2012 Articles
Due to copyright restrictions, the full text of articles linked below is available only to the NIH community. Those outside the NIH community can access citations and abstracts.
Other Years
-
Preliminary Report: Stem Cell-Derived Treatments for Eye Disease: Age-related macular degeneration (AMD) is a leading cause of vision loss in Americans 60 years of age and older. AMD is caused by loss or damage to light-sensitive cells (photoreceptors) at the back of the eye, and loss or damage to the supporting cells (called retinal pigmented epithelium, or RPE) that nourish the photoreceptors. Stargardt disease is an inherited form of macular degeneration that shows up earlier in life, rather than during aging. Scientists hope to treat these eye diseases by replacing the RPE cells, and have developed a protocol to coax human embryonic stem cells (hESCs) to differentiate into RPE cells. The eye is a good place to test stem cell-derived treatments, because it is somewhat self-contained and it is immune-privileged (i.e. the immune system is less active there). This means that cells transplanted into the eye are less likely to be attacked and destroyed by the recipient's immune system. Now scientists are testing whether hESC-derived RPE cells can be transplanted into the human eye to replace RPE cells damaged or destroyed by disease. In a Phase I clinical trial, designed to test the safety and tolerability of a proposed therapy, scientists injected hESC-derived RPEs into one eye of one patient with AMD. In a second Phase I trial, they injected hESC-derived RPEs into one eye of a patient with Stargardt disease. (See the description of the Phase I AMD trial and the Phase I Stargardt disease trial on the ClinicalTrials.gov website.) Both patients tolerated the treatment well, and both demonstrated improved vision in the treated eye. These very early results from the Phase I trials provide hope that patients may one day benefit from hESC-derived RPE treatment. However, the current report is preliminary and describes only two patients. In order to gain broad acceptance, the treatment must involve more patients, and it must still be tested in Phase II and Phase III Clinical Trials. Lancet [epub ahead of print]; laboratory of S. Schwartz (non-NIH supported). 2012 January 24.
Other Years