Stem Cell Therapy for Dry Age-Related Macular Degeneration

Overseen By Jeffrey H Stern, M.D., Ph.D.

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Luxa Biotechnology, LLC

Must not be taking: Chemotherapy, Immunosuppressants

Disqualifiers: Active malignancy, Toxoplasmosis, others

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

Compensation: Varies

Number of Visits: Unknown

Duration: Single dose administration

18 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial is testing a new treatment where cells from donor eyes are transplanted into patients with dry AMD to see if it is safe and can help improve their vision. Stem cell transplantation may improve visual acuity in patients with dry age-related macular degeneration.

Will I have to stop taking my current medications?

The trial information does not specify if you need to stop taking your current medications. However, if you are on systemic immunosuppressant therapy, you must not have taken it in the past six months to participate.

What data supports the effectiveness of the treatment RPESC-RPE-4W for dry age-related macular degeneration?

Research shows that stem cell-derived retinal pigment epithelial (RPE) cells, similar to those in RPESC-RPE-4W, have been successfully transplanted in patients with age-related macular degeneration, maintaining vision and being well tolerated. Additionally, clinical trials have shown promising outcomes in restoring visual function by transplanting these cells into the eye. ^{1 2 3 4 5}

Is stem cell therapy for dry age-related macular degeneration safe?

Research shows that stem cell therapy using retinal pigment epithelial cells derived from embryonic stem cells has been generally safe in early trials, with no serious adverse effects reported in humans, except for one case of infection after surgery. Animal studies also found no obvious negative reactions, suggesting the treatment is well-tolerated. ^{1 4 6 7 8}

How is the treatment RPESC-RPE-4W different from other treatments for dry age-related macular degeneration?

RPESC-RPE-4W is unique because it involves using stem cells to generate retinal pigment epithelium (RPE) cells, which are then transplanted into the eye to potentially restore vision in patients with dry age-related macular degeneration, a condition with no effective treatments currently available. ^{1 2 3 5 9}

Research Team

David S Liao, MD, PhD

Principal Investigator

Retina-Vitreous Associates Medical Group

Theodore Leng, MD, FACS

Principal Investigator

Spencer Center for Vision Research at Stanford University

Rajesh C Rao, M.D.

Principal Investigator

University of Michigan Kellogg Eye Center

Eligibility Criteria

This trial is for adults over 55 with dry AMD who can consent and are healthy enough for eye surgery. Men must use contraception, women should be postmenopausal or unable to have children. Excluded are those with major health issues, recent immunosuppressants or clinical trials, certain infections, pregnancy, nursing, or specific allergies.

Inclusion Criteria

I understand the study and agree to participate.

I am medically fit for eye surgery (>60% Karnofsky).

I am postmenopausal or my female partner cannot get pregnant.

Exclusion Criteria

I do not have major health issues that would prevent me from joining the study.

Any other medical condition, which, in the Investigator's judgment, will interfere with the subject's ability to comply with the protocol, compromises subject safety, or interferes with the interpretation of the study results

Pregnant or nursing females

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a single dose of RPESC-RPE-4W cells in the eye, with doses varying from 50,000 to 250,000 cells

Single dose administration

Follow-up

Participants are monitored for safety and effectiveness after treatment, including visual acuity and structural changes

24 months

Treatment Details

Interventions

• RPESC-RPE-4W

Trial Overview The study tests the safety and tolerability of a stem cell-derived treatment called RPESC-RPE-4W in patients with dry age-related macular degeneration (AMD). It involves vitrectomy and subretinal injection procedures to deliver the therapy.

Participant Groups

Group I: 50,000 cells Experimental Treatment 1 Intervention

Six patients will receive single dose of 50,000 RPESC-RPE-4W cells in the eye.

Group II: 250,000 cells Experimental Treatment 1 Intervention

Six patients will receive single dose of 250,000 RPESC-RPE-4W cells in the eye.

Group III: 150,000 cells Experimental Treatment 1 Intervention

Six patients will receive single dose of 150,000 RPESC-RPE-4W cells in the eye.

Who Is Running the Clinical Trial?

Luxa Biotechnology, LLC

Lead Sponsor

Trials

1

Recruited

20+

National Institutes of Health (NIH)

Collaborator

Trials

2,896

Recruited

8,053,000+

National Eye Institute (NEI)

Collaborator

Trials

572

Recruited

1,320,000+

Regenerative Research Foundation

Collaborator

Trials

1

Recruited

20+

Findings from Research

Recent advances in stem cell research have shown that retinal pigment epithelium (RPE) cells can be generated from various stem cell types, offering a potential treatment avenue for dry age-related macular degeneration (AMD), which currently has no effective therapies.

Clinical trials approved by the FDA are underway to evaluate the effectiveness of stem cell-based RPE transplantation for dry AMD, with some early patients showing promising visual recovery outcomes.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Stem cell-based therapies for age-related macular degeneration: current status and prospects. Mu, Y., Zhao, M., Su, G.[2020]

Transplanted retinal pigment epithelium (RPE) cells derived from induced pluripotent stem cells successfully maintained vision in a patient with age-related macular degeneration, indicating potential for this treatment in restoring sight.

The RPE cell transplant was well tolerated by the patient, suggesting a favorable safety profile for this innovative approach in treating retinal diseases.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

What's old is new again: Autologous stem cell transplant for AMD. Byrne, LC.[2018]

Recent advances in cell biology have made it possible to generate retinal pigment epithelial (RPE) cells from various sources, which could be used to treat dry age-related macular degeneration (AMD), a condition currently lacking effective treatments.

Clinical trials for RPE transplantation have shown promising results in restoring visual function, but challenges such as implantation techniques and immune rejection still need to be addressed for successful treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Stem cell therapies for age-related macular degeneration: the past, present, and future. Dang, Y., Zhang, C., Zhu, Y.[2018]

Transplanted stem cell-derived retinal pigment epithelial (RPE) cells from the CR-4 embryonic stem line were well tolerated in a study involving 36 immunosuppressed mice, showing no obvious adverse reactions.

This research suggests that CR-4 RPE cell transplantation could be a promising therapeutic approach for age-related macular degeneration (AMD), a leading cause of vision loss, as it demonstrates safety in murine models of retinal degeneration.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A Preclinical Safety Study of Human Embryonic Stem Cell-Derived Retinal Pigment Epithelial Cells for Macular Degeneration. Mazzilli, JL., Snook, JD., Simmons, K., et al.[2021]

RPE transplantation using stem cells shows promise as a potential treatment for age-related macular degeneration (AMD) and Stargardt's macular dystrophy (SMD), both of which involve loss of retinal pigment epithelium (RPE).

Recent Phase 1/2 trials have demonstrated the safety of transplanting human embryonic stem cell-derived RPE into patients with AMD and SMD, marking significant progress towards effective treatments for these conditions.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Stem Cell-Based Therapy for Diseases of the Retinal Pigment Epithelium: From Bench to Bedside. Sachdeva, MM., Eliott, D.[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]

Human pluripotent stem cell-derived retinal pigmented epithelial (RPE) cells show promise as a potential treatment for untreatable blinding diseases caused by RPE cell degeneration.

Initial safety trials of embryonic stem cell-derived RPE transplants reported no serious adverse effects, suggesting that these treatments are safe and warrant further research.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

ESC-derived retinal pigmented epithelial cell transplants in patients: so far, so good. Sowden, JC.[2022]

Age-related macular degeneration (AMD) is a leading cause of blindness in the elderly, with no current treatment for advanced non-neovascular AMD (NNV-AMD), highlighting the urgent need for effective therapies.

Research into using human embryonic stem cell-derived retinal pigment epithelium (hESC-RPE) and induced pluripotent stem cell-derived RPE (iPSC-RPE) shows promise in restoring vision by replacing damaged RPE cells, but significant ethical and safety challenges remain before these therapies can be widely implemented.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Stem cell based therapies for age-related macular degeneration: The promises and the challenges. Nazari, H., Zhang, L., Zhu, D., et al.[2022]

Transplanting human neural stem cells (HuCNS-SC) into the subretinal space significantly increased the proliferation of host retinal pigment epithelial (RPE) cells in dystrophic rat models, showing about a threefold increase in cell proliferation compared to controls.

This study suggests that enhancing the natural regenerative capacity of RPE cells through HuCNS-SC transplantation could be a promising new approach for treating age-related macular degeneration and retinitis pigmentosa.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Subretinal Transplantation of Human Central Nervous System Stem Cells Stimulates Controlled Proliferation of Endogenous Retinal Pigment Epithelium. McGill, TJ., Osborne, L., Lu, B., et al.[2022]

References

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

Stem cell-based therapies for age-related macular degeneration: current status and prospects. [2020]

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

What's old is new again: Autologous stem cell transplant for AMD. [2018]

3. New Zealand pubmed.ncbi.nlm.nih.gov

Stem cell therapies for age-related macular degeneration: the past, present, and future. [2018]

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

A Preclinical Safety Study of Human Embryonic Stem Cell-Derived Retinal Pigment Epithelial Cells for Macular Degeneration. [2021]

5. England pubmed.ncbi.nlm.nih.gov

Stem Cell-Based Therapy for Diseases of the Retinal Pigment Epithelium: From Bench to Bedside. [2022]

6. 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]

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

ESC-derived retinal pigmented epithelial cell transplants in patients: so far, so good. [2022]

8. England pubmed.ncbi.nlm.nih.gov

Stem cell based therapies for age-related macular degeneration: The promises and the challenges. [2022]

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

Subretinal Transplantation of Human Central Nervous System Stem Cells Stimulates Controlled Proliferation of Endogenous Retinal Pigment Epithelium. [2022]