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No evidence to support stem cell therapy for pediatric optic nerve hypoplasia
Date:10/21/2013

pate in the study; Children's Hospital was to find case matched controls from their clinic. However, only two case-controlled pairs were evaluated because Beike Biotech was only able to recruit two patients. Treatments consisted of six infusions over a 16-day period of umbilical cord-derived mesenchymal stem cells and daily infusions of growth factors. Visual acuity, optic nerve size, and sensitivity to light were to be evaluated one month before stem cell therapy and three and nine months after treatment.

No therapeutic effect was found in the two case-control pairs that were enrolled. "The results of this study show that children greater than 7 years of age with ONH may have spontaneous improvement in vision from one examination to the next. This improvement occurs equally in children regardless of whether or not they received treatment. Other aspects of the eye examination included pupil responses to light and optic nerve size; these did not change following treatment. The results of this research do not support the use of stem cells in the treatment of ONH at this time," says lead author Cassandra Fink, MPH, program administrator at The Vision Center, Children's Hospital Los Angeles.

Confounding the trial was that subjects received additional alternative therapies (acupuncture, functional electrical stimulation, and exercise) while receiving stem cell treatments, which was contrary to the trial protocol. The investigators could not determine the effect of these additional therapies.

"This study underscores the importance of scientifically testing these procedures to validate them and also to ensure their safety. Parents of afflicted children should be aware that the science behind the use of stem cell technology is unclear. This study takes a step toward testing this technology and finds no beneficial effect," says William V. Good, MD, Senior Associate Editor, Journal of AAPOS and Clinical Professor of Ophthalmology and Senior
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Contact: Eileen Leahy
e.leahy@elsevier.com
732-238-3628
Elsevier Health Sciences
Source:Eurekalert

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