Introduction
As many are aware of, the new world is blooming with dangerous and unique discoveries, specifically in the field of medicine. Cells, the “microscopic life givers,” live in the body in numbers over 3 billion.
With cells, specifically stem cells, cures to many diseases are starting to make progress, with the progress slowly closing in.
Definitions
Stem cells - Stem cells are cells that are able to adapt to mostly any purpose. Stem cells are mainly found in bone marrow, muscles, and in blood however, the main and most plentiful source of stem cells are found in embryos.
Stem cells are widely regarded as “versatile,” with scientists claiming they can be used “for the treatment of a variety of diseases that plague mankind” (Bongso and Lee 1).
AMD - “Age-related Macular Degeneration” - A condition amongst elderly people leading to “vision loss among older Americans [that] currently has no treatment” (National Eye Institute (NEI)).
Overview
Currently, AMD plagues 20 million elderly citizens of the United States and continues to grow (Genentech). The focus of this article will be on dry AMD, specifically looking at advanced AMD, known as GA (geographical atrophy). Looking for the cure to this disease has been attempted by previous scientists throughout the years. Stem cell research has been a recent new angle for researchers to look at a possible treatment. Currently, the NIH in accordance with John Hopkins University has been looking at a stem cell therapy to heal symptoms of AMD.
A visual representation of how someone with AMD may see the world.
The Study and Trial
The NIH put forth the years of research done on stem cells to finally run trials on what many see as a leading step to curing AMD. This specific trial is in reference to the 1/2a trials.
The trial is fairly simple. Breaking it down, the trial involves using iPS-derived retinal cells.
These stem cells have been taken from an adult and reprogrammed into a more versatile embryo-like stem cell. The picture below accurately represents the reprogramming:
In the case of AMD, the iPS-derived cells would work to repair and reconstruct RPE (retinal pigment epithelium) in the eye, to improve light detection by the photoreceptor cells.
iPS cells transplanted into the patient's eye may leave them with an improved condition.
In this trial, patients had their blood extracted for stem cells.
The trial consisted of the transplantation of the iRPE stem cells, used to replicate healthy and replace damaged epithelial cells in the eye’s RPE.
The trial consisted of participants with certain criteria:
Age: 55 years of age or older;
Diagnosis of AMD and presence of GA involving the central retina in at least one eye;
Best-corrected visual acuity (BCVA) between 20/100 and 20/500 (first cohort), or between 20/80 and 20/500 (second cohort), and a fellow eye that has same or better BCVA;
No history of exudative (wet) AMD or prior treatment for exudative AMD in EITHER eye;
No glaucoma or significant elevation of intraocular pressure;
Medical status permitting safe surgery
Not currently receiving intervention in another study
(further information of criteria explained on the NEI website)
Impact
The National Eye Institute (NEI) has clearly marked on their clinical trial study: “Placement of the iRPE patch is not expected to benefit participants’ vision in a manner that would aid reading or other fine discriminatory tasks, given the already-advanced state of vision loss of the eye. The study will lead to knowledge that will enhance understanding of autologous stem cell-based therapy for potential treatment of GA associated with AMD” (NEI).
Though a full cure is far from this point, future AMD treatment and stem cell therapy can be branched off from the results of 1/2a trials, laying a footpath for future research, medicine, and therapies.
Works Cited
Bongso, Ariff, and Eng Hin Lee, editors. Stem Cells: From Bench to Bedside. World Scientific, 2005. Google Books. Accessed 15 August 2023.
Genentech. “About AMD.” Genentech, https://www.gene.com/patients/disease-education/amd-fact-sheet. Accessed 15 August 2023.
National Eye Institute (NEI). “First U.S. patient receives autologous stem cell therapy to treat dry AMD.” National Institutes of Health (NIH), National Institutes of Health (NIH), 31 August 2022, https://www.nih.gov/news-events/news-releases/first-us-patient-receives-autologous-stem-cell-therapy-treat-dry-amd. Accessed 15 August 2023.
NEI. “Clinical Trial Highlight: Stem Cell Transplants for Dry AMD.” National Eye Institute, 1 March 2022, https://www.nei.nih.gov/learn-about-eye-health/eye-conditions-and-diseases/age-related-macular-degeneration/clinical-trial-highlight-stem-cell-transplants-dry-amd. Accessed 15 August 2023.
Ufret-Vincenty, M.D., Rafael. “Macular degeneration: Advances in stem cell and gene therapy | Aging | Eyes and Vision.” UT Southwestern Medical Center, 9 January 2023, https://utswmed.org/medblog/amd-stem-cell-gene-therapy/. Accessed 15 August 2023.
Ye, Lei et al. “Induced pluripotent stem cells and their potential for basic and clinical sciences.” Current cardiology reviews vol. 9,1 (2013): 63-72. doi:10.2174/157340313805076278.
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