Stem Cell Therapy for Age-Related Macular Degeneration

Recruiting at 1 trial location

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Astellas Institute for Regenerative Medicine

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

Duration: Not specified

36 Participants Needed

Trial Summary

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications.

What data supports the effectiveness of the treatment Human Embryonic Stem Cell-Derived Retinal Pigment Epithelial Cells for Age-Related Macular Degeneration?

Research shows that human embryonic stem cell-derived retinal pigment epithelial cells can improve visual function in animal models of retinal disease and have been used safely in early-stage Stargardt macular degeneration, suggesting potential benefits for age-related macular degeneration. ^{1 2 3 4 5}

Is stem cell therapy for macular degeneration safe?

Research shows that using human embryonic stem cell-derived retinal pigment epithelial cells for macular degeneration appears generally safe, with no major safety issues like tumor formation or severe inflammation reported in early studies. ^{2 5 6 7 8}

How is the treatment using human embryonic stem cell-derived retinal pigment epithelial cells different from other treatments for age-related macular degeneration?

This treatment is unique because it uses cells derived from human embryonic stem cells to replace damaged retinal pigment epithelial cells, potentially restoring vision by forming functional layers in the eye. Unlike other treatments, it aims to directly repair the underlying cell damage in the retina. ^{1 2 3 5 9}

What is the purpose of this trial?

The purpose of this study is to is to evaluate the occurrence of late onset (i.e., greater than 5 years after treatment) adverse events of special interest (AESI) in participants who have received sub-retinal transplant of human embryonic stem cell derived - retinal pigment epithelial (hESC-RPE) cells in an AIRM-sponsored clinical trial. The events of special interest are adverse events (AEs) that are presumed to have a potential causal relationship to the hESC-RPE cells.

Research Team

Medical Monitor

Principal Investigator

Astellas Institute for Regenerative Medicine

Eligibility Criteria

This study is for people who have previously received a specific stem cell treatment for macular degeneration in an AIRM-sponsored trial. Participants must be able to understand the nature of this follow-up study.

Inclusion Criteria

I have received hESC-RPE cell therapy for macular degeneration in an AIRM trial.

Participant is able to understand

Exclusion Criteria

There are no exclusion criteria

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants received sub-retinal transplant of human embryonic stem cell derived - retinal pigment epithelial (hESC-RPE) cells in a previous AIRM-sponsored clinical trial

Not specified

Long-term Follow-up

Participants are monitored for late onset adverse events of special interest (AESI) through annual questionnaires

10 years

Annual questionnaire (web-based or telephone)

Life-long Follow-up (UK participants)

UK participants are monitored for adverse events of special interest (AESI) for life

Treatment Details

Interventions

• Human Embryonic Stem Cell-Derived Retinal Pigment Epithelial Cells

Trial Overview The focus is on monitoring safety long-term, specifically looking for late onset adverse events that might be related to the stem cell therapy they received over 5 years ago.

Participant Groups

Group I: hESC-RPE cells Experimental Treatment 1 Intervention

Participants previously enrolled into an Astellas Institute for Regenerative Medicine (AIRM) sponsored clinical trial and treated with Human Embryonic Stem Cell-Derived Retinal Pigment Epithelial (hESC-RPE) cells

Who Is Running the Clinical Trial?

Astellas Institute for Regenerative Medicine

Lead Sponsor

Trials

9

Recruited

150+

Findings from Research

Human embryonic stem cell-derived retinal pigment epithelium (hESC-RPE) cells have shown the ability to inhibit T cell responses, suggesting they possess immunomodulatory properties that could be beneficial for transplantation.

In experiments with RCS rats, hESC-RPE cells survived without the need for immunosuppression, provided retinal rescue, and increased levels of the anti-inflammatory cytokine IL-10, indicating their potential for safe allotransplantation in treating age-related macular degeneration.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Immunological Properties of Human Embryonic Stem Cell-Derived Retinal Pigment Epithelial Cells. Idelson, M., Alper, R., Obolensky, A., et al.[2019]

Researchers successfully generated retinal pigment epithelium (RPE) from 18 different human embryonic stem cell lines, demonstrating a reproducible method for creating replacement tissue for retinal diseases.

In an animal model of retinal degeneration, RPE derived from these stem cells improved visual performance by 100% compared to untreated controls, showing significant potential for treating conditions like macular degeneration without causing harmful side effects.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Human embryonic stem cell-derived cells rescue visual function in dystrophic RCS rats. Lund, RD., Wang, S., Klimanskaya, I., et al.[2022]

Human embryonic stem cell-derived retinal pigment epithelium (hESC-RPE) showed long-term functional rescue in animal models of retinal degeneration, sustaining visual function for over 220 days without causing teratoma formation.

In a Good Laboratory Practice-compliant study, hESC-RPE transplantation in immune-deficient mice demonstrated no evidence of tumor formation, indicating a promising safety profile for potential clinical applications in treating retinal diseases.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Long-term safety and function of RPE from human embryonic stem cells in preclinical models of macular degeneration. Lu, B., Malcuit, C., Wang, S., et al.[2022]

The engineered RPE patch, derived from human embryonic stem cells, was successfully implanted in two patients with severe age-related macular degeneration (AMD), showing promising results in visual acuity improvement of 29 and 21 letters over 12 months.

The procedure demonstrated safety with only local immunosuppression required long-term, supporting the potential of hESC-RPE patch transplantation as a regenerative treatment for AMD.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Phase 1 clinical study of an embryonic stem cell-derived retinal pigment epithelium patch in age-related macular degeneration. da Cruz, L., Fynes, K., Georgiadis, O., et al.[2022]

Two phase I/II studies involving 18 patients with advanced macular degeneration showed that implanting human embryonic stem cell-derived retinal pigment epithelium (hESC-RPE) is safe and may improve visual acuity in over half of the treated patients.

No significant adverse events related to the cell therapy were observed, aside from one case of postoperative infection, suggesting a favorable safety profile for future studies and potential treatments for other retinal diseases.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Subretinal Transplantation of Embryonic Stem Cell-Derived Retinal Pigment Epithelium for the Treatment of Macular Degeneration: An Assessment at 4 Years. Schwartz, SD., Tan, G., Hosseini, H., et al.[2022]

References

1. United States pubmed.ncbi.nlm.nih.gov

Integration of Subretinal Suspension Transplants of Human Embryonic Stem Cell-Derived Retinal Pigment Epithelial Cells in a Large-Eyed Model of Geographic Atrophy. [2018]

2. England pubmed.ncbi.nlm.nih.gov

A phase I clinical trial of human embryonic stem cell-derived retinal pigment epithelial cells for early-stage Stargardt macular degeneration: 5-years' follow-up. [2021]

3. United States pubmed.ncbi.nlm.nih.gov

Effect of Human Central Nervous System Stem Cell Subretinal Transplantation on Progression of Geographic Atrophy Secondary to Nonneovascular Age-Related Macular Degeneration. [2021]

4. United States pubmed.ncbi.nlm.nih.gov

Immunological Properties of Human Embryonic Stem Cell-Derived Retinal Pigment Epithelial Cells. [2019]

5. United States pubmed.ncbi.nlm.nih.gov

Human embryonic stem cell-derived cells rescue visual function in dystrophic RCS rats. [2022]

6. England pubmed.ncbi.nlm.nih.gov

Long-term safety and function of RPE from human embryonic stem cells in preclinical models of macular degeneration. [2022]

7. United States pubmed.ncbi.nlm.nih.gov

Phase 1 clinical study of an embryonic stem cell-derived retinal pigment epithelium patch in age-related macular degeneration. [2022]

8. United States pubmed.ncbi.nlm.nih.gov

Subretinal Transplantation of Embryonic Stem Cell-Derived Retinal Pigment Epithelium for the Treatment of Macular Degeneration: An Assessment at 4 Years. [2022]

9. United States pubmed.ncbi.nlm.nih.gov

Comparison of FRPE and human embryonic stem cell-derived RPE behavior on aged human Bruch's membrane. [2022]