Stargardt disease and Macular Dystrophy are uncommon but life-threatening inherited eye diseases that mainly involve the macula — the central region of the retina used for clear, straight-ahead vision. Globally, although awareness of the diseases is still poor, the incidence is increasing incrementally due to better diagnosis and genetic testing. Usually, the patients’ color vision is impaired, their vision deteriorates, and they struggle to read. Though they manifest at varying rates, macular dystrophies including best disease also exhibit comparable symptoms.
The Potential of Stem Cell Therapy
RPE cells are one such stem cells which have not yet developed into functional cells. The primary motive behind the use of stem cell therapy for Stargardt’s disease is to substitute healthy RPE cells made from cultivated stem cells for defective RPE cells. This would help in improving some of the lost function or at least arrest the worsening of vision.
Two primary kinds of stem cells are employed in studies:
Embryonic Stem Cells (ESCs): The advantage of using these cells is that, these cells can become any cell type in the body and they are derived from early-stage embryos.
Induced Pluripotent Stem Cells (iPSCs): Created by recalibrating adult cells to behave like embryonic stem cells. It also helps in reducing the risk of immune rejection.
Research and Development
Advanced Cell Technology which is now owned by Astellas Pharma tested the most promising study. In this study, scientists grafted ESC-derived RPE cells into Stargardt’s disease patients. The best part of this study was that, the implanted cells survived, integrated into the retina, and in some instances, even enhanced visual acuity.
Another key benefit was the safety profile—none of the patients suffered from severe side effects, including tumor formation or immune rejection. These initial results have opened the door to further trials worldwide, with larger patient groups and longer follow-up times.
Challenges to Overcome
Despite the promising advancements, there are a few challenges to be overcome before stem cell therapy is a standard treatment for Stargardt’s disease:
- Long-term Safety: Ongoing observation is necessary to confirm that transplanted cells do not grow out of control or form tumors.
- Scalability and Cost: Stem cell production for the purpose of treatment is still costly and time-intensive.
- Effectiveness in Late-Stage Disease: In advanced stages where the majority of photoreceptor cells are lost, restoration of vision by replacing RPE cells alone might be insufficient. Combined treatments could become necessary in the future.
Looking Ahead: A Hopeful Future
Stem cell research is changing at an extraordinary pace. Stargardt treatment helps in using modern approaches in most dynamic manner, it has helped in converging regenerative medicine, gene editing, high-tech imaging and other retinal degenerative diseases.
Conclusion
Stem cell treatment provides a strong and promising commitment towards the treatment of Stargardt’s disease way forward. How we identify and treat hereditary retinal disease is drastically different from the ability to regenerate retinal cells and restore sight, even partially.