Author: Irv Arons

A New Approach for Repairing/Rejuvenating Damaged Photoreceptors and Other Retinal Tissue for Restoring Vision

Over the past several years, I have written about new technologies for treating retinal diseases, including the use of drugs (Avastin, Lucentis and Eylea for wet AMD), laser treatment (Ellex’s 2RT – Retinal Regeneration, for dry AMD), and the use of stem cells and gene therapy for a wide range of ophthalmic diseases.

Earlier this year, I became aware of a new company, jCyte, who was investigating the use of retinal progenitor cells to replace damaged or destroyed photoreceptors to restore vision to those whose photorecptors had stopped working, especially in those with the latter stages of  retinitis pigmentosa (RP). (Read more...)

Reversing Retinal Cell Death With Inkjet Printing?

For the past several months, I have been working with Mark Hillen, the editor of The Ophthalmologist, in providing him with background information about stem cells and gene therapy used in ophthalmology to treat various retinal diseases. Articles, based on my information, along with material developed by Mark, have been published in past issues of his magazine. (Stem Cell Clinical Trials and Gene Therapy Clinical Trials.)

The Ophthalmologist is a new professional journal, published by Texere Publishing, for a target audience of ophthalmologists and industry professionals, mainly based in Europe.

The latest issue (February 2014) contains an (Read more...)

Laser Refractive Keratoplasty: The Rest of the Story

In 1989, I wrote a paper published by Arthur D. Little, A White Paper – The Evolution and Prospects for Laser Refractive Keratoplasty. This was the first paper I wrote on the potential for (at that time) LRK, now known as PRK, that later turned into LASIK. It provides a comprehensive look at the early history of refractive surgery as I knew it.

Last December, I learned of an article based on a press release put out by the Optical Society of America (OSA), that described the invention of using the excimer laser to ablate human tissue in the (Read more...)

AMD Update 25: Results of The AREDS2 HOME Study of Notal Vision’s Home Monitoring Device for AMD Announced

In April of 2010, I wrote about the inclusion of Notal Vision’s ForseeHome AMD Monitor in the AREDS2 clinical trial.
The overall objective of the two arm randomized clinical trial was to determine if home monitoring of participants at high risk of progression from late-stage dry AMD to neovascular AMD, using the comprehensive visual field and telemedicine solution based on the ForeseeHome Device in AREDS2 (referred to as the ForeseeHome comprehensive solution), would improve detection of progression to choroidal neovascularization (CNV) when compared with standard care (may have included use of the Amsler Grid).

AMD Update 24: DARPins Phase 2 Trial Results Fall Short

Back in February, I first reported on Allergan’s DARPins in my Update 23: DARPins, The Next “Game Changer” for Wet AMD? In that report, I wrote that Molecular Partners’ MPO112 (Allergan’s AGN-150998) showed promise of improving vision and having a long ocular half-life which appeared to be a vast improvement over both Lucentis and Eylea, perhaps requiring injections every 3-4 months compared to bi-monthly for Eylea and monthly for Lucentis and Avastin. (I also noted a second agreement with Molecular Partners, the licensors of the DARPin technology to Allergan, in which a combination dual action anti-VEGF/PDGF drug therapy was also (Read more...)

Gene Therapy in Ophthalmology Update 21: New Gene Therapy Company, Spark Therapeutics, Launches

Children’s Hospital of Philadelphia (CHOP) announced that it had spun off its work in gene therapy to a new, fully integrated company, Spark Therapeutics, that will assume control over two current gene therapy clinical trials: a Phase III study for Leber’s Congenital Amaurosis, an inherited disease that results in blindness caused by mutations of the RPE65 gene, and a Phase I/II study for hemophilia B. The new company is also advancing toward the clinic with gene therapy programs to address neurodegenerative diseases and additional hematologic disorders and other forms of inherited blindness. One such program, in the latter category, (Read more...)

Gene Therapy in Ophthalmology Update 20: Oxford BioMedica Clinical Trials Resume

Back in June, Oxford BioMedica announced that it had voluntarily paused recruitment for its clinical trials for wet AMD  (RetinoStat Phase I), Stargardt’s Disease (StarGen Phase I/IIa) and Usher’s Syndrome (UshStat Phase I/IIa). The company had halted recruitment of the aforementioned studies, as a precautionary measure, while it investigated the detection of very low concentrations of a potential impurity in its clinical trial material derived from a third party raw material.
Oxford has since performed extensive characterization studies using its newly developed, state-of-the-art analytical methods to identify the impurity as highly fragmented DNA derived from fetal bovine serum (FBS), the (Read more...)

Research in Retinal Disease: The Foundation Fighting Blindness Invests $2.1 Million in Seven New Research Efforts

As I continually search the web for interesting news about new technologies for treating retinal diseases, I came across this news from the Foundation Fighting Blindness’ website  yesterday afternoon. It relates to some of the annual grants to researchers that the FFB will be funding this year. It includes better ways of looking at retinal cells (via use of the adaptive optics laser scanning ophthalmoscope) and several projects involving gene therapy, along with a couple looking at ways of, hopefully, stopping the progression of dry AMD.

The following write up is reprinted with permission of the FFB.

A New Approach to Treating Chronic Kidney Disease: Replenishing Your Nephrons

As some of you may know, I have chronic kidney disease (CKD). Thanks to my wife and the scare put into me by my nephrologist, that I would soon have to begin dialysis, I have managed to get my CKD  in remission, or at least under control. My GFR number (that indicates when you must start dialysis) has held steady, or actually gotten better since my wife put me on a strict diet and I have lost about 15 pounds – and my nephrologist took me off of lisinopril (for blood pressure control), which seemed to raise my GFR by (Read more...)

Gene Therapy in Ophthalmology Update 19: A New Virus Vector for Safer Delivery of Gene Therapies

Researchers at the University of California at Berkeley, along with some assistance from the Flaum Eye Institute and Center for Visual Science at the University of Rochester, have come up with a new version of an adeno-associated virus (AAV) vector that can deliver genes deep into the retina using an intravitreal injection of the vector into the vitreous, a less-invasive technique, instead of an intraretinal injection below the surface of the retina, which is the common way gene therapy is currently delivered.
The study was authored by postdoctoral fellows Deniz Dalkaral (then with Helen Wills Neuroscience Institute of UCal Berkeley (Read more...)

Stem Cells in Ophthalmology Update 25: ACT Patient in Dry AMD Trial Goes from 20/400 to 20/40!

The story started innocently enough. On Wednesday, May 15th, the journal Cell reported on a study that claimed biologists had finally created human stem cells by the same technique that produced Dolly the cloned sheep in 1996. They transplanted genetic material from an adult cell into an egg whose own DNA had been removed.
OK, an important story but what followed boggles the mind. Many science reporters wrote about the discovery which got picked up by several news sources. However, a sharp-eyed member of the Investor Stemcell Forum (iCell), a group originally started by investors in Advanced Cell Technology (Read more...)

A New Technique for Restoring Normal Vision to the Blind: The Technology of Prof. Sheila Nirenberg of Weill Cornell Medical School

About a year ago, a colleague put me in touch with a Boston-based venture capitalist who was interested in a new method for restoring vision to the blind that was under development at Cornell University. I did some cursory research about the technology and wrote a brief report about what I learned.

I really didn’t understand the front end of the technology – how the research team was able to acquire and manage a useful visual signal that could be converted into sight by the brain, but since the back end involved the use of gene therapy, which I was (Read more...)

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.

A Personal Journey: How I Went From Being A Bench Chemist to An Expert Resource in Ophthalmology and Medical Lasers

A short while ago, I was asked by Maureen Duffy, editor of VisionAware, the blog of the American Foundation for the Blind, how I became so knowledgeable about ophthalmology and why I started my blog. I prepared some background information for Maureen and she published it as a guest blog on her site, but because of space limitations, she was only able to use an abridged version. Since I don’t have the same space limitations, I decided to publish the “unabridged” version here.

So, here is my story:

The Beginning of My Career in Chemistry
I graduated from (Read more...)

People in the NorthWest with X-linked retinoschisis

XLRS Natural History Study Beginning in Portland, Oregon

March 29, 2013 – Oregon Health & Science University (OHSU) is launching a three-year natural history study for people with X-linked retinoschisis (XLRS). Funded by the Foundation Fighting Blindness, the investigation’s primary goal is to identify outcome measures — such as changes in vision or retinal structure — that could be useful in evaluating the effectiveness of potential therapies in clinical trials. The study will also help determine the types of XLRS patients most suitable for future therapeutic studies.

Knowledge gained from the XLRS natural history study will aid in the (Read more...)

Gene Therapy in Ophthalmology Update 18: A RetroSense Update

I first learned about the potential of using gene therapies in treating ophthalmic disorders back in November 2010. That’s when I was introduced to gene therapy by Sean Ainsworth, the founder and CEO of RetroSense Therapeutics. I haven’t written about this company or the unique approach it is taking to try and treat retinitis pigmentosa and the dry form of AMD since that first article, The Use of Gene Therapy in Treating Retinitis Pigmentosa and Dry AMD. With several news events occurring with the company recently, I felt it was time to bring readers of this blog up-to-date.

Gene Therapy in Ophthalmology Update 17: Hemera Biosciences Obtains Initial Funding

In December 2011, following that year’s AAO Meeting, I wrote about Hemera Biosciences and its complement regulation therapy via the use of gene therapy to prevent membrane attack complex (MAC), the final stage of the complement cascade that is implicated in both dry and wet AMD. (Gene Therapy in Ophthalmology Update 5: A Complement-Based Gene Therapy for AMD)

I am now happy to report that Hemera has obtained initial funding, along with the issuance of a US Patent and can now begin manufacturing its drug, soluble CD59 (protectin), perform animal toxicology and initiate a phase 1 clinical study.

Oraya IRay Update 2: INTREPID Two-year Results Meet Primary Clinical Endpoint – Results in At Least 35% Fewer anti-VEGF Injections — Oraya Joins with Optegra to Provide Treatments in the UK

The last time we checked in on Oraya in May 2011, the company had announced it had completed enrollment in its INTREPID clinical trial, being conducted at seven European sites with the enrollment of a minimum of 150 patients. (Oraya IRay Update: Company Completes Enrollment in European Clinical Trial)
The INTREPID trial is the first sham-controlled double-masked study to evaluate the effectiveness and safety of a one-time radiation therapy in conjunction with as-needed anti-VEGF injections for the treatment of wet AMD. A total of 21 sites in five European countries participated in the trial with a total enrollment (Read more...)

AMD Update 23: DARPins, The Next “Game Changer” for Wet AMD?

As many of you know, I am now retired and no longer attending ophthalmic industry meetings, the source for much of my writing when I was producing the “Technology Update” columns for Ocular Surgery News for over eleven years. I now scour the web searching for interesting ophthalmic industry news in the field of my current interest –  new technologies for treating retinal diseases, for ideas of stories to write about for this Journal. I also rely on tips from former industry colleagues and new friends that inquire, “Have you heard about…”, which sometimes leads to interesting stories to investigate (Read more...)