Tuesday, May 22, 2012

Stem Cells in Ophthalmology Update 21: Clinical Trial Details

As with my inquiry into clinical trials underway using gene therapy in ophthalmology, Table 3 Gene Therapy in Ophthalmology - Ongoing Clinical Trial Details, I have undertaken a similar survey of the number of patients currently treated using stem cells. I found nine ongoing clinical trials and one about to begin.

As in the previous study, I visited the clinical trial information sites and extracted the pertinent information for use in this table, Table 3. Stem Cell Therapy in Ophthalmology - Ongoing Clinical Trial Details.

I then contacted each of the principals or sponsor involved in the study and requested the number of patients treated to date. The response to date has not been as good as with the gene therapy trials, but here is what I was able to find out.

Only two companies/institutions involved answered my request, and so I can only reliably report that 17 patients have been given stem cells for treatment of ophthalmic disorders to date.

I hope to gather additional information from either the clinical investigators or others and will update this table as new information is obtained.

Here, then, is the initial version of this table.



A downloadable pdf file of the above table is available by request.

To obtain copies of the current versions of the first two tables, listed below, please send me an email.

Table 1. Stem Cell Companies/Institutions Active in Ophthalmology

Table 2. Stem Cell Therapy in Ophthalmology by Application

Friday, May 18, 2012

Gene Therapy in Ophthalmology Update 11: Clinical Trial Details

I have found fifteen gene therapy clinical trials underway to treat various eye disorders. In thinking about this, I wondered how many patients had been treated to date in these clinical trials. Not able to find this information, I decided to undertake the task of obtaining the information by asking the people running the trials.

This resulted in this new table, Table 3 Gene Therapy in Ophthalmologly - Ongoing Clinical Trial Details.

I have visited each National Clinical Trial (NCT) site, derived the pertinent information, and placed it into the table.

I then requested the number of patients treated to date in each trial from the principal investigator or sponsor. I was able to get information from nine of the fifteen trials – either from the principal source or from a reliable secondary source.

It turns out that since the first gene therapy clinical trial for Leber’s Congenital Amaurosis by University College London and Moorfields Hospital in February 2007, more than 50 patients and 54 eyes have received gene therapy treatment (at least 4 second eyes have been treated by Children’s Hospital in Philadelphia in their Leber’s trial).

As new information is obtained, I will continue to issue updates.

Here then, is the initial version of the table:



Irv Arons May 2012 (Version 1, 5/18/12)

NYR - Not yet recruiting
R - Recruiting

1. Reported at ARVO 2012


A downloadable pdf file of the above table is available by request.

To obtain copies of the current versions of the first two tables, listed below, please send me an email.

Table 1. Gene Therapy Companies/Institutions Active in Ophthalmology

Table 2. Gene Therapy in Ophthalmology by Application


Editors Note:

Two quick corrections in the published table:

1. Oxford Biomedica should read Oxford BioMedica.

2. King Khaled Eye Hospital is treating MERTK-related autosomal recessive RP, and not autosomal-dominent RP as listed.

Both of these corrections will appear in future updates.

Version 2 (May 22, 2012), containing several data updates will be available on May 22nd by email request.




Wednesday, May 02, 2012

Stem Cells in Ophthalmology Update 20: ACT Adds Mass Eye & Ear as Fourth Clinical Site for Dry AMD Trials

As announced this morning, Advanced Cell Technology said 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.

The following information was taken from the company’s news release:

"We are delighted to announce that Mass. Eye and Ear will participate as a site for our clinical trial for dry AMD," said Gary Rabin, ACT's chairman and CEO. "Dr. Dean Eliott and his team are deeply committed to finding new treatments for preventing blindness, and we very much look forward to tapping into his expertise and insight into the progression of macular degenerative disorders. The primary teaching hospital for ophthalmology at Harvard Medical School, Mass. Eye and Ear is ranked as among the top ophthalmology hospitals in the country by U.S. News & World Report and has a reputation that is unrivaled."

The Phase I/II trial is a prospective, open-label study designed to determine the safety and tolerability of the hESC-derived RPE cells following sub-retinal transplantation into patients with dry AMD. The trial will ultimately enroll 12 patients, with cohorts of three patients each in an ascending dosage format.

"Dry AMD represents one of the largest unmet medical needs in ophthalmology," commented Dr. Dean Eliott, M.D. a full time retina surgeon, scientist and Associate Director of the Retina Service at Mass. Eye and Ear. "We appreciate the opportunity to get some first-hand experience with the protocol and be involved with the international team that has been assembled around the U.S. and European trials."