Macular degeneration affects the area of your retina responsible for central vision. This condition causes blurriness, dark areas and distortion to your vision; eventually leading to significant vision loss but usually not total blindness.

Studies of two patients suffering from Stargardt’s macular dystrophy reported significant improvements in their eyesight following stem cell therapy, according to a new study.

Embryonic Stem Cells

Embryonic stem cells are an extraordinary type of cell with the power to transform into any cell type in our bodies, serving as the “top bricks” in an infinitely replicating pyramid that eventually gives rise to all of the different kinds of cells we possess. Furthermore, embryonic stem cells may even transform into specific specialized cells such as neurons or eye cells; meaning embryonic stem cells could even create new eyes or retinas to replace ones damaged by disease.

Scientists have developed a way to turn embryonic stem cells into retinal pigment epithelial (RPE) cells that are responsible for our central vision. When deficient due to AMD, RPE cells become critical components of macula which is then lost. As part of their bioengineered implant that mimics Bruch’s membrane and can be directly injected into eyes for restored retinal functionality.

Research published in Science Translational Medicine on April 4 was an important advancement for regenerative medicine, offering hope that macular degeneration could potentially be treated using human embryonic stem cells.

These stem cells are extracted from excess embryos that result from in vitro fertilization treatments for infertility, with participants of IVF programs being permitted to make decisions regarding what to do with their surplus embryos – many opting to donate them for research purposes.

Scientists conducted a small clinical trial by injecting RPE cells directly into two patients suffering from advanced macular degeneration. While the treatment did not halt vision loss entirely, it significantly improved one patient’s condition due to new photoreceptor cells being created within her eye. Furthermore, one patient suffering from Stargardt’s macular dystrophy saw her vision increase from 20/500 to counting fingers held in front of her.

Researchers were successful in identifying the genes involved in the transformation of stem cells into retinal cells. This represents an important milestone, enabling us to study those genes responsible for beginning eye development and creating therapies to replace damaged or defective cells.

Induced Pluripotent Stem Cells

Induced pluripotent stem cells (iPSCs), an alternative to embryonic stem cells, can be created by reprogramming mature cells back to an embryonic state with transcription factors and then being allowed to transform into any cell type in the body – making iPSCs an invaluable tool for scientists looking to study diseases or create treatments.

Researcher have achieved remarkable successes transforming somatic cells into iPSCs and differentiating them into photoreceptor-like cells for use against retinal degeneration. Furthermore, transplanting these cells into animals with eye disease led to improved vision – giving hope to macular degeneration patients that their sight may return one day.

Dr. Golestaneh of the NYSCF Research Institute is leading a team focused on how to prevent AMD by reprogramming adult cells into retinal progenitor cells that can restore normal vision and replace damaged cells. By studying molecular mechanisms that control these progenitor cells, their team aims to find effective strategies for manipulating them for patient benefit.

This technique may also be effective against other forms of macular degeneration, including geographic atrophy (an advanced form of dry age-related macular degeneration). Here, iPSCs would be programmed into becoming retinal pigment epithelial (RPE) cells before implanting them as patches into the eye to nourish photoreceptor cells and potentially delay or even stop further progression of the disease.

Japan’s regulatory body recently granted Japan’s inaugural clinical study using patient-derived iPS cells to treat age related macular degeneration. Massayo Takahashi of Riken Institute’s center for developmental biology will oversee this trial using skin cells from participants to generate replacement retinal cells and transplant them back into participants’ eyes – potentially providing sight restoration therapy that could benefit many with AMD. Multiple similar trials will soon commence around the globe in this fast-developing field of regenerative medicine.

Autologous Stem Cells

Stem cells are being developed as treatments for various conditions, including retinal degeneration. This disease causes irreparable loss of photoreceptor cells that leads to vision impairment as well as decreased quality of life; current therapies only delay its onset or slow its progression; therefore researchers are searching for better therapies that directly replace photoreceptor cells while providing essential trophic support to ensure survival of these cells.

One approach involves programming stem cells derived from blood taken from patients to become retinal pigment epithelial (RPE) cells, which would take on the role of replacing aged retinal pigment epithelial (RPE) cells and nourish photoreceptors in the eye. Although animal studies have proven its success, developing such treatments for humans remains challenging owing to immunogenicity of transplanted cell grafts which may cause rejection; to mitigate against this risk immunosuppression therapy is recommended.

Another potential therapy involves transplanting RPE cells derived from induced pluripotent stem cells (iPSC) to replace damaged photoreceptors in the eye, potentially preventing further damage and restoring some vision. RIKEN team has successfully implanted RPE transplants produced using this approach into five patients suffering advanced dry age-related macular degeneration (geographic atrophy). This study has demonstrated how transplanted cells can replace photoreceptors while improving visual acuity.

Researchers are testing the efficacy of cryopreserved umbilical cord blood and cord tissue stem cells as an effective therapy for macular degeneration, which promises exciting results in the near future.

Unfortunately, not all clinics offering stem cell therapies are legitimate; some have even caused serious medical complications for their patients, including blindness and retinal detachments. According to researchers at the New England Journal of Medicine, several cases were documented where patients lost their sight after receiving unapproved stem cell injections at a Florida clinic that offered various unapproved procedures intended to treat various ocular conditions.

Regenerative Medicine

Regenerative Medicine applies engineering and life science principles to promote regeneration of damaged organs, tissues and cells. As an interdisciplinary field that unites developmental biology with materials science, cell biology and engineering to create innovative treatments to replace diseased cells or repair damage to organs. Regenerative Medicine has experienced rapid expansion since its debut on the healthcare scene; with projected revenues reaching $29.6 billion by 2022.

Researchers from the National Eye Institute and University of Southern California recently conducted a groundbreaking research study, discovering that stem cells may help restore vision in those suffering advanced age-related macular degeneration. iPSCs derived from human skin cells were coaxed into becoming retinal pigment epithelial (RPE) cells before being implanted as patches covering an area damaged by macular degeneration in an eye patch implanted into its recipient’s eyeball, in hopes that these would take over from ageing diseased cells by feeding photoreceptor cells as well as transmitting signals between retina and brain.

iPSCs were also expected to secrete factors that would help maintain retinal cell survival and, while encouraging, were insufficient to fully stop or reverse macular degeneration progression. Still, this study represented an important step forward for regenerative therapy for macular degeneration.

As more clinical trials and regulatory authorities improve oversight, patients may soon realize the advantages of stem cell treatment for macular degeneration. It represents an exciting field with the potential to vastly enhance quality of life for millions worldwide who suffer from this debilitating and widespread condition.

Up until recently, treatments for retinal degeneration were limited to neuroprotective medications that helped slow progression but could not restore previously lost vision. Stem cell therapies offer more viable solutions for treating macular degeneration and other hereditary ocular diseases with ongoing research, innovation, and rigorous clinical trials; hopefully one day these therapies may become a permanent cure for many millions suffering from retinal degeneration and related ocular conditions.