January 2009


Sounding the alarm ...

by Maxine Lipner, Senior EyeWorld Contributing Editor

DECK: About stem cell treatment scam for optic nerve hypoplasia: practitioner warns about website offering false hope

A normal optic nerve in a 2-year-old

Optic nerve hypoplasia in a 2-year-old Source: Lawrence Tychsen, M.D.

UUmbilical cord stem cells can offer hope for many hematological diseases, however, they are also being held out as a cure by one Chinese website for panoply of problems including the ocular disorder, optic nerve hypoplasia. Lawrence Tychsen, M.D., and his partner Gregg Lueder, M.D., Professors of Ophthalmology and Visual Sciences at Washington University School of Medicine and pediatric ophthalmologists at St Louis Childrens Hospital, St. Louis, Mo., have been raising the alarm over sham treatments that may send some devoted parents into near bankruptcy.

Optic nerve hypoplasia occurs at a rate of approximately one in 5,000 live births. The cause of the condition, which occurs during fetal development, remains unknown. Typically the condition is diagnosed in early infancy. The baby at a few months old develops nystagmus and also appears to have subnormal vision, Dr. Tychsen said. They may have sluggish pupillary responses, due to afferent pupillary defects. Funduscopic examination shows reduced diameter to the optic nerve, often with a surrounding hypopigmented ring. While some of the accompanying signs of the disorder (nystagmus and strabismus) can be treated with eye muscle surgery, there is currently no cure for the optic nerve condition. However, one website (http://www.stemcellschina.com) purports that parents might find the answer with the use of umbilical cord stem cells at a center in China. No one else is proposing this treatment anywhere in the U.S., or so far as I know, the Western Hemisphere. The doctors in China are vague about how they propose it works, Dr. Tychsen said. The website builds on the fact that many are enamored with but ill-informed on the specifics of stem cell treatments. They exploit a lack of detailed knowledge about the realities and limitations of stem cell therapy. They appear to be exploiting the hype to the general public about the possible magic of (future) embryonic stem cell treatments, he said. They perpetuate confusion with the notion that the Chinese treatment is effective but simply not permitted in the United States. Umbilical cord stem cells are used quite efficaciously for hematological and oncologic diseases, according to Dr. Tychsen. Cord stem cellswhen you genetically match them from donor to recipientare very useful treatment for blood disease, but not for re-growing nerves Dr. Tychsen said. They do not point out this vital distinction. All they say is, We inject stem cells which will transform themselves into nerve cells and correct brain disease.

Web site hype

The Chinese website offers case reports claiming cord stem cell treatment for conditions ranging from autism to multiple sclerosis. Dr. Tychsen and his partner were alerted about the site by their patients with optic nerve hypoplasia. We have families each week coming into us and relating, Our neighbors are telling us about this wonderful therapy in China that we should look into, he said.

Fees for the treatment can be quite steep. The cost mentioned in the newspaper articles and TV features that Ive read are in the range of $50,000.00, Dr. Tychsen said. Parents, motivated by compassion and love are willing to do almost anything to help their children. Unfortunately, the Chinese doctors are, I believe, preying upon that good will. Combined with ignorance about the mechanisms of stem cell treatments, it can lead to the false hope that the therapy will be efficacious, Dr. Tychsen said. They tell patients that they must travel to China because this effective treatment is not allowed in the U.S. They do not bother to explain that it is not allowed in the U.S. because it has no rational, scientific basis. They imply that the roadblocks are ethical and moral constraints in America, which is, to put it bluntly, hokum.

Suspect science

The science behind the treatment is suspect. The therapy would not be expected even from a theoretical perspective to be efficacious, Dr. Tychsen said. It is implausible that an umbilical cord stem cell, injected into spinal fluid, would make its way to the optic nerve and transform into 10s of thousands of optic nerve axons. Besides, these are non-genetically matched cells for the recipient, and would be immunologicially destroyed. In addition, patients may actually be put at risk by the treatment. First, do no harm, Dr. Tychsen said. One would have concerns about what is being extracted from whom in China, how it is prepared or purified, and what exactly is being injected. Also, theres the looming risk of infection. They could conceivably be injecting, along with what they purport to be cord cells, toxic or infectious accompaniments or contaminants, Dr. Tychsen said.

If practitioners are approached by their patients about the Chinese treatment, Dr. Tychsen recommends taking a look at their article on this (http://www.stlouischildrens.org/content/newsreleasearchive08.htm?page_id=4741&inCtx12view=10&inCtx12pg=0&inCtx12news=3&site_id=1&minor=0&inCtx12news_id=22&major=1). He also suggests educating the parents regarding the science involved. Practitioners should take a few minutes to explain why cord stem cells would not be transformed into thousands of optic nerve fibers, he said. That has not been shown even in rodent experiments, and would be orders of magnitude more daunting in a non-human primate or a human. Hope for optic nerve hypoplasia is actually likely to come from a different source. Perhaps a more practical hope would be neurotrophic (neuromolecular) growth factors in the future, rather than stem cell infusions, Dr. Tychsen said. In addition, even without direct, optic nerve treatment parents worst fears for their children may be exaggerated. Parents of an infant with optic nerve hypoplasia tend to despair when the child is an infant because they think, Im looking at a life of terrible blindness for my baby, Dr. Tychsen said. What they should realize is that most children with optic nerve hypoplasia do fairly well. Most are mainstreamed in school. Many can read with magnifiers. Most adapt much better than initially expected. Children with the disorder do not deteriorate visually and they learn to effectively exploit all of the vision that they have.
Most parents of older children are relieved by how well their children are faring. If you compare the parents when youre counseling them at the first diagnosis to how they are doing five years later, they are, on the whole, much happier, Dr. Tychsen said. They say, I wish I could have seen into the futureyou told us our child would adapt and cope and lead a good life, but we were so afraid.

Editors' note: Dr. Tychsen has no financial interests related to his comments.

Contact information

Tychsen: 314-454-6026, tychsen@vision.wustl.edu.

Sounding the alarm ... Sounding the alarm ...
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