susser's therapy.
"There's a reason the FDA is so stringent, and it should be: to protect people," Goldstein said. "Look at the Vioxx controversy. There's a case where people criticized them for not being tough enough."
Payne said umbilical cord stem cells have been used for approved therapies for decades and have raised no safety concerns. The cells Ramirez uses are screened for diseases at an Atlanta lab, he said.
The institute uses other methods to ensure that its testing is reliable, such as comparing the results of children in similar age groups and similar disease severity, he said.
Kalichman, the UCSD ethicist, questioned why big drug companies and investors aren't rushing to fund what is supposedly a successful treatment.
"Why isn't this being underwritten by some big drug company that could reap tens of billions of dollars profit? If something like this could happen, it would be effective against almost anything."
The Sussers know revered stem cell scientists don't believe in the treatments. They're unswayed.
"Sometimes someone has to be the guinea pig," Judy Susser said. "As long as it's not hurting Adam."
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