First U.S. Patient With Dry AMD Receives Autologous Stem Cell Therapy

In a groundbreaking clinical trial, surgeons at the National Institute of Health in Bethesda, Maryland, implanted tissue made from a patient’s cells in hopes of treating advanced dry age-related macular degeneration (AMD). With no treatments currently available for AMD, this revolutionary new therapy could pave the way for the plethora of potential uses for stem cell therapy. 

Leveraging Stem Cell Therapy to Treat Dry AMD

Among older Americans, dry AMD is a leading cause of vision loss where cells in the macula, the part of the eye responsible for processing your central vision, slowly deteriorate and die off without regenerating. Progression varies by patient, but common symptoms include seeing less or dulled colors, blurred vision, and gaps or dark spots in a patient’s vision. Dry AMD is not related to dry eye but is the opposite of wet AMD, where blood vessels leak into the area behind the retina, resulting in scar tissue and causing retinal cells to stop functioning properly.

In an effort to develop an innovative new treatment for dry AMD, researchers at the Ocular and Stem Cell Translational Research Section developed induced pluripotent stem (iPS) cells using the patient’s blood cells. Scientists can program iPS cells to become almost any cell type in the body. In this case, researchers programmed the patient’s iPS cells to become ​​retinal pigment epithelial (RPE) cells. RPE cells are vital to dry AMD because they support light-sensing photoreceptors in the retina. Deterioration of RPE cells causes vision loss. 

With the patient-derived and modified RPE cell patch, which measures 2 x 4 mm and contains about 75,000 RPE cells, a leading surgeon in the field, Amir H. Kashani, M.D., Ph.D., implanted the cells into the patient. The procedure is part of a ½ Phase clinical trial to determine the treatment’s safety. If all goes well, the new treatment could serve as a stepping stone for not only autologous cell therapy but stem cell therapy as well, which could tremendously benefit medicine. 

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Growing Interest in iPS Cell Therapy and Its Applications

iPS cells are a very recent development in science and medicine, coming to light in a 2006 paper by Kazutoshi Takahashi, which won the researcher a Nobel Prize. Since then, researchers around the world latched on to the idea, trying to wrangle the technology to develop treatments to regenerate or replace damaged or missing tissue. 

Now, 12 years after the iPS cell’s introduction, over 100 ongoing clinical trials are trying to leverage the technology’s potential for a range of diseases worldwide. According to a Nature article published in 2020, about 36% of all trials using iPS cells are conducted in America, followed by 15% in France. In total, 16 countries at the time had ongoing clinical trials using iPS cells as the primary form of treatment. 

The potential applications of iPS cell therapy include a broad range of diseases, most of which are non-communicable diseases. Opthamalic diseases are the most common treatment area for iPS cell therapy, with 24.4% of clinical trials aiming to treat some kind of eye disease. Other non-communicable diseases make up 22.1% of iPS cell therapy-focused clinical trials. Additional areas of interest include cardiovascular diseases and neurological disorders. 

Many researchers see the potential in iPS cell therapy and continue to work to leverage the innovative therapy’s capabilities. The first US patient to receive autologous iPS cell therapy for dry AMD is a huge leap forward for the field of study, which could provide treatment options to disease areas with no other alternatives.

Author

Reed Slater