The Safety and Efficacy of Stem Cell Therapies in the treatment of Macular Degeneration and Stargardt’s Macular Dystrophy

Date of Award

Spring 2020

Degree Name

Master of Medical Science (Physician Assistant)

Department

Physician Assistant; College of Health Sciences

First Advisor

Erin Wolf

Abstract

Introduction: Age related macular degeneration (AMD) and Stargardt’s macular dystrophy are conditions which affect the retinal pigment epithelium eventually leading to photoreceptor death and central vision loss. For both conditions the current mainstay of treatment is related to dietary supplements with a focus on prevention of disease progression rather than vision recovery. Currently researchers are looking into utilization of stem cell therapies in the treatment of both AMD and Stargardt’s macular dystrophy. This paper will explore the safety and efficacy of stem cell therapies as compared to the current standard of care guidelines.

Methods: A literature search was completed through Pubmed, EbscoHost, and Google Scholar in November of 2018 and through May of 2019. Seven studies were selected based on their ability to address the research question, their design, and results.

Results: Based on the studies reviewed there is evidence to say that stem cell derived RPE therapies could hold promise for photoreceptor recovery in patients with AMD and Stargardt’s macular dystrophy. Overall, six of the studies showed an improvement of visual acuity in the eye which underwent intervention. All studies addressed the concern for tumorigenicity and found that there was no evidence of malignant proliferation during the study timeframe.

Discussion: As compared to the current mainstay of treatment, all of the studies selected for review showed promise in terms of overall safety and promise for photoreceptor rescue. The studies had three overlapping measured endpoints in visual acuity, fundoscopic photography, and optic coherence tomography. However, the interventions themselves slightly varied from study to study. Five used human embryonic stem cells, one used induced pluripotent stem cells and the last fetal retinal cells. The follow-up period was also different for each of the studies with a majority of the data collection spanning 1-2 years.

Conclusion: Stem cell therapies should be studied further prior to incorporation into treatment guidelines. Studies show promise in the setting of age-related macular degeneration and Stargardt’s macular dystrophy however the data is currently limited in its generalizability. Further research should focus on standardization of the stem cell intervention and long term implication and safety. A more diverse patient population should be recruited in order to expand applicability to a broader group of individuals.

Additional Files

LS_Poster Presentation.pptx.pdf (4252 kB)
Poster

References_LS_PICO.pdf (82 kB)
References

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The Safety and Efficacy of Stem Cell Therapies in the treatment of Macular Degeneration and Stargardt’s Macular Dystrophy

Introduction: Age related macular degeneration (AMD) and Stargardt’s macular dystrophy are conditions which affect the retinal pigment epithelium eventually leading to photoreceptor death and central vision loss. For both conditions the current mainstay of treatment is related to dietary supplements with a focus on prevention of disease progression rather than vision recovery. Currently researchers are looking into utilization of stem cell therapies in the treatment of both AMD and Stargardt’s macular dystrophy. This paper will explore the safety and efficacy of stem cell therapies as compared to the current standard of care guidelines.

Methods: A literature search was completed through Pubmed, EbscoHost, and Google Scholar in November of 2018 and through May of 2019. Seven studies were selected based on their ability to address the research question, their design, and results.

Results: Based on the studies reviewed there is evidence to say that stem cell derived RPE therapies could hold promise for photoreceptor recovery in patients with AMD and Stargardt’s macular dystrophy. Overall, six of the studies showed an improvement of visual acuity in the eye which underwent intervention. All studies addressed the concern for tumorigenicity and found that there was no evidence of malignant proliferation during the study timeframe.

Discussion: As compared to the current mainstay of treatment, all of the studies selected for review showed promise in terms of overall safety and promise for photoreceptor rescue. The studies had three overlapping measured endpoints in visual acuity, fundoscopic photography, and optic coherence tomography. However, the interventions themselves slightly varied from study to study. Five used human embryonic stem cells, one used induced pluripotent stem cells and the last fetal retinal cells. The follow-up period was also different for each of the studies with a majority of the data collection spanning 1-2 years.

Conclusion: Stem cell therapies should be studied further prior to incorporation into treatment guidelines. Studies show promise in the setting of age-related macular degeneration and Stargardt’s macular dystrophy however the data is currently limited in its generalizability. Further research should focus on standardization of the stem cell intervention and long term implication and safety. A more diverse patient population should be recruited in order to expand applicability to a broader group of individuals.