Couple's search for hope leads to controversial treatment in China

Eight-month-old Cash uses his forehead to touch everything.

Toys, food, even the floor.

"He gets carpet burns on his head from scooting around on the floor so much," said his mother, Morgan Williams.

Born blind, Cash hasn't learned to grasp things with his hands. His parents are confident that he'll do just fine despite his vision loss. Still, said Jesse Williams, Cash's father, if there was a chance to restore your child's eyesight, wouldn't you do that?

"We want to give him every opportunity to have vision," he said. "And right now, the only hope we have for that is in China."

The Williamses want to take Cash to a hospital in Qingdao, on China's eastern coast, for stem cell treatments that they are told will improve his vision. Although their journey is still months -- and at least $50,000 -- away, the family hopes to join the thousands of foreign patients who travel to the Far East seeking a cure for seemingly incurable conditions.

Despite warnings that stem cells harvested from umbilical cord blood cannot cure blindness, multiple sclerosis and other conditions, people still flock to China, Thailand and other countries each year.

"We have good reason to believe this will work," said Morgan Williams, 23. "More children get results than don't."

Many doctors and scientists are not convinced.

Of the nine children he has seen who have received stem cell therapy in China for eye disorders, not one has shown objective signs of improvement, said Dr. Lawrence Tychsen, professor of ophthalmology in pediatrics and neurobiology at Washington University School of Medicine in St. Louis.

"Everyone desperately wants the child to improve, but it's sham therapy," said Tychsen, who serves on the board of the Child Vision Center, a nonprofit research facility in Fort Worth. "It's basically 21st-century snake oil."

The Foundation Fighting Blindness has also raised concerns.

"No one we are aware of has seen any objective outcomes from children who have been treated in China that conclusively prove the so-called stem cell treatment is having a positive effect," said Stephen Rose, chief research officer.

The Food and Drug Administration does not issue warnings about products unless they are imported to the United States, but since other countries do not require the same rigorous premarket review, there's no assurance that what patients receive when they go overseas is safe and effective, said Karen Riley, an FDA spokeswoman.

The Williamses say the skepticism has not dissuaded them.

"Nobody has told us we're crazy," Morgan Williams said. "No one has said 'don't do it.'"

Dr. Pierre Robert, Cash's pediatrician, supports the family's decision.

"I believe the stem cell therapy to be the only hope Cash has for any chance of vision," he said.

Any improvement

Since 2005, when Beike Biotechnology was founded in Shenzhen, more than 8,000 people have been treated with no serious or adverse side effects or safety concerns and with a high success rate, the company says on its website. About 400 international patients have been treated for eye disorders since 2006, a company spokesman said.

Dozens of success stories online led the Williamses to Beike.

They checked out the facility before establishing a fund for their son's treatment and starting a blog at Jesse Williams, 33, a sales manager for a Plano car dealership, said he'll save and raise as much money as it takes.

The couple, who grew up in Grapevine and now live in Plano, are already anticipating the 20-hour flight to China, where they will stay with Cash in the 32-room Qingdao Chengyang People's Hospital.

The hardest part, the couple said, will be leaving behind their little girls, Cassidy, 2, and Kylie, 4.

But it won't be easy in China, either.

Cash will be sedated while 10 million to 15 million stem cells are delivered through an IV or a lumbar puncture. He must lie still for six hours afterwards. Cash will also undergo acupuncture and other therapies. When he returns home after 30 days, he'll need hyperbaric oxygen chamber treatments for three months to help the stem cells grow, Morgan Williams said.

Six injections of certified stem cells tailored to the patient's condition cost about $26,300, a Beike spokesman said. Hospitalization, daily rehabilitation and transportation is included.

Morgan Williams said they were told to factor in airfare and other expenses in planning for Cash's trip.

Although Beike has reported that its therapy is safe, Tychsen said risks include infection and allergic reactions associated with injecting foreign cells into a child's central nervous system.

This year, a 46-year-old woman being treated in Thailand died from an infection after undergoing stem cell therapy for kidney disease. Last year, a 13-year-old patient developed brain tumors after undergoing stem cell therapy in Russia.

Any improvement, even if it means just being able to recognize light from dark, will make the trip worthwhile, Morgan Williams said.

"Right now he has no independence," she said. "If he plays with a toy and throws it, he can't find it."

Septo-optic dysplasia, the disorder that Cash has, affects about 1 in 10,000 births and can lead to neurological problems and small stature, symptoms Cash has not shown.

A Beike spokesman said the "program cannot cure blindness or other underlying conditions, rather they can improve the patient's quality of life in dealing with their particular disease." Although stem cell research is being done in the United States, it's not moving fast enough for families such as the Williamses.

"We think America is the land of opportunity," Jesse Williams said. "But here we are going to Communist China to do something we can't do here."

U.S. researchers wary

Researchers such as Dr. Lawrence Goldstein, who is on the board of the International Society for Stem Cell Research, empathize with families who are desperate for a cure. But they balance that with reality: It takes time to do research properly. Stem cell therapies have been proved effective only for blood disorders such as leukemia and some skin conditions.

Limited research is being done around the country, but it does not come close to matching the need. There are no legally marketed stem cell treatments in the U.S.

"It's a really hard problem, and as a society we are woefully underinvested in it," said Goldstein, a researcher at the University of California, San Diego. "As scientists, we do the best we can to move these things along as quickly as possible."

But the concept of using stem cells from umbilical cord blood to treat blindness is an impossibility, scientists say. The stem cells would first have to miraculously overcome multiple hurdles and then find their way to the optic nerve and penetrate the eye for that to work, Tychsen said.

"It would be the equivalent of setting a monkey in front of a typewriter and somehow he's going to be able to type King Lear," he said.

Beike said it provides certified stem cell products tailored for the patient, and the company has published scientific papers, a spokesman said.

Among the company's most famous success stories is that of a teenager who had only light perception in one eye and 20/4,000 vision in the other when she was treated four years ago. The girl's vision improved so much that she recently was able to start driving.

There are many explanations for such "miracles," and not the least is the placebo effect. "People who know they are being treated and really want to get better look for any signs of improvement," Goldstein said.

That's why double-blind clinical trials are held in the U.S., he said. In such trials, neither the researchers nor their subjects know whether they are in the experimental group or the control group.

"We know from years of medical experience that if you don't have a double-blind trial you can get fooled," he said.

Such success stories appearing on China Stem Cell News' website are plentiful, along with links to clinics providing the therapy.

"You can go to the Web, type in stem cells and bring up dozens, if not hundreds of advertisements for clinics all around the world for diseases for which there is in many cases no conventional treatment," Goldstein said.

"In less well-regulated environments, you can advertise anything, charge anything and pretty much claim anything."

That can't happen in the United States, he said.

"If you have a treatment, you have to prove that it works," Goldstein said.

Providing hope

Some patients say they're living proof.

In 2008, Fort Worth police Sgt. Preston Walker went to Costa Rica for relief from the fatigue and depression of multiple sclerosis. The stem cell treatment cost about $20,000, but it appeared to work.

Today, Walker has more energy and rides his 10-speed bike 100 miles a week.

"I don't know if the stem cells just provided hope or if they actually are helping with the healing process," he said. "I have no idea; I only know that I have no more fatigue or depression and I feel good."

Cash is a happy baby who will learn to read in Braille when he is older, Jesse Williams said.

"We are prepared for the fact that he may never see," Morgan Williams said.

The couple said they just want to give Cash the best future possible.

"It's not like we don't accept him the way he is now," Jesse Williams said. "He is, after all, our precious baby boy."

Jan Jarvis, 817-390-7664