Macular Degeneration and Stem Cell Research

Macular Degeneration and Stem Cell Research

Posted on May.07, 2012, under Innovations for Visually Impaired

Macular degeneration and stem cell research is in its infancy – as is most stem cell research. However, there has been some progress to report in humans .  And even though the progress is quite small, it is still ground breaking.

A  bio-tech company called Advanced Cell Technology (ACT) has its principal laboratory and GMP facility in Marlborough, Massachusetts, and its corporate headquarters in Santa Monica, California. The company specializes in cellular therapy for specific health conditions such as macular degeneration and Stargardt’s disease.

What makes this company unique in the area of stem cell research and treatment is that they have discovered and developed a “first-ever proven alternative method for successful hESC generation without harm to the embryo,  called the “single-cell blastomere” technique, on which it holds broad intellectual property (IP) protection.”

Retinal Pigment Epithelial Cells (RPE)

The RPE layer is one of the layers of the retina that sits right next to the photoreceptor cells which are critical for our vision.  A healthy RPE is critical to the function and health of photoreceptor cells and to the retina as a whole.  If the RPE loses some of it’s function the result is photoreceptor loss or degeneration which leads to loss of central vision.  The progression of macular diseases such as dry macular degeneration to wet macular degeneration may also be the result of the RPE layer becoming less functional.

Macular Degeneration Stem Cell Research

What cells will be transplanted and what procedure will be used to transplant the cells?  Based on the importance of the RPE to macular health, the first stem cell transplant for macular diseases will use hESC-derived RPE cells.  The cells will be given as a subretinal injection into a pre-selected are of the macula.

On July 12, 2011 the first patients in the dry macular degeneration and Stargardt’s disease clinical trial were treated by Dr. Steven Schwartz, M.D at Jules Stein Eye Institute (UCLA). All clinical trials in humans start out as Phase I trials. A Phase I trial has one main purpose – it is  to determine safety. The number of participants in this phase is always very small.  Only 12 people will be recruited for each study. However, the trial will proceed very slowly with only 1-3 participants initially then followed by  a thorough review several weeks later before proceeding with another patient or procedure.

If the transplant proves safe, the next step is to go to a Phase II trial to start looking at efficacy or effectiveness in vision improvement.  According to ACT if RPE cell therapy proves effective it may impact over 200 retinal diseases.

To read more about this groundbreaking research with updates on patients and new clinical trial sites go to:

Macular Degeneration Stem Cell Research

Leslie Degner, RN, BSN