Hi everyone,

I've had to sit on this information since last October. I was finally given permission to go public with it this past Friday, which is the reason why I haven't been busy on these boards. Once I sent this email to all my friends that share my eye disease, my email inbox exploded!

There's a company called Neurotech that has manufactured this really cool device called Encapsulated Cell Technology. Before you read the rest of this, read this article first:

http://www.health24.com/news/Eye_vision/1-909,34447.asp

Okay, all set? Here we go....

I sent the article to Dr. Tao, chief scientific advisor of Neurotech (the makers of the ECT), and asked her that, given that this article is now public, can I go public with the rest? Her answer was yes. I've known about these results for about 4 months now, and have been chomping at the bit to be able to tell you, as Paul Harvey says, "The rest of the story".

The article mentions that all ten of the RP (Retinitis Pigmentosa) patients showed some improvement in their sight, even though sight improvement wasn't the focus of the trial. The focus was on safety. They just wanted to be sure that this ECT wouldn't be harmful to humans.

What the article DIDN'T say was that there was one patient who had significant improvement over this short 6 month trial. His acuity in the treated eye went from 20/120 to 20/50, and he got a little of his peripheral vision back as well. Moreover, six months AFTER they removed the ECT, he showed no further detioration.

When he went for the six month followup, one of the doctors looked at his eyes and said, "You know, I'm not sure you have RP". They gave him a blood test.

He's a CHMer, guys!!!

Let me say that again: HE HAS CHOROIDEREMIA, NOT RP!

The ONE person who had significant improvement is one of us!!!

Okay, take a deep breath everyone and calm down.... Now, let me continue.

I learned of this because this patient contacted the CRF after his diagnosis of CHM. I called him and he told me the whole story. After I got off the phone with him, I immediately called Dr. MacDonald (no relation, I wish someone in my family was this smart. He's a leading researcher in Choroideremia). I said, "Dr. Mac, have you seen the results on the Phase 1 ECT trial for RPers yet?" He said yes, he'd just received it a few days ago. I asked, "Did you notice that one patient did better than the rest?" He said yes, that he'd been wondering about that. I said, "Ready for this? He doesn't have RP, he has CHM".

There was dead silence for a few seconds, then a voice said, "Hold on a second, I dropped the phone". LOL

I said, "So, by your reaction, I'm assuming I SHOULD be as excited as I am, correct?" Dr. Mac replied, "Oh yes". Come to find out, Dr. Mac had been telling the people at the NEI even before the trial started that, in his opinion, CHMers would do really well with the ECT. As it turned out, he was leaving in a few days to go to the NEI, and he put, "Lobby HARD for inclusion of CHMers" on his to-do list when he went there. He also advised me to contact Dr. Tao at Neurotech and Dr. Sieving at the NEI, which I did. They both requested that I keep mum about this until they went public with the results. I agreed. Until today, only the Board of the CHM Research Foundation (CRF) knew about this.

The end result of all this is that the NEI is considering using CHMers in the Phase 2 trial, scheduled to begin recruitment in late spring! We don't know what the protocols will be yet, so we're not sure what age group or vision level they will be looking for. However, when speaking with Dr. Tao about this, she said that the CRF, along with Dr. MacDonald, will be the main sources of CHMers for the study!

This is NOT set in stone yet, but with Dr. MacDonald lobbying hard for us, the Board of the CRF doing the same, and the makers of the ECT (Neurotech) pushing for inclusion of CHMers, I think we have an excellant chance of seeing the VERY FIRST clinical trial for a treatment of Choroideremia!!!

I will keep everyone posted as things develop.

If you want to read more detail about the ECT, here's a link to Neurotech's website:

http://www.neurotechusa.com/product_tech.asp

I'm SO happy to be able to share this news!
Cory

You are not a human being having a spiritual experience, but a spiritual being having a human experience.

<<<<<<I'm SO happy to be able to share this news!>>>>>

So am I, so am I.... !!! and you know why LOL


And as you walk you make your path Kat

Cory-



It will be a treat to look into you eyes and smile!!!!!!!!!!!

Best of Luck, and here's to medical advancements!

TLMM

Cory, that's great news!! When will thay start the testing on CHMers? I hope you get to move tot he front of the line!

Also, I'm going to send this article to my boss - his son has macular degeneration.

Congrats, Cory, that's heeeeeyuge!

Monica

This is your life. Are you who you want to be? ~ Switchfoot

Monica, depending on his type of MD, there might be good news for him as well, as their starting Phase 2 trials on a form of MD using this same device.

From the Foundation Fighting Blindness website:

Neurotech, a biotechnology company with operations in the U.S. and France, has announced that it is initiating a Phase II human clinical trial of its Encapsulated Cell Technology (ECT) for the treatment of vision loss due to the dry form of age-related macular degeneration (AMD). Two Phase II studies of the ECT for the treatment of retinitis pigmentosa (RP) are also planned for the second quarter of this year, once the dry AMD trials are underway.

The forthcoming trials for dry AMD will be conducted at the National Eye Institute (NEI) in Bethesda, Maryland.

The ECT is a tiny device — six millimeters in length — that contains cells that produce a vision-preserving protein called Ciliary Neurotrophic Factor (CNTF). In a Phase I clinical trial of the ECT with CNTF at the NEI, the device was well-tolerated, and some patients also showed improvements in their visual acuity.

Cory

You are not a human being having a spiritual experience, but a spiritual being having a human experience.

Well, Cory, maybe it's proof that people who work hard get lucky. Terrific news!

Chris.

Wow Cory, this is so exciting. I am really thrilled for you. I could feel the excitement in your post!!

Charlie

That's very exciting Cory.

Cory,

Finally...some great news in this area.

Tex

>>the VERY FIRST clinical trial for a treatment of Choroideremia!!!<<

Holy cow, Cory. That's one heck of a development. I hope this pans out (of course) but a little bit of caution might not be such a Bad Thing(tm). It's only a first trial. It sure sounds like progress, though. What an amazing coincidence that one guy was misdiagnosed!

 

Q, you're right, it is only a first trial. However, considering that in the 37+ years since I've been diagnosed, it will be the first EVER trial, it's pretty historic and obviously something to get excited about.

As for the misdiagnosis, I'm used to that for the most part. Most local ophthalmologists seem to be a bit lazy, glance into the eye and say, "You have RP. Have a good day". Especially in the early stages, Retinitis Pigmentosa (RP) and Choroideremia (CHM) look almost exactly alike when viewed through an ophthalmoscope. From what I've been told, the difference, and it's a small one, is that CHMers have a bit more clumping in the Retinal Pigment Epithelium (RPE)and Choroid, which isn't easily seen. The RPE and Choroid lie behind the Retina. The retina contains our photoreceptors (rods & cones) and the other two layers are the "support mechanism" for lack of a better term.

The ones that don't have egos will admit they're not sure and refer the patient to a retinal specialist, who then can make a more informed diagnosis using a battery of tests, including Electroretinagrams (ERG's) and a genetic blood test.

What got me about this guy was that he was diagnosed at Johns Hopkins, by one of the leading retinal researchers in the WORLD, Dr. Finkelstein! That's what blew me away...

Cory
PS: Since we went public with this news, this gentleman has suddenly achieved "rock star" status in the CHM world... I told him to be ready to sign autographs at our conference in Chicago... LOL

You are not a human being having a spiritual experience, but a spiritual being having a human experience.

>>What got me about this guy was that he was diagnosed at Johns Hopkins, by one of the leading retinal researchers in the WORLD, Dr. Finkelstein! That's what blew me away ...<<

The take home message being that even Dr. Finklestein makes mistakes? You know what your mother always said about what happened to the only perfect guy in the world ....

Seriously though ... thank goodness Dr. Finklestein is human.

Hip Hip Hurray!!!!!
Great News, Cory
Keep us up to date on the latest developments.
jbean

Congrats Cory.........this is great news indeed!!

hi cory

that is wonderful news and i really hope the trial provides results or a direction for more work and god willing a cure, so the newly diagnosed and those like you who have tavelled with this for so long.

and big hugs and god bless to you for all the work you do and the committment you show to others in this situation.

thanks also for sharing a medical historical moment, here's hoping for ongoing positive outcomes for you all

hugs
kath

Wow Cory, that is awesome news for you!
Glad to hear it............thanks for sharing the good news old friend,
James