I realized that I’ve put posted more than 20 articles on the subject of using stem cells in ophthalmology, and thought interested readers might like to see a compilation in one place, with links to the full writeups, so here it is.
Menu 21 is a similar list with all of my gene therapy in ophthalmology articles.
Stem Cells
A Primer on the Use of Stem Cells in Ophthalmology
The what, who, and why of stem cells in ophthalmology.
What stem cells are all about, who’s involved, and what diseases of the eye are being treated.
Stem Cells in Ophthalmology -- An Update: AstraZeneca Joins the Fray
AstraZeneca and University College London (UCL) have announced a research partnership, to develop medicines that use stem cells to repair damaged eyesight in people with diabetes.
Stem Cells in Ophthalmology Update 2: ACT Gets Go-ahead to Treat Stargardt’s
Advanced Cell Technology’s request to begin treating patients with Stargardt’s disease was approved by the FDA. This will be the second trial using human embryonic stem cells. The first being granted to Geron to treat spinal disease.
A multi-center trial will involve twelve patients. If the results are positive, ACT anticipates filing for an IND to treat age-related macular degeneration, as well as filing to begin clinical trials in Europe in the very near future.
Stem Cells in Ophthalmology Update 3: ACT Files IND to Treat Dry AMD
Furthering its lead in stem cell research in ophthalmology, Advanced Cell Technology Inc., announced today that it had filed an Investigational New Drug (IND) application with the U.S. Food and Drug Administration, to initiate a Phase I/II multicenter study for the treatment of dry Age-Related Macular Degeneration (dry AMD) using human embryonic stem cell (hESC) derived retinal pigment epithelial (RPE) cells.
Stem Cells in Ophthalmology Update 4: ACT Receives FDA Approval to Use hESCs to Treat Dry AMD
Advanced Cell Technology Inc., announced today that it had received approval from the FDA to commence its clinical trial using retinal pigment epithelial (RPE) cells derived from human embryonic stem cells (hESCs) to treat the dry form of age-related macular degeneration. ACT is now permitted to initiate a Phase I/II multicenter clinical trial to treat patients with dry AMD, the most common form of macular degeneration in the world. There are currently no approved treatments available for this prevalent disease of an aging global population. Dry AMD, representing a substantial global market opportunity and afflicts between 10-15 million Americans, and a further 10 million Europeans.
Stem Cells in Ophthalmology Update 5: Gene Defects Common in Induced Stem Cells
As the senior editor, John Gever, of MedPage Today reported, following the publication of three studies about induced pluripotent stem cells in the March 3rd, issue of Nature, “The road to regenerative medicine based on induced pluripotent stem cells (iPSCs) may have developed a giant pothole, with new studies showing that the cells are prone to several types of genetic defects.”
Stem Cells in Ophthalmology Update 6: Stemedica Paper Accepted for Presentation at ARVO
A safety study on the use of stem cells in the eye, in a clinical study underway at the Fyodorov Federal Institution of Eye Microsurgery in Moscow, to treat diabetic retinopathy and diabetic optical neuropathy with stem cells derived from bone marrow, has been accepted for presentation as a poster at the upcoming ARVO (2011) Annual Meeting in Fort Lauderdale at the beginning of May. An abstract is shown below.
Stem Cells in Ophthalmology Update 7: Research Studies with Induced Pluripotent Stem Cells Suggest Opposite Results
Two research studies were published this week, about the use of induced pluripotent stem cells (iPSCs) in treating retinal problems, but with opposing results.
Stem Cells in Ophthalmology Update 8: ...and So It Begins
Advanced Cell Technology announced today that it had enrolled the first patients in its two Phase I/II clinical trials, using retinal pigment epithelial (RPE) cells derived from embryonic stem cells (hESCs) for treating Stargardt’s Macular Dystropyy (SMD) and for the treatment of the dry form of age-related macular degeneration (Dry AMD). The first of twelve patients in each trial were enrolled at the Jules Stein Eye Institute at the University of California, Los Angeles (UCLA).
Stem Cells in Ophthalmology Update 9: First Patients Treated
As I reported back on June 16th, Advanced Cell Technology had enrolled the first two patients in its Phase I/II clinical trials using retinal pigment epithelial (RPE) cells derived from embryonic stem cells (hESCs) for treating Stargardt’s Macular Dystrophy (SMD) and for the treatment of the dry form of age-related macular degeneration (Dry AMD). The company announced today that these first patients had now successfully received their first dose of the stem cells.
Stem Cells in Ophthalmology Update 10: ACT Expands Trials for Embryonic Stem Cells for Stargardt’s to the UK
In a news announcement today, Advanced Cell Technology said it had received approval to expand its stem cell treatment for Stargardt’s Macular Dystrophy to Moorfield’s Hospital in the UK.
Stem Cells in Ophthalmology Update 11: Catheter Delivered Stem Cells to Treat Geographic Atrophy in Dry AMD
The story of Centecor (now Janssen)/J&J’s program to deliver stem cells to the macular in the hopes of treating geographic atrophy in dry AMD.
Stem Cells in Ophthalmology Update 12: Updated Table of Company Participants
This table is now out-date. See the offer for up-to-date tables at the end of this menu.
Stem Cells in Ophthalmology Update 13: Advanced Cell Technology Update
In the wake of the 60 Minutes expose of illegitimate stem cell activities, I thought I would bring you good news about a couple of legitimate, government approved clinical trials using stem cells.
Stem Cells in Ophthalmology Update 14: Current Stem Cell Clinical Trials
Again, this table is also out-of-date – see the offer below for the latest tables.
Stem Cells in Ophthalmology Update 15: Wills Eye Joins ACT’s Clinical Trials for Dry AMD Using Embryonic Stem Cell-derived RPE
The announcement that Wills Eye had joined the clinical trials.
Stem Cells in Ophthalmology Update 17: Recent ACT Updates
The four press releases from ACT while I was on vacation.
Stem Cells in Ophthalmology Update 18: StemCells Inc. Demonstrates That its Human Neural Stem Cells Preserve Vision – Gets FDA Authorization to Initiate Clinical Trial for Dry AMD
On January 30, 2012, StemCells Inc. announced the publication of preclinical data demonstrating that its proprietary HuCNS-SCr cells (purified human neural stem cells) protect host photoreceptors and preserve vision in an animal model of retinal disease. The preclinical results are highly relevant to human disorders of vision loss, the most notable of which is dry age-related macular degeneration (AMD). The study is available online at and will be featured as the cover article in the February issue of the international peer-reviewed European Journal of Neuroscience.
Stem Cells in Ophthalmology Update 19: ACT Adds Bascom Palmer as Another Clinical Site for Dry AMD Trials
Advanced Cell Technology said that Bascom Palmer Eye Institute had received IRB approval to become the third U.S. clinical site for testing ACT’s human embryonic stem cell-derived retinal pigment epithelial cells in the treatment of dry age-related macular degeneration. Bascom Palmer, one of the country’s premier eye institutes, joins UCLA’s Jules Stein Eye Institute and the Wills Eye Institute as the third U.S. site participating in the clinical trials.
Stem Cells in Ophthalmology Update 20: ACT Adds Mass Eye & Ear as Fourth Clinical Site for Dry AMD Trials
Advanced Cell Technology announced that Mass Eye & Ear Infirmary had received IRB approval to become the fourth clinical site for ACT’s embryonic stem cell trials in treating the dry form of age-related macular degeneration.
Harvard’s Mass Eye & Ear joins UCLA’s/Jules Stein, Wills Eye Institute, and Bascom Palmer in the ongoing Phase I/II clinical trial using subretinal injections of embryonic stem cell-derived retinal pigment epithelial cells.
In addition, it is my understanding that Moorfields Eye Hospital in London is awaiting final approval of an IND application to also participate in ACT’s clinical trial for dry AMD, along with its current participation as part of the company’s Stargardt’s disease stem cell clinical trial.
Stem Cells in Ophthalmology Update 21: Clinical Trial Details
In attempting to determine how many patients have been treated with stem cells for eye disorders, I quickly found that no one was keeping track – at least no one that I could find.
So, I decided to try and get this data. I have now obtained partial data for three of the 9 clinical trials underway and present this information in my new table.
Again, to obtain the latest version of this table, please request it via email.
Stem Cells in Ophthalmology Update 22: A Stargardt’s Clinical Trial Patient’s Story – In Her Own Words
Maurie Hill, a young woman with Stargardt’s disease was accepted into Advanced Cell Technology’s embryonic stem cell clinical trial. She was the first person to receive 100K of RPE cells derived from human embryonic stem cells.
In this blog post she describes her experiences.
Stem Cells in Ophthalmology Update 18: StemCells Inc. Demonstrates That its Human Neural Stem Cells Preserve Vision – Gets FDA Authorization to Initiate Clinical Trial for Dry AMD
On January 30, 2012, StemCells Inc. announced the publication of preclinical data demonstrating that its proprietary HuCNS-SCr cells (purified human neural stem cells) protect host photoreceptors and preserve vision in an animal model of retinal disease. The preclinical results are highly relevant to human disorders of vision loss, the most notable of which is dry age-related macular degeneration (AMD). The study is available online at and will be featured as the cover article in the February issue of the international peer-reviewed European Journal of Neuroscience.
Stem Cells in Ophthalmology Update 19: ACT Adds Bascom Palmer as Another Clinical Site for Dry AMD Trials
Advanced Cell Technology said that Bascom Palmer Eye Institute had received IRB approval to become the third U.S. clinical site for testing ACT’s human embryonic stem cell-derived retinal pigment epithelial cells in the treatment of dry age-related macular degeneration. Bascom Palmer, one of the country’s premier eye institutes, joins UCLA’s Jules Stein Eye Institute and the Wills Eye Institute as the third U.S. site participating in the clinical trials.
Stem Cells in Ophthalmology Update 20: ACT Adds Mass Eye & Ear as Fourth Clinical Site for Dry AMD Trials
Advanced Cell Technology announced that Mass Eye & Ear Infirmary had received IRB approval to become the fourth clinical site for ACT’s embryonic stem cell trials in treating the dry form of age-related macular degeneration.
Harvard’s Mass Eye & Ear joins UCLA’s/Jules Stein, Wills Eye Institute, and Bascom Palmer in the ongoing Phase I/II clinical trial using subretinal injections of embryonic stem cell-derived retinal pigment epithelial cells.
In addition, it is my understanding that Moorfields Eye Hospital in London is awaiting final approval of an IND application to also participate in ACT’s clinical trial for dry AMD, along with its current participation as part of the company’s Stargardt’s disease stem cell clinical trial.
Stem Cells in Ophthalmology Update 21: Clinical Trial Details
In attempting to determine how many patients have been treated with stem cells for eye disorders, I quickly found that no one was keeping track – at least no one that I could find.
So, I decided to try and get this data. I have now obtained partial data for three of the 9 clinical trials underway and present this information in my new table.
Again, to obtain the latest version of this table, please request it via email.
Stem Cells in Ophthalmology Update 22: A Stargardt’s Clinical Trial Patient’s Story – In Her Own Words
Maurie Hill, a young woman with Stargardt’s disease was accepted into Advanced Cell Technology’s embryonic stem cell clinical trial. She was the first person to receive 100K of RPE cells derived from human embryonic stem cells.
In this blog post she describes her experiences.
And, finally, the blog entry on how to obtain the current, up-to-date tables of both stem cell companies and clinical trials underway, and the same for gene therapy companies and trials.
Current Resources: The Use of Stem Cells and Gene Therapy in Ophthalmology
Because I have been updating this information almost on a daily basis, I’ve decided to no longer post the information on this Journal (Irv Arons’ Journal), as it is practically out-of-date almost as soon as I post it. Therefore, I will only offer my tables of information to those who are interested in receiving them, and request them via email. I usually put notices on the appropriate LinkedIn Forums and on my Twitter feed (see sidebar) when there is substantial new information to report.
Here is what is currently available:
Stem Cells
Stem Cell Companies (and Institutions) Active in Ophthalmology
A list of snineteen companies and institutions working with stem cells for ophthalmic applications. The table lists collaborators, the cell type being used, and the applications against which the cells will be applied.
Stem Cell Therapy in Ophthalmology by Applications
A list of six ophthalmic applications being tested in clinical trials. The table includes which companies/institutions are involved, the clinical trial status, and the clinical trial number for those which are currently active. (Eleven active clinical trials are listed, with live links.)
Stem Cell Therapy in Ophthalmology -- Ongoing Clinical Trials
A list of the the six ophthalmic application and the eleven clinical trials showing the number of patients to be treated in each clinical trial and the number treated to date (that I am aware of).
Stem Cell Therapy in Ophthalmology -- Ongoing Clinical Trials
A list of the the six ophthalmic application and the eleven clinical trials showing the number of patients to be treated in each clinical trial and the number treated to date (that I am aware of).
Gene Therapy
Gene Therapy Companies/Institutions Active in Ophthalmology
The table lists more than twenty-five companies and institutions actively pursuing gene therapy solutions to ophthalmic diseases. The table shows the delivery viral platform, the gene type being used (where known), the application, and clinical status.
Gene Therapy in Ophthalmology by Application
This table, like the one for stem cells, lists the ophthalmic indication, the company/institutions involved, the clinical status, and the clinical trial number. (Fifteen active clinical trials are listed, with live links.)
Gene Therapy in Ophthalmology -- Ongoing Clinical Trial Details
Gene Therapy in Ophthalmology -- Ongoing Clinical Trial Details
Again, as with the stem cell clinical trial table, this table lists all of the active clinical trials, the number of patients to be treated and the number of patients treated to date.
Anyone interested in receiving a copy of any or all of the above tables, please contact me via email (see the link in the side bar) and let me know which ones, or all four, that you wish to obtain. The tables are in pdf format, and as mentioned, contain live links to all of the clinical trials listed.
Irv Arons
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