University, declared: "Let's say
these guys are making $20,000 per patient. They're making hundreds of
millions of dollars. They can't take time to document it and publish
it? This is the wrong way to do it."
For many patients, the proper way — years of animal studies
followed by arduous human testing — is too slow. They hope the
experimental cells hit the right targets, like magic bullets, even if
humans are shooting in the dark.
"I went to the Dominican Republic expecting a miracle," DeVivo, of Valencia, wrote on her blog, prayfornathan.org. "Will he walk out of the clinic? Maybe he will say his first full, clear word. What will it be?"
Fertile ground for exploitation
A decade ago, when embryonic stem cells were first isolated,
the controversial cells were thought to be the only ones that could
give rise to all tissues in the body.
"Adult" stem cells, in contrast, were presumed to resupply
only the specialized tissue — say, blood or fat — that
harbored them.
But now, scientists can genetically reprogram specialized
cells to be as versatile as embryonic ones — albeit only in the
lab, with methods that would be unsafe in humans.
Scientific advances, media hype and public confusion have fueled stem cell tourism, critics say.
"There is a risk that patients who are desperate will
misunderstand the amount of progress," said Harvard University
professor George Daley, associate director of the Stem Cell Program at
Children's Hospital Boston. "It is fertile ground for exploitation."
Most stem cell purveyors claim to use blood stem cells. These
cells — readily extracted from circulating blood, bone marrow,
fetuses or umbilical cords — have been used since the 1960s to
treat blood-system diseases, such as leukemia and lymphoma.
There is no evidence that a shot of blood stem cells can
magically fix any problem in every organ system, experts say, but
that's what stem cell tourists are led to believe.
William Rader, a psychiatrist in Malibu, who owns the
Dominican Republic clinic where Nathan was treated, says on his Web
site that the fetal blood stem cell "searches out, detects and then
attempts to repair any damage or deficiency discovered."
Rader did not respond to requests for an interview.
Justin Lowery's case shows why mainstream scientists want controlled studies — and why patients won't wait.
Justin, of Carneys Point, N.J., was born blind, the result of severely underdeveloped optic nerves.
In February, when he was 10 months old, the impact of his
handicap was obvious as Physical Therapist Karen Conner worked with him
at home. Justin couldn't roll onto his belly, crawl or pull himself to
a standing position.
"What are you doing, big boy?" cooed his mother, Lora, as
Conner bounced Justin on a giant ball to strengthen his abdominal
muscles.
The baby did not smile or laugh. He didn't reach for toys,
even ones with lights and sounds. He was easily startled. He recoiled
at most objects put in his hands.
Fast-forward to April, two days after Justin's first birthday
— about a month after he and his parents flew home from Beike's
clinic in Hangzhou, China.
A reporter watched as Justin's eyes followed a lighted ball
that Conner moved in front of his face. He also pulled blinking balls
out of a plastic jar, crawled to get toys, knelt, and pulled himself up
using the sofa. He often smiled.
Jonathan Salvin, Justin's ophthalmologist at Alfred I. du Pont
Hospital for Children in Wilmington, Del., found no change in Justin's
optic nerves but called his ability to follow objects with his eyes "a
pretty significant improvement."
Was it due to the Lowerys' $70,000 odyssey?
All Salvin could say for sure was that occasionally, vision gets better as the brain matures early in life.
"I have seen improvement like this in kids who haven't had this protocol," he said.
While the Lowerys can live with that ambiguity, researchers cannot.
"The unfortunate thing," said John Steeves, a
spinal-cord-injury researcher at the University of British Columbia,
"is that none of us is learning anything that advances our scientific
understanding."
Possibly placebo effect
Many patients or families, including Justin's, report neurological changes within 48 hours of receiving stem cells.
Even stem cell purveyors acknowledge that nerves cannot grow — much less regrow — that fast.
"We believe these immediate results occur from the neural
growth factors used during the transplant process," says Beike's
literature, suggesting growth chemicals revive existing nerves.
Critics cite other factors:
Care that includes surgically opening an injured spinal cord may free compressed nerves.
Believing is seeing, also known as the placebo effect.
No one wants to feel bilked, especially after fundraisers and loans.
"The problem with folks who say, 'Things are a little better'
is that they've just spent a lot of time and money," said Bruce Dobkin,
a neurologist and Rehabilitation expert at the University of
California-Los Angeles. "And they think, 'Maybe I'll continue to get
better.'"
Pain worse after transplant
Six years ago, Tim Case, a workaholic New York real estate
developer, was talked into trying an all-terrain vehicle, just for fun,
by his 11-year-old son.
Case crashed into a tree and, in a heartbeat, ended his life as an alpha male.
Although Case, 48, is a quadriplegic, he is not as disabled as
actor Christopher Reeve — who became a friend after the accident
— was before he died. Case doesn't need a Ventilator, and has
limited use of his arms and hands.
Case, however, has a problem Reeve didn't have: unrelenting, excruciating pain.
Case believes the pain — which was intermittent and
tolerable after the accident — was exacerbated by the cell
transplant he received in 2003 from neurosurgeon Huang Hongyun in
Beijing.
"I think it's just as important to report the negative as the
positive," Case said last month from a wheelchair in his Long Island
home.
Doctors used to dismiss such pain as imaginary. After all, how
can a limb that can't feel a pinprick feel pain? It is now clear that
broken nerves can send dysfunctional signals to the brain.
As Case can attest, these signals can defy pain drugs, including opiates.
On CareCure, an online patient-support forum run by Rutgers' Young, Case begged for advice.
"By 4:30 a.m., I am awake, screaming in pain, stiffness,
burning, very high spasm," he wrote in 2006. "Pain is killing, just
overwhelming."
Early last year, he wrote: "There has to be an efficient way to end my life. Some way to end the pain."
Has it lessened since then?
"As I tell my wife," he said with a sad smile, "the only thing that seems to change is my ability to tolerate more pain."
Still, he insisted the treatment had been worth the risk. He would try a newer version if he thought it would help.
"We're desperate," he said. "There's nothing else."
That, Huang and other purveyors say, is why cells should be used even though it is not clear how they work.
"It is more reasonable and respectful to the patients," Huang
e-mailed last month. "Any standard should consider patients as the key
factor."
Huang worked at Rutgers for three years under Young's
tutelage, surgically implanting specialized cells into rats with spinal
injuries. While these cells are not stem cells, they are believed to be
key to the olfactory (smell) system's ability to replenish nerves
— something no other part of the nervous system can do.
In 2002, Huang returned to Beijing and began offering the rat
operation to humans. He said that on the standard
neurological-Impairment scale, patients had gained at least one grade
of Motor function — such as extending a formerly paralyzed wrist.
Soon he expanded the treatment to other problems, such as stroke and
amyotrophic lateral sclerosis. He also traveled widely, talking up his
methods to Western scientists.
Young, who was born in Hong Kong, has urged his former protege
to conduct controlled studies, called clinical trials — as Young
plans to do late this year with doctors he has trained in China.
Huang "is delivering a treatment that he believes is
effective," Young said. "But I have strongly discouraged him. The
Chinese government dislikes it."
Huang insisted that officials did not object to his procedure, which he called safe.
Of 1,255 patients treated through last year, 76 had
complications, "including temporary headache, temporary modest fever,
incision infection, Cerebrospinal Fluid leakage, etc," he said.
For another view, UCLA's Dobkin and two colleagues found seven
patients who planned to get Huang's cell transplants for spinal-cord
injuries. The Western doctors examined the patients before, after and
up to a year later.
Their tiny study, published in 2006 in Neurorehabilitation and
Neural Repair, found that five of the seven patients had serious side
effects, including pneumonia, bleeding and meningitis.
None had significant improvements.