Risk vs. hope: Heading overseas for stem cells

Published: December 22, 2006  |  Source: stemcells.nih.gov
120

FORT LAUDERDALE, Fla. – David Aldrich, 49, was paralyzed in a boating accident more than four years ago, and had made his wishes known. If he were hospitalized again and in a near-death situation, he did not want to be resuscitated.

He thought there was no hope he would walk again.

But with his family’s prodding and financial backing, the former licensed boat captain from Florida traveled to China, where doctors are treating people with spinal cord injuries and other conditions with stem cells obtained from umbilical cord blood. Out-of-pocket costs are high – $20,000 to $25,000 on average.

While some umbilical cord stem cell treatments are available in the United States with special permission from the U.S. Food and Drug Administration, Aldrich said he opted to go to China because spinal cord treatments are not yet available here. The Miami Project to Cure Paralysis is doing research on embryonic stem cells in animals, but is not ready to try the treatments in humans.

Slowed by political opposition, embryonic stem cell research is limited to a small number of cell lines that qualify for federal research dollars. Private companies have raised more than $440 million to advance their work outside the confines of federal funding, but for the most part are still in the early stages of laboratory work and animal experiments.

Several states have enacted stem cell research programs, but they are in their infancies. In Florida, the Supreme Court has been asked to sort out the language in competing proposals for constitutional amendments: One would prevent spending money on embryonic stem cell research; another that would require the state to appropriate $20 million annually for 10 years to pay for such research. Voters could decide the issue in 2008.

Congress is expected to take up the issue again next year.

Opponents of embryonic stem cell research argue that the work is unethical because they say it requires ending a human life – that of an embryo.

“We are in favor of all types of adult stem cell research and using umbilical cord blood,” said Lynda Bell, spokeswoman for the Florida Right to Life Committee. “We support any research that does not require the destruction of a human embryo, but when you’re destroying human life to do experimentation, that is wrong.”

As the debate rages, some desperate patients are going to other countries – China, Mexico, the Dominican Republic, Brazil, Portugal, Sweden and elsewhere – hoping for miracle cures now.

Kayce Barnes, of Florida, has taken her 7-year-old daughter, Brianna, to the Dominican Republic twice for infusions of cells she hopes will save her child from Batten’s disease, a rare, fatal degenerative neurological condition.

“If there’s any chance to save my daughter’s life,” she said, “I’ll do it.”
Brianna’s treatment, offered by a California company called Medra, is performed at a hospital in the La Romana region of the Dominican Republic.

A stem cell trial to treat Batten’s disease is planned at Oregon Health and Science University’s children’s hospital, but only six children will be treated initially and Brianna is not a candidate. If the treatment proves safe, the trial could be expanded to include more children, but it could take several years to win FDA approval for all children with the condition.

“She was denied from that trial because they want kids who are less medically involved, kids with very few symptoms,” Barnes said.

Researchers in the United States have successfully treated conditions such as sickle cell disease, some genetic diseases and immune deficiencies using umbilical cord stem cells, but the treatments require permission from the FDA and other regulatory oversight, such as institutional review boards, safeguards that may be lacking in other countries.

Bernard Siegel, executive director of the Genetics Policy Institute, which has offices in Wellington, Fla., and Washington, D.C., said going offshore for such treatments is risky.

“We pay close attention to these so-called stem cell therapies that are taking place outside the United States, and my own personal view is that we must take a very cautionary approach to this,” said Siegel, who has organized several international meetings of stem cell researchers.

Three scientific journals, Science, Nature and The New England Journal of Medicine, have reported on China’s stem cell programs, and a British trade group that visited there two years ago reported that China might be at the forefront of international stem cell research.

Aldrich’s sister-in-law, Sally Aldrich, a retired nurse who lives in New Hampshire, found the site for Stem Cells China and began making inquiries.

She explored other options before she and her husband, Paul, offered David the China option.

“There are people who are saying it’s too early to do this, but David and Paul and I are adventurers. You don’t get anywhere without risk,” she said. “For people like David and Christopher Reeve, all it takes for them to die is an infection.”

Reeve, a prominent actor paralyzed when he was thrown from a horse, died Oct. 10, 2004, of an infection.

W. Dalton Dietrich, scientific director of the Miami Project, said there have been preliminary talks with the FDA about how best to conduct clinical trials using a combination therapy that the Miami researchers have shown can restore 70 percent of the movement in paralyzed limbs of rats.

“We’re at that phase of the research where we’re really thinking seriously about translating it into people, moving things from the bench to bedside, but it’s difficult to tell how long that will take,” Dietrich said.
When people consult him about leaving the country for stem cells, he said, he advises against it.

“We tell them this is premature and may be dangerous,” he said.

David Aldrich thinks the treatments and the Physical Therapy he received in China have given him more movement in his fingers and legs. His spinal injury is classified as “incomplete,” meaning the cord was not severed. His vision was impaired, and he was on a Ventilator for months and in the hospital for a year.

He credits the China treatment and Rehabilitation with his ability to sit up on his own, and he said his vision also has improved.

“I have more flexibility in my fingers. That’s pretty exciting. I can move both legs, and I’ve got a lot more control of all my limbs. I believe what I’m experiencing is a result of the strength training, and the rehab work while I was there. You can’t just put in stem cells one day and get up and walk the next day,” he said.

Aldrich said he hopes the climate for research in this country improves soon.

“People are looking for hope, and it’s too bad they have to go outside the U.S. to find it. That’s all any of us have to look forward to is hope, to cure our disease or improve our quality of life.”

The possibility that stem cells may cure many ills has led to an explosion of clinics around the world offering cell-based therapies, but experts urge caution.

Many countries have no guidelines for use of stem cells in research or human treatment applications, so patient safeguards are lacking.

If you opt for a treatment in another country, you will likely be excluded from later clinical trials in the United States because your previous treatment might skew the results.

Costs can be high and are not covered by insurance.

For more information on stem cell research, visit www.genpol.org/ and http://stemcells.nih.gov/.

– By Nancy McVicar, South Florida Sun-Sentinel