Success Story

FDA Approved Drug Treatment for AMD

An FDA Approved Drug Treatment For Age Related Macular Degeneration (AMD). Studies undertaken by Massachusetts Eye and Ear Infirmary researchers led to the first FDA-approved drug treatment for neovascular AMD: photodynamic therapy (PDT) with Visudyne®, a combination laser/drug therapy. Now over a decade in use, Visudyne® was a revolution in patient care. Injected systemically and activated by light, the drug targets and destroys pathogenic blood vessels under the retina, slowing and limiting vision loss without damaging surrounding healthy tissue.

New Gene Therapy for Dry AMD

With support from MLERFi, researchers at Tufts University School of Medicine have developed a new gene therapy that will be tested in human patients In 2016. Specifically, this therapy will target the ‘dry’ form of AMD that currently accounts for 90% of AMD patients, none of whom can be currently treated. This gene therapy uses a harmless virus to deliver a therapeutic gene (that was developed in the laboratory) to the eyes of AMD patients.

New Genes for Macular Degeneration Discovered

Researchers at Tufts University School of Medicine have discovered several new genes for macular degeneration in various biologic pathways, made possible by genotyping and genetic sequencing. The team was the first to identify rare genetic variants for this disease, which are strong predictors of disease and provide targets for new therapies. Most recently they discovered more rare variants in 3 genes, and demonstrated that rare variants explain disease in some families with the typical form of Age-related Macular Degeneration.

Long-term Suppression of Uveitis Using Proteins

Inflammation inside the eye is called uveitis. It is the third leading cause of vision loss in the United States. While most uveitis responds to steroid therapy, 20% do not, and the uveitis becomes chronic. Researchers at the Boston University School of Medicine have found that there may be a way of developing long-term suppression of uveitis by using specific proteins always found in the healthy eye. These proteins stop immune cells from making inflammation, and make the immune cells themselves control each other from making inflammation.

Exciting Discoveries re: Two Rare Children’s Blinding Diseases

Researchers at Children’s Hospital have made exciting discoveries in two related rare children’s blinding diseases: Familial Exudative VitreoRetinopathy (FEVR) and Norrie disease. Both diseases share similar symptoms of abnormal leaky blood vessels in the eye that can steal a child’s eyesight early in life. The research team found that defective genes damage junctions in the blood vessel cells that normally act as glue to seal and protect blood vessels. When the seal is damaged, fluid leaks out from blood vessels to surrounding tissues, harming neurons necessary for vision, hearing and brain functions.

Possible Treatments To Prevent Optic Nerve Damage And Preserve Vision

MLERF research examined the idea that the environment can play a role in the development of glaucoma. The investigation is working to determine if glaucoma could be, in part, an autoimmune disease and if bacteria normally present in the eye could contribute to glaucoma. When glaucoma was induced in mice that were completely germ-free (i.e. had no bacteria), they exhibited the characteristic increase in pressure within the eye, but showed no optic nerve damage.

A Retinal Prosthesis

MLERFi and the Massachusetts Eye and Ear Infirmary have been committed partners for more than two decades in pursuit of the most ambitious technological innovations to date―developing a retinal prosthesis designed to restore useful vision to patients who are blind from age-related macular degeneration and retinitis pigmentosa. The primary goal of the prosthesis is to improve the quality-of-life of severely blind patients by allowing them to navigate in unfamiliar environments.

The First Therapy for Diabetic Retinopathy.

Over the course of two decades, intense and collaborative efforts uncovered the biological underpinnings of a protein called vascular endothelial growth factor (VEGF) in regulating blood vessel growth, and its role in promoting vascular disease inside the eye. Together, these multiple investigations laid the foundation for a new class of inhibitors that thwart the growth of leaky, new blood vessels that can lead to vision loss. In 2004, anti-VEGF drug pegaptanib (Macugen®) received FDA approval for treating AMD, followed by ranibizumab (Lucentis®) in 2006.

Drug to Prevent Dry Eye

Researchers at Schepens Eye Research Institute have determined that there is an increased possibility that a drug can be used to prevent or treat “dry eye.” Pablo Argueso, Ph.D. is studying dry eye disease, a debilitating disease of the cornea (the clear window of the eye) and conjunctiva (the white part of the eye that surrounds the cornea). Dry eye disease affects more than four million Americans. Dr. Argueso discovered that several proteins–called Notch proteins–are important in keeping the cells of the conjunctiva from over growing or from dying.

Using Computational Modelling to Process Visual Information

Researchers at Boston Children’s Hosptal have been conducting #InspiredResearch which combines neurophysiology, visual psychophysics and computational modeling to investigate how neural circuits store, represent and process visual information in the human brain. By recording the activity of neurons and neuronal ensembles in cortex, the Kreiman Lab is providing insights about how the brain interprets the visual world. Inspired by the architecture and neurophysiology of the visual system and funded by the Lions, the laboratory has developed computational models that may enable machines to see and understand visual information.