Stem Cell Treatment for Children with Eye Nerve Disease Called “Medical Hoax”
Two pediatric eye surgeons expressed alarm over what they label a “21st century snake oil” scam. Recent newspaper stories – including several from Missouri -- have reported parents flying their children to main land China for umbilical cord stem cell (CSC) infusions. The cost of these treatments, paid for entirely out-of-pocket by the parents, can be $50,000 or more. CSCs are extracted from the umbilical cords of Chinese mothers and their newborns, and injected into the fluid around the spinal cord of the American children. The parents are led to believe by doctors abroad that these CSCs are an effective treatment for optic nerve hypoplasia (ONH), a disease causing partial-blindness at birth.
ONH is growth failure of one or both optic nerves during the first trimesters of pregnancy. The nerve is the “cable” connecting the eye to the brain and each nerve should have one million fibers; in ONH the number of fibers ranges from 200,000-800,000. ONH is not hereditary and the exact cause is unknown. ONH affects about 1 in 5,000 newborns.
Lawrence Tychsen MD and Gregg Lueder MD, Professors of Ophthalmology at St Louis Children’s Hospital and Washington University School of Medicine, diagnose and treat dozens of children each year with ONH. They are concerned that the CSC reports will mislead many parents of children with ONH, who may bankrupt savings, go deeply into debt, or organize fundraisers to pay for sham treatment.
Although some parents claim improvement in their child’s vision after returning from China, Tychsen and Lueder caution that no objective visual gains after CSC treatment have been demonstrated in any child with ONH. They can measure visual improvements objectively in infants and toddlers using non-invasive nerve and brain imaging and electronic measures of visual brain activity. They add that one would expect “a powerful placebo effect after these purported treatments. The temptation to believe vision had improved, after the expenditure of so much time and money, would be difficult to resist.” Aside from grave ethical concerns, they say that the injections could be dangerous, introducing infection or toxic matter into the brain fluids.
Tychsen, who is also a neurobiologist studying visual brain development in infant monkeys, listed a number of reasons to disbelieve reports of improvement. He says that, first, CSCs placed in human spinal fluid would not be transported into the fibers of the optic nerve. Second, CSCs have never been shown to transform into optic fibers, even in fish or rodent experiments. In a monkey or human, the task would be “several orders of magnitude more complex”. Third, to improve vision 100,000 or so fibers would need to grow, not just a few, and each of the fibers would need to connect precisely in the brain.
He explained that “CSCs are used legitimately throughout the US to treat blood diseases (such as leukemia) when the donor and recipient are genetically matched (allogeneic transplant). But CSCs from an unrelated person are rejected and destroyed. Even if an unmatched CSC survived, found its way inside the optic nerve, and transformed itself into a new fiber, the fiber would need to find the correct connection among more than 500,000 connections in the visual brain.” Tychsen says such a series of events would be “so improbable as to qualify as miraculous, the equivalent of a chimpanzee typing the five acts of King Lear at one sitting”. He believes that experiments by neuroscientists devoted to the discovery of nerve growth molecules may hold the best hope for future cures.
Dr. Lueder, who is ophthalmology representative to the American Academy of Pediatrics, pointed out that parents of ONH children should not despair; “Many babies born with ONH will have some improvement as they mature, because they learn to exploit more effectively the optic fibers that remain.” Children with ONH can also achieve some improvements with surgery for eye crossing and nystagmus (roving movements of the eyes). He added that many ONH children function reasonably well in school using enlarged print, magnifiers and other aids for the visually impaired.