Eyeworld

I GENE THERAPY N FOCUS 40 | EYEWORLD | SEPTEMBER 2020 Contact Bharti: neinews@nih.gov Gamm: dgamm@wisc.edu Jurkunas: Ula_Jurkunas@meei.harvard.edu Sohn: elliott-sohn@uiowa.edu the retina to integrate with the retina in place of the patient's degenerated photoreceptor cells." At this point, Dr. Sohn said the visual pathways in the brain would need to already be developed for this treatment. "It may be possible if a baby was missing photoreceptors that stem cell-based photo- receptor treatment could help restore those pathways to help them to see, but that would be something to investigate further down the road vs. starting with someone who is totally blind and already had vision when they were young," Dr. Sohn said. At this point, Dr. Sohn said this research is at a laboratory stage with testing being done on small and large animals. His advice, in general, for ophthalmologists is if they have a patient with an inherited retinal condition to send them to an experienced inherited eye disease special- ist; there might be a clinical trial for a treatment in progress or coming soon. continued from page 39 Reference 1. Pellegrini G, et al. Long-term restoration of damaged corneal surfaces with autologous culti- vated corneal epithelium. Lancet. 1997;349:990–993. Relevant disclosures Bharti: None Gamm: None Jurkunas: None Sohn: None Cautioning patients about stem cell scams David Gamm, MD, PhD, writes in a docu- ment for his patients that the hope for stem cell technology is real, however, there is a "difference between hope and hype." Dr. Gamm warns patients about clinics of- fering "miracle cures" that could actually cause physical and/or financial harm. He notes that almost all stem cell therapies are still in clinical trial or labo- ratory stages and, thus, patients should be skeptical of stem cell therapies that require a fee or claim to be a "cure-all." In his document, which he gave EyeWorld permission to cite, Dr. Gamm writes that ethical scientists engaging in clinical trials enroll patients "without asking for, or ac- cepting, payment (often, they pay YOU)." To avoid scams, Dr. Gamm explains what the retina is and the types of disor- ders that can affect its function. He tells patients that most of these disorders do not have a cure or treatment available yet, which is why there is research looking at turning stem cells into retinal cells and transplanting them. "There is no magic to stem cells, but there is a great deal of excellent, well-de- signed, and well-intentioned research being performed in the stem cell field," Dr. Gamm writes. "Stem cells have unique but variable properties that, if thoughtfully tested and applied, may be of consider- able help to some patients in the foresee- able future. We're optimistic about this future … and you should be, too." "I think we are at the beginning of [stem cell therapy for the eye] because there is so much more that we can do." —Ula Jurkunas, MD

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