Saturday, January 14, 2012

Gene Therapy in Ophthalmology Update 7: 2012 the Year for Gene Therapy?

While I have previously written about the progress being made in the use of stem cells in ophthalmology (see Stem Cell Update 13) and described the 9-10 clinical trials currently underway or about to start (see Stem Cell Update 14), recent events point to 2012 becoming a breakthrough year for the use of gene therapy to overcome genetic defects that cause several ophthalmic diseases.

In the accompanying table, I list the fourteen clinical trials that I know about in the use of gene therapy in treating ophthalmic disease. Half of the trials are aimed at treating Leber’s Congenital Amaurosis (LCA), while three are for treating the wet form of AMD; one is underway for treating Choroideremia; one for Stargardt’s Disease; and two are aimed at different forms of retinitis pigmentosa (Autosomal Recessive RP and Usher Syndrome 1b).

In addition, I show at least twenty four clinical trials in either the pre-clinical (animal study) mode, or a couple in the IND-preparation mode. That is close to forty clinical trials using gene therapy to treat ophthalmic diseases.

The treatment of Leber’s using gene therapy has been ongoing for at least three years and, as I will show in the next update (Gene Therapy Update 8), those trials are going quite well, with many of the patients showing improved vision.

Finally, as another indicator that gene therapy will play an important role in ophthalmology in this year, Ocular Surgery News is about to begin a special section, OSN Retina, to be part of it’s coverage of the ophthalmic scene. The January 25 issue of Ocular Surgery News will include OSN Retina - a leading destination that will provide retina specialists with more relevant information specific to their field.. The premiere issue will include a feature on how  “Retinal gene therapy may pave the way for attempts to reverse genetic disease: Advancements in retinal gene therapy have prompted a collaborative effort to attain FDA approval.”

For those of you who wish a better understanding of how gene therapy works, and until I write the Primer on the Use of Gene Therapy in Ophthalmology, which I have threatened to write for the past year and a half, you can gain an understanding by reading my first article about gene therapy, written back in November 2010, The Use of Gene Therapy in Treating Retinitis Pigmentosa and Dry AMD by Retrosense.

Here then is my latest version of Gene Therapy in Ophthalmology by Application:

A pdf file of the table is available by email request.


At 5:23 PM, Blogger Emelie said...

Great news!
Is that Stargardt's clinical trial (nct01367444) still happening? I heard it was stopped?

At 5:45 PM, Blogger Irv Arons said...

empe, I just went into the FDA site, and it said that as of Dec. 19, 2011, the study was ongoing and still recruiting. Here is the link:

Irv Arons

At 9:38 AM, Blogger Emelie said...

Great! Thank you!
Your blog has a new follower!

At 9:48 AM, Blogger Steve Fan said...

Thank you for the info.

At 1:46 AM, Anonymous Anonymous said...

Hello Irv, Thank you for sharing this blog and the materials of great importance to all of those who are looking forward to new developments in the sciences of Gene and Stem Cell therapy. I am a patient who lost his site a year back one eye due to Glaucoma and the other due to CRAO. Now there is no hope of recovery as of date and I have been reading up on different sources on the internet on any developments. I did also come across this video of a interview of Dr. Peter Crapo by Fox News. Here he talks about tissue and nerve regeneration. Unfortunately, the video does not complete and I missed out on the complete interview. Perhaps you can have a look on the same and comment accordingly. The link to the video is:

At 11:11 AM, Blogger Irv Arons said...

Anonymous, I watched the video and am not aware of the program the doctor was trying to describe, so I don't know if he is working with gene therapy or not.

The only program I am aware of working on optic nerve atrophy is one with stem cells, being undertaken in China.

See my Stem Cell Therapy by Appplication table for more details.

At 3:43 PM, Anonymous Anonymous said...

I was curious why the LHON research is in the same block as the LCA research, when one is optic nerve and the other is retina? I was also wondering if you have any insight as to the progress of the LHON research?

At 8:05 PM, Blogger Irv Arons said...

Anonymous, it was just convenient to put the disease states in the same block, since they were both attributed to Leber's.

As for progress of the LHON study, I'm sorry but I don't have any information about it.


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