
Exploring stem cells as neurologic treatment: Not a closed case
While some results have proved to be less than promising, potential remains intact
The role that stem cells can play in ophthalmology may be akin to science fiction, but stem cells can be useful to develop a better model of brain diseases.
Reviewed by Alfredo A. Sadun, MD, PhD
Research into the use of stem cells as a treatment of neurologic disorders is in its infancy, and maybe, more wishful thinking than reality.
Mixed results
So far, the results are more frightening than promising.
One company,
Dr. Sadun, the
“The septum pellucidum and corpus callosum are affected but sometimes the hypothalamus is also, and this is a major clinical issue because of the association of the hypothalamus and serious endocrine problems,” he said.
Beike Biotech performed a study of 48 patients with optic nerve hypoplasia, for which the outcome measure was determined by the responses to a patient questionnaire completed 16 days after the procedure. The company reported that 60% of patients improved immediately, and 77% at one year. They did not do any visual outcome studies.
Independent review
However, as Dr. Sadun pointed out, an independent review of this study was performed at
The optic nerve head size was evaluated at one, three, and nine months postoperatively. The visual acuity, and contrast sensitivity were assessed. The findings of the study (Fink et al. AAPOS 2013;5) indicated that nothing improved, Dr. Sadun recounted.
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This research is exciting to Dr. Sadun, who said he believes that stem cells may be beneficial for treating the corneal endothelium, trabecular meshwork, and
“I am not sanguine that stem cells will work for diseases of retinal ganglion cells because there are no endogenous stem cells for retinal ganglion cells; the mesenchymal cells may be helpful because they secrete neuroprotective factors in the vitreous,” he explained.
Dr. Sadun noted that replacement of the retinal ganglion cells requires consideration of the problem of specificity. “The cells are attached to specific parts of the brain to exactly the right cell and interfering with this circuitry is likely to be very problematic,” he said.
Conclusion
Dr. Sadun provided two take-home messages. First, regarding research, if it is about treatment today with stem cells, neurons have circuitry while stem cells are clonal. Stem cells are not going to work when dealing with precise circuitry issues. “At the moment, the use of stem cells for optic neuropathies is nonsense,” he said. Second is the idea of using stem cells to develop a better model of brain diseases. Dr. Sadun said he believes this is where the promise lies.
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Disclosures:
Alfredo A. Sadun, MD, PhD
E: [email protected]
Dr. Sadun has no financial interest in the subject matter.
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