Monday, April 22, 2013

Recently Published Articles: Current Status of Stem Cells and Gene Therapy in Ophthalmology

In the past couple of months, I was asked to update an article I wrote on stem cells in ophthalmology, originally published in Retina Today, for its sister publication, Advanced Ocular Care, and to write a similar article about the current status of gene therapy for another ophthalmic publication, Retinal Physician. These two articles have now been published in the respective journals and made available online.

Here is a brief summary of each article, along with the link to its online version and a note about finding the current versions of the tables associated with each, online.

The Current Status of Stem Cells in Eye Care, Advanced Ocular Care, March 2013

As noted, this is an update of the original article that appeared in the May/June issue of Retina Today.

“From an inauspicious start several years ago, the use of stem cells in the treatment of several ocular and retinal diseases has picked up steam over the past year.”

The article goes on to describe what stem cells are, the applications of stem cells in the various parts of the eye, a brief discussion of the status of some of the clinical trials, and concludes with a quote from Dr. Stephen Rose, chief research officer of the Foundation Fighting Blindness, who wrote, “Of course, it would be nice if all parts of our bodies, including our retinas, came with extended warranties so you could just swap them out when they go bad. But now that I think about it, that’s what stem cells might do for us someday.”

“With the first approval of a gene therapy treatment for treating a genetic disorder in the Western world, the future of gene therapy for treating ocular disorders looks bright.”

The article goes on to discuss what gene therapy is and how it works; the applications of gene therapy in ophthalmology and clinical trial status for four ocular diseases – Leber’s Congenital Amaurosis, wet AMD, Stargardt Disease, and Usher Syndrom 1b; attempts to answer some remaining questions; and concludes with a quote from officials with the Office of Cellular, Tissue and Gene Therapies (OCTGT) for the FDA, “The recent history of gene therapy has been a mixture of promise and disappointment ... Despite the setbacks of the past, the OCTGT shares the enthusiasm of the field and is confident that ongoing clinical investigations will lead to commercially available gene therapy products that are safe and effective and advance the public health.”

Current Versions of Stem Cells and Gene Therapy Tables

Because of the lag between submission and publication of the above articles, the tables that are linked to the print and online versions of the above articles are currently out-of-date. I constantly update their contents and publish the latest versions online, which are accessible from my blog entry about each set of tables:

Stem Cell/Cell Therapy Companies/Institutions Active in Ophthalmology

A list of thirty-two companies and institutions working with stem cells/cell therapies for ophthalmic applications. The table lists collaborators, the cell type being used, and the applications for which the cells will be used.

Stem Cell/Cell Therapy in Ophthalmology by Application

A list of sixteen ophthalmic applications being studied in clinical trials. The table includes the companies/institutions involved, the clinical trial status, and an active link for the clinical trial for those listed. (Thirty-six active and completed clinical trials are shown.)

Stem Cell/Cell Therapy in Ophthalmology -- Ongoing & Completed Clinical Trial Details

A list of the the sixteen ophthalmic applications and the thirty-six clinical trials showing the number of patients to be studied in each trial and the number studied to date (that I am aware of). Active links are provided for each ongoing or completed trial.

Gene Therapy Companies/Institutions Active in Ophthalmology

The table lists more than thirty-two 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 nineteen ophthalmic indications, the company/institutions involved, the clinical status, and the clinical trial number. (Sixteen active clinical trials are listed, with live links.)

Gene Therapy in Ophthalmology -- Ongoing Clinical Trial Details

Again, as with the stem cell clinical trial table, this table lists the sixteen active and completed clinical trials, the number of patients to be treated and the number of patients treated to date.

Note: The links in the tables associated with the clinical trials are “active” or “live” and will take you to each clinical trial on


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