Aug 16, 2016

One Step Closer To A “Smart” Stem Cell Therapy

Today, there are no approved stem-cell treatments for blinding eye diseases. Dr. Andras Nagy’s research is trying to solve this problem by developing a safe and “smart” stem-cell therapy. To do this, his research team is combining gene therapy and stem cell therapy to generate specialized stem cells that have been genetically modified to release a sight-saving drug. As a result, these specialized, genetically-modified stem cells have the ability to replace dying cells, and also prevent further cell death.

Dr. Nagy is famous for making multiple major breakthroughs in regenerative medicine. First, he developed a technique to create stem cells from other cells of the body. Next, he developed a technique that manipulates how cells express different molecules. Last year, Dr. Nagy was awarded funding from Fighting Blindness Canada for his project that combines these techniques to develop a new “smart” stem cell therapy for age-related macular degeneration (AMD), the leading cause of blindness in people over the age of 50.

The vision loss that occurs during wet-AMD is caused by the growth of leaky blood vessels, which is caused by the over production of a protein called vascular endothelial growth factor (VEGF). Current treatments administer anti-VEGF drugs 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.

Although there a number of very effective anti-VEGF drug therapies to treat AMD, these treatments require regular injections into the eye, which can be a burden to patients and their families, particularly those who need to travel long distances. To help solve this problem, Dr. Nagy is working on a single “smart” cell therapy that would cure AMD with a single treatment of genetically-modified cells.

After one year of funding from FBC, Dr. Nagy and his team are more optimistic than ever about their research, stating: “So far we have been successful in generating cells that can produce VEGF Sticky-trap. This is by far the safest approach to treat macular degeneration and other related diseases.”

We are very excited about Dr. Nagy’s progress, so we asked him: How much longer will it be before there are approved stem cell treatments for blinding diseases? And, what can we do to speed things up?

Dr. Nagy’s thoughtful answer points to the importance of working together to raise awareness about stem cell science and warns of the dangers of untested approaches. He explains: “It is only a matter of time until the first cell therapy clinical trials for eye diseases start in Canada. As clinical trials for cell therapy are very expensive, funding is absolutely crucial in accelerating progress. In addition, for researchers, thinking ahead early enough and getting the right people involved is an asset. For the public, it is important to raise awareness and understanding about stem cell science. Stem cell tourism and the use of unproven, unregulated and potentially dangerous therapies can add a burden to Canada’s health care system and, as a result, decrease funds available for regulated cell therapy.” Thank you to all of the FBC donors who are helping to drive this research forward.

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