Blindness Fact Sheet

CIRM funds many projects seeking to better understand diseases of blindness and to translate those discoveries into new therapies.

Description

Over a million Americans are legally blind, with another 3.2 million suffering significant visual impairment1. While there are several causes of blindness, the leading cause of all visual impairment is age-related macular degeneration, which affects 1.7 million Americans. 

California’s stem cell agency funds research into potential therapies for three of the causes of blindness. All the research teams are seeking to use various forms of stem cells to rescue or replace cells in the eye damaged or threatened by the diseases. Several groups are working on ways to restore vision for people with age-related macular degeneration (AMD). Other projects are looking to preserve vision in patients with retinitis pigmentosa (RP), and to restore clarity to the surface of eyes impacted by corneal disease.

Macular Degeneration

AMD affects 8 million Americans and accounts for over 50% of vision loss cases in white Americans. In AMD, the layer of cells that support the photoreceptors is destroyed. Without this support system, the photoreceptors, the cells that actually allow us to sense light start to malfunction. CIRM-funded teams are looking at various methods of replacing this layer of support cells called RPE (retinal pigment epithelial) cells. Some are using embryonic stem cells as a starting point to generate new RPE cells. Others are using stem cells obtained by reprogramming adult cells to be like embryonic cells, which could potentially come from the patients’ themselves.

Retinitis Pigmentosa

Retinitis pigmentosa (RP) is an inherited and progressive vision loss disease that has an incidence of 1:4000 and leaves most patients legally blind by mid-life. RP destroys the light-sensing photoreceptors in the retina. CIRM-funded researchers are seeking to use stem cells to rescue these photoreceptors from further damage and potentially replace them with new ones.

Limbal Stem Cell Deficiency

The cornea, the outer surface of the eye, is constantly refreshed by stem cells that reside in neighboring tissue. But some people just don’t have enough of these stem cells, called limbal stem cells, to make enough new cornea cells. CIRM-funded researchers are trying to correct this condition, limbal stem cell deficiency, by retrieving the few existing limbal stem cells, and using various techniques to expand them in the laboratory until there are enough cells to rebuild a healthy cornea.

Clinical Stage Programs

University of Southern California

This team is using embryonic stem cells to produce the support cells, or RPE cells, needed to replace those lost in AMD. Because these cells exist in a thin sheet in the back of the eye, they are assembling these sheets in the lab by growing the RPE cells on synthetic scaffolds. These sheets are then surgically implanted into the eye. They are testing the human embryonic stem cell-derived RPE cells in a Phase 1/2a clinical trial to treat the advanced dry form of AMD. 

University of California, Irvine

For retinitis pigmentosa, the team is using donor tissue to isolate cells that are part way down the path from neural stem cells to adult eye tissue. These retinal progenitor cells are grown in large quantities in the lab and then injected into the eye. The team suggests the cells could help in two ways. They may be able to protect the photoreceptors not yet damaged by the disease, and they may be able to form new photoreceptors to replace those already lost. The team tested the safety of transplanting human retinal progenitor cells into patients with RP in a phase 1/2 clinical trial that is now completed. CIRM is now funding a Phase 2 trial, sponsored by a jCyte, that is testing this treatment in a larger group of RP patients (see trial below).

jCyte

The same team from UC Irvine is now conducting a Phase 2b clinical trial for retinitis pigmentosa using the same stem cell derived retinal progenitor cell therapy. The trial, which is sponsored by the company jCyte, will test the treatment in a larger patient population to determine whether the treatment is effective at restoring some vision. After finishing patient enrollment, the team will conduct patient follow up studies and collect of all clinical outcome measures.

CIRM Grants Targeting Vision Loss

Researcher name Institution Grant Title Grant Type Award Amount
Sophie Deng University of California, Los Angeles Regeneration of a Normal Corneal Surface by Limbal Stem Cell Therapy Late Stage Preclinical Projects $4,244,211
Martin Friedlander Scripps Research Institute Autologous Retinal Pigmented Epithelial Cells Derived from Induced Pluripotent Stem Cells for the Treatment of Atrophic Age Related Macular Degeneration Early Translational I $5,806,321
Henry Klassen jCyte, Inc Phase 2b Clinical Study of Safety and Efficacy of Intravitreal Injection of Retinal Progenitor Cells (jCell) for Treatment of Retinitis Pigmentosa Clinical Trial Stage Projects $8,295,750
Gabriel Travis University of California, Los Angeles Development of a Stem Cell-based Transplantation Strategy for Treating Age-related Macular Degeneration Early Translational I $5,487,136
Peter Coffey University of California, Santa Barbara Development of Cellular Therapies for Retinal Disease Research Leadership $4,690,963
Biju Thomas University of Southern California A Novel Tissue Engineering Technique to Repair Degenerated Retina Inception - Discovery Stage Research Projects $215,133
Henry Klassen University of California, Irvine Retinal progenitor cells for treatment of retinitis pigmentosa Disease Team Therapy Development - Research $17,144,825
Karl Wahlin University of California, San Diego An IPSC cell based model of macular degeneration for drug discovery. Inception - Discovery Stage Research Projects $232,200
Deepak Lamba Buck Institute for Age Research 3D Modeling of Retina using Polymer Scaffolds for Understanding Disease Pathogenesis Basic Biology IV $1,212,553
Magdalene Seiler University of California, Irvine Morphological and functional integration of stem cell derived retina organoid sheets into degenerating retina models Therapeutic Translational Research Projects $4,769,039
Kang Zhang University of California, San Diego Generation of fibroblast cell lines in patients with common blinding eye diseases Tissue Collection for Disease Modeling $1,034,425
Jonathan Lin University of California, San Diego Small Molecule Proteostasis Regulators to Treat Photoreceptor Diseases Quest - Discovery Stage Research Projects $1,160,648
Thomas Novak Cellular Dynamics International Generation and characterization of high-quality, footprint-free human induced pluripotent stem cell lines from 3,000 donors to investigate multigenic diseases hiPSC Derivation $16,000,000
Steven Schwartz University of California, Los Angeles Clinical Translation of Autologous Regenerative Cell Therapy for Blindness Therapeutic Translational Research Projects $5,068,026
Deborah Requesens Coriell Institute for Medical Research The CIRM Human Pluripotent Stem Cell Biorepository – A Resource for Safe Storage and Distribution of High Quality iPSCs hPSC Repository $9,942,175
Theodore Leng Stanford University NeuBright, a purified allogeneic cell therapy product for treatment of Dry Age-related Macular Degeneration Therapeutic Translational Research Projects $4,238,532
Magdalene Seiler University of California, Irvine Restoring vision by sheet transplants of retinal progenitors and retinal pigment epithelium (RPE) derived from human embryonic stem cells (hESCs) Early Translational IV $3,998,948
Mark Humayun University of Southern California Phase 1 Safety Assessment of CPCB-RPE1, hESC-derived RPE Cell Coated Parylene Membrane Implants, in Patients with Advanced Dry Age Related Macular Degeneration Disease Team Therapy Development III $17,128,661
Mark Humayun University of Southern California Stem cell based treatment strategy for Age-related Macular Degeneration (AMD) Disease Team Planning $3,088
Sophie Deng University of California, Los Angeles Regeneration of Functional Human Corneal Epithelial Progenitor Cells Early Translational II $697,507
David Schaffer University of California, Berkeley Engineered Biomaterials for Scalable Manufacturing and High Viability Implantation of hPSC-Derived Cells to Treat Neurodegenerative Disease Tools and Technologies III $1,239,276
Sophie Deng University of California, Los Angeles Regeneration of Functional Human Corneal Epithelial Progenitor Cells Early Translational II $1,524,947
Shaomei Wang Cedars-Sinai Medical Center IND-enabling study of subretinal delivery of human neural progenitor cells for the treatment of retinitis pigmentosa Late Stage Preclinical Projects $4,954,514
Henry Klassen University of California, Irvine Human retinal progenitor cells as candidate therapy for retinitis pigmentosa Early Translational II $1,803,768
Karl Wahlin University of California, San Diego Microenvironment based optimization of retinal induction using CRISPR-CAS9 reporter pluripotent stem cells as an expandable source of retinal progenitors and photoreceptors. Inception - Discovery Stage Research Projects $232,200
Mark Humayun University of Southern California Stem cell based treatment strategy for Age-related Macular Degeneration (AMD) Disease Team Research I $18,904,916
Jeffrey Goldberg Stanford University Embryonic Stem Cells for Corneal Endothelial Degeneration Inception - Discovery Stage Research Projects $235,836
David Hinton University of Southern California Therapeutic potential of Retinal Pigment Epithelial cell lines derived from hES cells for retinal degeneration. SEED Grant $651,607
Total:
$140,917,205.00

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