Therapy offers Ray of Hope for Evans City Girl

November 1, 2007

Therapy Offers Ray of Hope for Evans City Girl


By Karen Roebuck Lexi Smith says she likes being blind. Even so, the 8-year-old Evans City girl says she really wants to see her cat, Jesse, more than anything. She hopes an experimental stem cell transplant in China next summer will give her that chance. "Even if it wasn't experimental, I'd be nervous about going to a foreign country and getting a treatment," said Lexi's mother, Heather, 30, who is trying to raise $50,000 for the treatments and trip. "I can't see myself not trying this. I think we'd be forever asking ourselves, 'What if we had tried this?' " Although umbilical stem cells are used in this country to treat more than 50 diseases, they are not used to treat Lexi's condition, optic nerve hypoplasia, an underdevelopment of optic nerves. Several months ago, Beike Biotechnology, based in Shenzhen, China, began treating children with the condition with stem cell transplants, and intravenous infusions of blood cord serum and a chemical that stimulates nerve growth. The company recommends hyperbaric oxygen treatments for three months after patients return home. A 10-month-old Kentucky boy is in China now, the fourth optic nerve hypoplasia patient to receive the treatment. Some American experts, however, express doubts about the therapy. The treatments likely will not work but should not be dangerous, said Albert Donnenberg, director of UPMC's Hematopoietic Stem Cell Laboratory and professor of medicine at the University of Pittsburgh School of Medicine. Donnenberg, who read the company's literature, sees three obstacles: The stem cells must get to the right place in the body, turn into the right type of cells and not be rejected by the immune system, he said. "They're giving such few cells, you wouldn't know (if they were rejected)," Donnenberg said. Dr. Richard Hertle, chief of pediatric ophthalmology at Children's Hospital of Pittsburgh, said he has seen nothing about Beike's work in medical journals. "I've looked at this, and I just don't see enough evidence to send my patients over there," said Hertle, who has taught and worked in China. "If this was really that spectacular, this would have gotten pushed through and been the lead article in the New England Journal of Medicine or the journal Nature." Hertle said the younger a child receives treatment, the better it tends to work. Treatments in the United States help blind children with optic nerve hypoplasia and other conditions gain some vision, although not by restoring the nerve, as Beike is claiming to do, he said. Lexi, who has limited light perception, turns 9 on Nov. 25. "I think, at this point, (Lexi) has an excellent chance of some vision recovery, given what we've seen with the other four children, although there are no guarantees," said Kirshner Ross-Vaden, Beike's Chicago-based vice president of foreign patient relations. Lexi is in the gifted program at Evans City Elementary School, part of the Seneca Valley School District. An aide translates her assignments to and from Braille. At 1 month old, she was diagnosed with the absence of the septum pellucidum, a thin membrane in the middle of the brain often linked to hormonal problems, which Lexi has not had, and optic nerve hypoplasia. "I was always told there was nothing that could be done," Smith said. She scoured the Internet in vain until Lexi's dad, Jeff Moraski, saw a news report about a blind man being treated with stem cells. Smith eventually found the story of Rylea Barlett, 5, of Webb City, Mo., who has optic nerve hypoplasia. After reading on Beike's Web site about its success treating brain injuries and other neurological conditions, Rylea's mother, Dawn Barlett, 30, e-mailed the company and asked if it would be willing to try the procedure on her daughter. Rylea, who had no light perception, began treatments July 4. She can distinguish shapes and some details, such as whether her mother is wearing glasses or lipstick, up to six feet away and has 2-400 vision, according to her mother. "Within two months, she grew 1 1/2 inches, where she hadn't grown in two years," her mother said. Beike formed two years ago to treat patients with neurological conditions with umbilical stem cell transplants and the company recruits American and foreign patients. The transplants are done at 11 facilities in China, two of which accept foreign patients, Ross-Vaden said. As of October 2006, Beike said it performed stem cell transplants on more than 2,000 patients with neurological problems, including Alzheimer's disease, brain injury, cerebral palsy and multiple sclerosis. "Stem cells are not a cure, and I think that has to be stressed. These cells are a treatment for a lot of disorders," Ross-Vaden said, "but they're not going to completely resolve or reverse something." When Smith read Rylea's story about three weeks ago, she said, "I just sat at the computer and I bawled. I was shaking, crying. There was hope." Her family immediately started raising money to take Lexi to China in June. "I think it will be exciting -- getting the treatment and being in the (hyperbaric) oxygen tent," Lexi said. "I'm excited about that, because I like tents, and it's like a tent." And she longs to see her cat, Jesse. "I'm a cat lover," Lexi said. "I have to see what cats look like."