Stem Cell Therapy for Age-Related Macular Degeneration

Enrolling by invitation 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial examines the long-term safety of a new treatment for age-related macular degeneration, a common eye condition that can cause vision loss. The treatment uses special cells, called hESC-RPE cells (human embryonic stem cell-derived retinal pigment epithelial cells), to potentially improve sight. Researchers aim to identify any serious side effects that might appear more than five years after treatment. This trial includes individuals who previously received this stem cell therapy in a past study for macular degeneration and can understand the trial's purpose. As a Phase 1 and Phase 2 trial, the research focuses on understanding how the treatment works in people and measuring its effectiveness in an initial, smaller group.

Will I have to stop taking my current medications?

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

Is there any evidence suggesting that this treatment is likely to be safe for humans?

Research has shown that cells made from human embryonic stem cells, known as retinal pigment epithelial (hESC-RPE) cells, are generally well-tolerated. One study found these cells remained safe for up to 37 months after transplantation into individuals with age-related macular degeneration (AMD). Another study suggested these cells could be a promising treatment for retinal diseases like AMD, with no major safety concerns reported.

While these results are encouraging, this research remains in the early stages. The initial phases focus on assessing safety and treatment tolerance, so any serious issues would likely have emerged by now. However, more research is needed to fully understand long-term safety, especially beyond five years after treatment. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Researchers are excited about using Human Embryonic Stem Cell-Derived Retinal Pigment Epithelial (hESC-RPE) cells for treating age-related macular degeneration (AMD) because they offer a fresh approach that could potentially restore vision. Unlike traditional treatments like anti-VEGF injections, which mainly aim to slow vision loss, hESC-RPE cells could replace damaged cells in the retina and possibly improve vision. This treatment works by directly targeting and repairing retinal damage, offering hope for more than just stabilizing the condition. The potential to regenerate damaged tissue is a significant advancement, setting it apart from current therapies that manage symptoms rather than offer a path to recovery.

What evidence suggests that this treatment might be an effective treatment for age-related macular degeneration?

Research has shown that retinal pigment epithelial (RPE) cells, derived from human embryonic stem cells, might help treat age-related macular degeneration (AMD). These cells can replace damaged retinal cells, which are crucial for vision. The damage and loss of these retinal cells significantly contribute to the progression of AMD. Early studies suggest that transplanting RPE cells may improve vision by restoring the function of these vital cells. Although more research is necessary, initial results offer promise for individuals with AMD. Participants in this trial will receive Human Embryonic Stem Cell-Derived Retinal Pigment Epithelial Cells to assess their effectiveness in treating AMD. ^{1 2 5 6 7}

Who Is on the Research Team?

Medical Monitor

Principal Investigator

Astellas Institute for Regenerative Medicine

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Published Research Related to This Trial

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]

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]

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]

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]

Citations

1. nature.com nature.com/articles/s41421-018-0053-y

Human embryonic stem cell-derived retinal pigment ... Human embryonic stem cell-derived retinal pigment epithelium transplants as a potential treatment for wet age-related macular degeneration.

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC6412543/

Human Embryonic Stem Cell-Derived Retinal Pigment ... The dysfunction and death of RPE cells significantly contribute to the progression of age-related macular degeneration (AMD), which is the leading cause of ...

3. sciencedirect.com sciencedirect.com/science/article/pii/S0161642018300241

Transplantation of Human Embryonic Stem Cell-Derived ... Transplantation of human embryonic stem cell (hESC)-derived retinal pigment epithelial (RPE) cells offers the potential for benefit in macular degeneration.

4. clinicaltrials.gov clinicaltrials.gov/study/NCT02286089

NCT02286089 | Safety and Efficacy Study of OpRegen for ... The main objective of the study is evaluation of the safety and tolerability of OpRegen - Human embryonic stem cell-derived retinal pigment epithelial (RPE) ...

5. cell.com cell.com/cell-stem-cell/fulltext/S1934-5909(25)00304-2

Safety and tolerability of RPESC-RPE transplantation in ... Stem cell-based cell replacement therapies are under development to implant RPE cells into areas of RPE atrophy to improve vision for dry AMD ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT01469832

Study Details | NCT01469832 | Safety and Tolerability of ... The purpose of this study is to evaluate the safety and tolerability of RPE cellular therapy in patients with SMD.

7. sciencedirect.com sciencedirect.com/science/article/abs/pii/S0140673614613763

Human embryonic stem cell-derived retinal pigment ... Our results show that hESC-derived cells were well tolerated for up to 37 months after transplantation in individuals with atrophic age-related macular ...