Thursday, December 31, 2015

Stem Cells in Ophthalmology Update 24: Current Tables Now Online

My current stem cell/cell therapy tables are now online for anyone interested to access. Here is a brief description of what is available and how to access them:

Stem Cell/Cell Therapy Companies/Institutions Active in Ophthalmology

A list of forty-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 eighteen ophthalmic applications being studied and/or 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. (sixty-one active and completed clinical trials are shown.)

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

A list of the the eighteen ophthalmic applications and the sixty-one 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.


Updated December 31, 2015. 

Tuesday, December 01, 2015

Articles Published in Ophthalmic Journals in 2015

In addition to the several updates posted on this blog in 2015 (see previous posting), I have prepared four articles that have appeared in ophthalmic journals this year.

Here is a brief summary of the four articles, including links to the online versions:

Regenerating the Retina - February 2015

This article, published in the February 2015 issue of The Ophthalmologist, describes the use of stem cell-derived retinal progenitor cells (RPCs), that are being investigated for reviving/repairing/rejuvenating damaged photoreceptors to bring back sight to those who have lost it due to a retinal degenerative disease, including choroideremeia (CHM), retinitis pigmentosa (RP), Leber’s congenital amaurosis (LCA) and Stargardt’s disease (Stargardt Macular Dystrophy - SMD). It discusses the four companies that are conducting clinical research, as well as the work underway at several university and institutional laboratories.

To read the complete write up, please use the following link:

Update: Since the article appeared, two of the companies discussed, jCyte and ReNeuron have either begun a clinical trial (jCyte), or announced the start of one (ReNeuron).

With the likelihood of a gene therapy and/or a stem cell treatment for retinal diseases to be approved for marketing within the next two to three years, it is time for the ophthalmic community – the suppliers, practitioners, patients and payers –  to start thinking about how much these regenerative medicine treatments are likely to cost, and how patients and the healthcare system will pay for them..

In The Economics of Gene Therapy, appearing in the May 2015 issue of The Ophthalmologist, I propose a pricing model for Regenerative Medicine in Ophthalmology, based on the population of patients to be treated, and suggest that an annuity program model, based on performance and duration of efficacy, could be used to pay for it.

Let the dialogue begin.

To read all about it, please follow this link:

An update of the latest clinical information in the use of gene therapy in treating several retinal diseases, including Leber’s Congenital Amaurosis (LCA); the wet form of Age-Related Macular Degeneration (wet-AMD); Choroideremia (CHM); Stargardt’s Macular Dystrophy (SMD); Retinitis Pigmentosa (RP); and Ushers Syndrome (US). Included is the proposed model of pricing for some gene therapy treatments, based on the population of patients likely to be treated.

To read the full article, published in the October issue of Retinal Physician, please follow this link:

Optogenetics is the introduction of protein-based, light-activated chemicals into still functional retinal cells in the vision chain, that upon activation, send electrical signals along the optic nerve to the brain, providing rudimentary vision, that was lost with the death or damage of the photoreceptors.

This report, The Optogenetics Option, describes the efforts of four companies and ten universities, using either gene therapy, or other means, to deliver light-activatable proteins or chemicals (photoswitches) to still functioning cells within the retina (ganglion and/or bipolar cells) or, in some cases, the use of light activatable implants, that will deliver light signals to the brain to provide some rudimentary vision when the photoreceptors, that normally provide that function, cannot.

To read the article, published in the November issue of The Ophthalmologist, please follow this link:

Blog Articles Published in 2015

2015 was another busy year in writing about new developments in treating retinal diseases. During the year, I published seven blog entries (and another four published articles - to be indexed separately). Here are the highlights of the blog writeups:

Since I last wrote about Iluvien, Alimera Sciences and pSivida have announced additional marketing approvals for its use in treating chronic DME. The product is now approved for use in fourteen countries, including the U.S. In addition, pSivida is about to begin a Phase III study of its Medidur device for the treatment of uveitis, which should lead to a fast-track to approval.

But the real reason for this update was the recently published story about how an eye doctor reached out to Paul Ashton, CEO of pSivida, to package an old HIV anti-viral, ganciclovir, in his drug delivery system, to save the sight of a person undergoing chemotherapy for leukemia, who also needed a bone marrow transplant. The chemo and radiation treatment for the bone marrow transplant weakened his immune system, preventing control of his cytomegalovirus condition that began attacking his retina. Instead of painful weekly anti-viral injections, the doctor sought to use Paul’s drug delivery system to systemically treat the virus in the eye.

To read the whole story and the results, and for the latest information about Iluvien, please follow this link:

With the presentation of the three-year safety results of the INTREPID study (at EURETINA), to evaluate the safety and efficacy of the Oraya Iray Therapy in conjunction with as-needed anti-VEGF injections for wet AMD, and the recent collaboration agreement with Carl Zeiss Meditec, Oraya Therapeutics is well on its way in implementing its growth strategy in commercializing Oraya Therapy in Europe and, some day, in the U.S.

The company now has nine centers providing therapeutic treatment in Europe; four centers in the UK, one in Switzerland, and four in Germany.

To read more about this combination treatment to reduce the number of anti-VEGF treatments needed to control wet AMD, and the collaboration agreement with Carl Zeiss Meditec, please see:

Avalanche Biotechnologies in Menlo Park and the University of Washington in Seattle announced a licensing agreement to develop the first gene therapy treatment for treating color blindness. The deal brings together a gene therapy technique developed by Avalanche with the expertise of vision researchers at the University of Washington.

In addition, Avalanche will incorporate research licensed from UCal Berkeley to deliver the gene therapy treatment non-surgically via an  injection into the vitreous, rather than into the retina.

To read more, please follow this link:

As Crystal Gayle sang in her smash hit, `Don't It Make My Brown Eyes Blue', now (soon) you will be able to permanently change your brown eyes to blue. A California company has come up with a laser procedure that will safely accomplish this in about 30 seconds per eye. Human clinical testing is underway and the company, Strōma Medical, hopes to have the procedure on the market (outside of the U.S. first) in less than two years, once the clinical trials are completed.

To read the rest of the story, please follow this link:

My article about the use of lasers to treat floaters, written five year ago, remains the most widely read piece on my blog. I frequently am asked if I have updated the list of doctors who now use lasers to treat floaters - in addition to the three I profiled in my U.S. writeup (Using Lasers to Treat Vitreous Floaters: Laser Vitreolysis) and the six doctors in the UK/Europe piece (Using Lasers to Treat Vitreous Floaters: Laser Vitreolysis in the UK and Europe).

I took notice when Ellex Medical announced a new YAG laser specifically for treating floaters in the fall of 2012, and about six months ago, I decided to request a list of doctors who have obtained the laser, and were now treating patients’ floaters. Better than a list, Ellex decided, to respond to my request, by building an app to locate doctors using their laser and put it on their website. That app is now active and is applicable to doctors worldwide using the new Ultra Q Reflex YAG laser to treat floaters. As of June 30th, there are 58 physicians listed worldwide using this laser.

To read more about the laser and use the app, see my update:

Some insight into the Spark Therapeutics Phase III clinical trial results and longevity data. In this report, the company’s principal investigator, Dr. Stephen Russell presented highlights of the initial findings in this important Phase III clinical trial, that could lead to the first gene therapy approval in the United States.

Basically, it was found that the injection of SPK-RPR65 did lead to increased functional vision for the treated patients, compared to the control subjects - and the effect appears to last for over three years, based on the original patients treated in the Phase I study.

Is this the “forever fix”, or a step along the way? To read the full story, please follow this link:

Three German ophthalmological groups provided guidance for German ophthalmologists to better identify those patients with wet AMD that might benefit from the use of the Iray Stereotactic Radiotherapy System as an adjuctive treatment to the use of anti-VEGFs in the treatment of neovascular macular degeneration, based on studies conducted by the company to date.

To read the full story, please follow this link: