Novel Strategies Set the Stage for Next Generation Therapies

While significant progress has been achieved in treating retinal diseases, researchers at Mass. Eye and Ear/Schepens Eye Research Institute remain deeply committed to improving current therapies, refining diagnostic tools, and developing new therapies that leverage advances in biotechnology and human genetics. Collaborations are ongoing throughout the department’s Centers of Excellence and Institutes. Some current avenues of study and research include:

AMD Center of ExcellenceTo target new disease pathways, researchers in the AMD Center of Excellence are studying genetic and epidemiological risk factors that make some people more susceptible to AMD. They are also trying to improve their understanding of early disease progression using dark adaptation, novel imaging devices and metabolomics. Researchers are also developing neuroprotective agents in combination with anti-VEGF therapies to prevent photoreceptor cell death – the ultimate cause of vision loss in AMD.

Ocular Genomics InstituteIn 2013, the Ocular Genomics Institute (OGI) published the most thorough description of gene expression in the human retina to date (BMC Genomics), which is crucial to understanding how diseases of the eye develop and lead to vision loss. This is a valuable resource for the vision research community, and the data are available via the OGI website. OGI researchers also demonstrated that the complement system, which is part of the immune system, plays a critical role in the early stage of an inherited macular degeneration (Human Molecular Genetics). Drugs that inhibit specific complement system activities are being clinically tested
as treatments for AMD.

Ocular Regenerative MedicineMembers of the Ocular Regenerative Medicine Institute (ORMI) are participating in a Phase I/II clinical trial to evaluate the safety of human embryonic stem cell (hESC)-derived retinal pigment epithelial (RPE) cells for dry AMD. Mass. Eye and Ear is serving as a clinical trial site for the U.S. and European study, which is being conducted by Advanced Cell Technology, Inc., a leader in the field of regenerative medicine. ORMI members are also developing engineered biomaterials that may be used to deliver neuroprotective agents or stem cells to the retina with plans to conduct a first-in-man restorative stem cell trial in early 2015.

Mobility Enhancement & Vision RehabilitationMembers of the Mobility Enhancement and Vision Rehabilitation Center of Excellence are working to find creative ways to help patients with impaired vision achieve greater independence and mobility, and a better quality of life. One vision-enhancing technology is SuperVision+, a free smart phone magnifier app available for iOS and Android platforms. In addition to magnifying small print (i.e., medication bottles and restaurant menus), the app has a unique image-stabilization feature that “locks” shaky images caused by hand tremors. Another tech-savvy application is utilizing video games to help patients develop navigation skills (way-finding) and improve their sense of independence. Center members are also involved in research addressing contrast sensitivity, fundus-related perimetry, and visual hallucinations in patients with vision loss, as well as development of a retinal prosthesis.