Jun 19, 2015

Combining Stem Cells & Gene Therapy for a ‘Smart’ Sight-Saving Treatment

Dr. Andras Nagy is world-renowned for discovering a method to create stem cells from other cells of the body, a breakthrough which overcame a major hurdle in regenerative medicine. He is also widely recognized for his research that manipulates how cells express different molecules. In his new FBC-funded project, he is using this expertise to transform the treatment of wet age-related macular degeneration (AMD) with a revolutionary plan that would require just a single injection of genetically-modified cells.

In wet-AMD, vision loss is caused by the growth of leaky blood vessels, which is triggered by the over production of a protein called vascular endothelial growth factor (VEGF). For this reason, current treatments focus on administering anti-VEGF drugs (such as Lucentis and Eylea) directly into the eyes, a process that requires regular (monthly or bimonthly) injections. Dr. Nagy’s team recently developed a new anti-VEGF drug which they call “VEGF Sticky-trap.” The team’s long-term objective is to use VEGF Sticky-trap to create a new “smart” cell-based therapy. To do this, they are using stem cells to create eye cells with the ability to secrete VEGF Sticky-trap. They will test if transplanting these cells works as a treatment for wet-AMD. If successful, this strategy of combining cell and gene therapy to deliver drugs has the potential to have a much broader medical impact, far beyond just the treatment of AMD.