What side effects are associated with this type of intervention?

Common treatments for Age-Related Macular Degeneration (AMD) include anti-VEGF injections, laser therapy, and photodynamic therapy. Anti-VEGF injections, such as ranibizumab and aflibercept, can cause side effects like eye pain, increased intraocular pressure, and inflammation. Laser therapy may lead to scarring and vision loss in treated areas. Photodynamic therapy can cause temporary vision changes and photosensitivity. For treatments similar to OpRegen, which involve human embryonic stem cell-derived retinal pigment epithelial cells, potential side effects may include immune reactions, graft rejection, and complications related to the surgical procedure, such as infection or retinal detachment.

What prior FDA approvals exist?

The FDA has approved several treatments for Age-Related Macular Degeneration (AMD), primarily focusing on anti-VEGF (vascular endothelial growth factor) therapies such as ranibizumab (Lucentis), aflibercept (Eylea), and bevacizumab (Avastin). These treatments work by inhibiting abnormal blood vessel growth and leakage in the retina. While these therapies are effective for wet AMD, OpRegen represents a novel approach for dry AMD, involving the transplantation of human embryonic stem cell-derived retinal pigment epithelial (RPE) cells to potentially engraft, survive, and moderate disease progression. This cell-based therapy aims to address the underlying cellular degeneration in AMD, offering a different mechanism of action compared to traditional anti-VEGF treatments.

What other types of research exist?

Promising novel alternatives to OpRegen for AMD patients include: 1) Gene therapy approaches such as RGX-314, which delivers a gene encoding for an anti-VEGF protein to reduce disease progression. 2) Other stem cell therapies like induced pluripotent stem cell (iPSC)-derived retinal pigment epithelial cells, which are being explored for their potential to replace damaged cells. 3) Pharmacological treatments like anti-VEGF drugs (e.g., brolucizumab) that offer improved dosing schedules and efficacy. These alternatives are currently in various stages of clinical trials and may provide viable options for AMD treatment in 2024.

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Stem Cell Therapy

OpRegen for Age-Related Macular Degeneration

Phase 1 & 2

Waitlist Available

Led By Tareq Jaouni, MD

Research Sponsored by Lineage Cell Therapeutics, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Diagnosis of dry (non-neovascular) age related macular degeneration in both eyes

Ability to undergo a vitreoretinal surgical procedure under monitored anesthesia care

Must not have

Evidence of neovascular AMD by history, as well as by clinical exam, fluorescein angiography (FA), or ocular coherence tomography (OCT) at baseline in either eye

History of retinal detachment repair in the study eye

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline, month 12

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a new treatment using special cells from human embryos to help people with a type of vision loss called dry-AMD. The treatment involves injecting these cells into the eye to see if they can survive and slow down the disease.

Who is the study for?

This trial is for individuals aged 50 or older with advanced dry-form age-related macular degeneration (AMD) in both eyes. Participants must have a central visual acuity of 20/200 or worse, be in good health to undergo surgery and follow-up procedures, and not have any significant eye diseases other than AMD. They cannot have active infections like TB, HIV, hepatitis B/C, CMV IgM, EBV IgM or a history of certain cancers unless in remission for over five years.

What is being tested?

The study tests the safety and effectiveness of OpRegen - cells derived from human embryonic stem cells intended to replace damaged retinal pigment epithelial (RPE) cells. It aims to see if these transplanted cells can integrate into the retina, survive long-term and slow down disease progression in patients with AMD.

What are the potential side effects?

While specific side effects are not listed here, potential risks may include those associated with vitreoretinal surgery under anesthesia such as infection or bleeding inside the eye. There could also be immune reactions since OpRegen involves transplanting foreign biological material.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have been diagnosed with dry age-related macular degeneration in both eyes.

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I can have eye surgery with anesthesia monitoring.

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I have advanced dry age-related macular degeneration with significant vision loss.

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I am 50 years old or older.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have been diagnosed with wet AMD in at least one eye.

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I have had surgery to fix a detached retina in one eye.

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My eye condition is only due to AMD, not other causes like myopia or histoplasmosis.

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I cannot receive systemic immunosuppression.

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I do not have significant eye diseases other than AMD that could affect my vision.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline, month 12

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline, month 12

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, month 12 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Percentage of Participants With Treatment Emergent Adverse Events

Secondary study objectives

Change From Baseline in Geographic Atrophy (GA) Lesion Area

Change From Baseline in National Eye Institute Visual Function Questionnaire-25 (NEI VFQ-25) Quality of Life

Change From Baseline in Visual Acuity

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: OpRegenExperimental Treatment1 Intervention

Up to 12 legally blind subjects with best corrected visual acuity of 20/200 or less in first three cohorts and 12 subjects with best corrected visual acuity of 20/64 and 20/250 in fourth cohort

0 / 9Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Age-Related Macular Degeneration (AMD) include anti-VEGF (vascular endothelial growth factor) injections, photodynamic therapy, and stem cell-based therapies like OpRegen. Anti-VEGF injections work by inhibiting the growth of abnormal blood vessels that can leak and cause vision loss. Photodynamic therapy uses light-activated drugs to destroy these abnormal vessels. Stem cell-based therapies, such as OpRegen, involve transplanting human embryonic stem cell-derived retinal pigment epithelial (RPE) cells to replace damaged RPE cells, aiming to restore and maintain retinal function. These treatments are crucial for AMD patients as they target the underlying causes of vision loss, potentially slowing disease progression and preserving vision.