No Evidence to Support Stem Cell Therapy for Pediatric Optic Nerve Hypoplasia

US experts report on their independent study of Chinese stem cell treatment protocol
in the Journal of AAPOS

San Francisco, CA, October 22, 2013

A study performed at Children's Hospital Los Angeles found no evidence that stem cell therapy improves vision for children with optic nerve hypoplasia (ONH). Their results are reported in the Journal of the American Association for Pediatric Ophthalmology and Strabismus (AAPOS).

ONH, an underdevelopment of optic nerves that occurs during fetal development, may appear either as an isolated abnormality or as part of a group of disorders characterized by brain anomalies, developmental delay, and endocrine abnormalities. ONH is a leading cause of blindness in children in North America and Europe and is the only cause of childhood blindness that shows increasing prevalence. No treatments have been shown to improve vision in these children.

With no viable treatment options available to improve vision, ophthalmologists are becoming aware that families with children affected by ONH are travelling to China seeking stem cell therapy, despite lack of approval in the United States and Europe or evidence from controlled trials.  The American Association for Pediatric Ophthalmology and Strabismus has also expressed its concern about these procedures. In response to this situation, pediatric neuro-ophthalmologist Mark Borchert, MD, Director of both the Eye Birth Defects and Eye Technology Institutes in The Vision Center at Children's Hospital Los Angeles, realized that a controlled trial of sufficient size was needed to evaluate whether stem cell therapy is effective at improving optic nerve function in children with ONH. He agreed to conduct an independent study when asked by Beike Biotech, a company based in Shenzhen, China, that offers treatment for ONH using donor umbilical cord stem cells injected into the cerebral spinal fluid.

Beike Biotech agreed to identify 10 children with bilateral ONH (ages 7-17 years) who had volunteered to travel to China for stem cell therapy and who agreed to participate 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 Scientist at the Smith-Kettlewell Eye Research Institute.


# # #


Notes for editors

"Failure of stem cell therapy to improve vision in children with optic nerve hypoplasia," by Cassandra Fink, MPH, Pamela Garcia-Filion, PhD, Mark Borchert, MD, DOI: 10.1016/j.jaapos.2013.06.012.  It appears in the Journal of AAPOS, Volume 17, Issue 5 (October 2013) published by Elsevier.

Full text of the article is available at to credentialed journalists upon request; contact Eileen Leahy at +1 732 238 3628 or e.leahy@elsevier.com to obtain a copy. Journalists wishing to set up interviews with the authors should contact Lorenzo Benet at +1 323 361 4823 or lbenet@chla.usc.edu.

About the Journal of AAPOS (http://www.journals.elsevierhealth.com/periodicals/ympa)
Journal of AAPOS presents expert information on children's eye diseases and on strabismus as it impacts all age groups. Major articles by leading experts in the field cover clinical and investigative studies, treatments, case reports, surgical techniques, descriptions of instrumentation, current concept reviews and new diagnostic techniques. The Journal is the official publication of the American Association for Pediatric Ophthalmology and Strabismus.

About the American Association for Pediatric Ophthalmology and Strabismus (www.aapos.org)
The goals of the AAPOS, the American Association for Pediatric Ophthalmology and Strabismus, are to advance the quality of children's eye care, support the training of pediatric ophthalmologists, support research activities in pediatric ophthalmology, and advance the care of adults with strabismus.

About Elsevier

Elsevier is a world-leading provider of information solutions that enhance the performance of science, health, and technology professionals, empowering them to make better decisions, deliver better care, and sometimes make groundbreaking discoveries that advance the boundaries of knowledge and human progress. Elsevier provides web-based, digital solutions — among them ScienceDirect, Scopus, Elsevier Research Intelligence,and ClinicalKey—and publishes over 2,200 journals, including The Lancet and Cell, and over 25,000 book titles, including a number of iconic reference works.

The company is part of Reed Elsevier Group PLC, a world leading provider of professional information solutions in the Science, Medical, Legal and Risk and Business sectors, which is jointly owned by Reed Elsevier PLC and Reed Elsevier NV. The ticker symbols are REN (Euronext Amsterdam), REL (London Stock Exchange), RUK and ENL (New York Stock Exchange).

Media contact
Eileen Leahy
Elsevier
+1 732 238 3628
e.leahy@elsevier.com


Jennifer Hull
AAPOS
+1 415 561 8505
jhull@aao.org