My New Career?

Not to belabor a point, but today did start as a rather poopy day. We had our first snow overnight. Just a couple of inches but it was the puppygirls’ first snow. My theory is this: when they went out and squatted to poop, they got cold, wet bottoms. Go inside and poop? No cold, wet bottom. Problem solved!

Of course, since my husband was first on scene and got to clean it up, he was NOT a happy camper. Not a good start to the day.

Then there was the snow itself. Getting to my eye appointment is a 90 mile trip one way. My husband does not like city driving and he does not like driving in the snow. Once more he was not a happy camper.

It is hard to explain to some people why we have been dutifully driving 90 miles one way, every six months just to be told my eyes are worse. “Please pay your copay.” For those kind of results, I could go five miles down the road!

However, it is all part of a master plan. I have real problems with defeat. Real problem being told there is no answer. If you cannot supply me with an answer, I will find someone who can!

So here I am, running to see Regillo every six months for the past two years….and I think I am getting closer!

First the good news/bad news. Or, in this case, bad news/good news. Although I find it hard to believe, I have fallen down below 20/400 acuity. Bizarre because I don’t feel blind and don’t think I function as a blind woman.. After all, I got the “you don’t look blind!” routine just last month.

However, the good news on that is my vision is now so bad, I can satisfy the truly awful vision requirement for the Astellas’ study that is supposed to launch in March! Regillo referred me again. Is this time four or time five? I have sincerely lost count. [Lin/Linda: I put the details to that study in Sue’s page The Waiting Game.]

He has also put me on the list for APL-2 which is supposed to go into phase 3 clinicals sometime in 2018. He started to offer me “something else” when we were talking about lamp stuff (which is apparently very dead in the water) and I surprised him by knowing exactly where he was going. Working on being memorable. I want my name at the top of the list. [Lin/Linda: Sue wrote about APL-2 in her page My Friend in Manila?]

Also thinking I may be getting closer because I got a new test today. They ran me on the autofluorescence test. This test uses a very bright light. When the image is examined, the areas of the macula that are already dead are black and the areas that are in distress shine. My eye probably lit up like a Christmas tree. If that gets me into a study and gets this stuff stopped? Good.

So, that is where we are. I got a new test. I chose to be hopeful it means they want more information for my new career as a lab rat! Regillo generally seemed positive. Maybe I will be going to Philadelphia.

Written Dec. 14th, 2017 Continue reading “My New Career?”

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Progress Daily

I really cannot win with this transportation business. Now they are on a string of late pick-ups and I am waltzing in 45 minutes after everyone else. Grrrrrrrrrr!

Oh well, can’t fix it. Time to start a page…..and as soon as I am into it, they will show up. Same concept as going to the bathroom in the restaurant to ‘make’ your meal arrive faster.  Aha! Recognition! You have done that, too!

PRELUDE, the study, is NCT02659098. I checked and this is the same study I put my name in for last year. I just shot off a message to my research contact and asked her to make sure my ‘registration’ is still good. I am nothing if I am not persistent. Sad to say it is one of my better traits (oh no!)

There are actually two, main measurable outcomes they are interested in. There are the efficacy of the delivery system and best corrected acuity after administration of the stem cells. In the clinicaltrials.gov post they refer to the stem cells as CNTO 2476. In other literature they named the stem cells Palucorcel.  I guess it is better than George (with apologies to the royal family. I have never liked the name George, although the little guy is a cutie!) Of course, Palucorcel does not exactly fall trippingly off the tongue.

Anyway, according to a one page write-up by Jessica Lynch, previous attempts to circumvent the vitreous and go in subretinally caused too many problems. They are, as I had been led to believe previously, trying to go around to the macula using the suprachoroidal space as their passage. (Anyone ever see Fantastic Voyage? I keep thinking how incredible it would be to jump in my microscopic submarine and motor through the suprachoroidal space!) After preclinical trials with mini pigs were successful, they launched into prime time with a phase 1 trial with people. As I said, they are now recruiting for phase 2. [Sue wrote about subretinal and suprachoroidal are in the previous page: Secret Passages in the Eyeball

Looking at the additional data on clinical trials.gov I discovered there are secondary outcomes for the study. They will be looking at quality of life and reading speed as well as whether the stem cell transplants slow or even stop the growth of the geographic atrophy. They are also looking at how many people convert to wet AMD. It sounds as if this study would be a long term commitment for the ‘lab rats’ chosen.

Going back to the Medscape article about phase 1, I discovered they had pretty good success threading through the space and the transplanted cells grew and started to function.

Cell placement was important. They used the microperimetry to figure out what retinal areas the subjects were using for eccentric viewing. Too close and that could be messed up. Cell placement other places was better.

Results? The subjects had some improvement in vision. That was SOME. Before you get too excited, remember this is RPE replacement. RPEs do not see. They support your photoreceptors. Some of the photoreceptors that are at death’s door may come back but the dead ones stay dead.

I did run off the journal write-up on phase 1 and I promise to tackle it and see if there were any other cool findings. Later. Right now I have laundry to sort. Maybe listen to an NCIS episode. It is now playing all the way through on my tablet!  What can I say? It really is the little things.

Progress daily, guys. Progress daily. We will get there.

written October 17th, 2017 Continue reading “Progress Daily”

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Secret Passages in the Eyeball

Back again. Chatty sort, ain’t I? ? I really thought I was done for awhile but I keep finding things; ya know? If (should I even use the word ‘if’?) you are tired of hearing me chatter, please write a page! We are anxious for more offerings. [Lin/Linda: if you haven’t read the pages by our guest authors, click here to see what we are talking about.]

Back to the topic at hand (I am not only chatty, but I go off on tangents, a lot of tangents??☝ ) the most recent thing to land in my inbox is a Medscape article Lin sent me. We are back to the stem cell research!

This article uses a lot of the concepts and vocabulary we have introduced over the months. I will review as we go along – I don’t remember it all myself! – and Lin might help us out with some links to previous pages. Pretty please and thank you.  [I think the best place to learn about stem cells is a short YouTube video.   Also, check out Sue’s early page Research.]

I am signed up for a Janssen Pharmaceutical study with stem cells. Guess who did this study? Do you ever feel LEFT OUT? Once again, I would love to know how to get on the A list for these things. Not sure all the places the study happened, though. One place was Kentucky. Maybe I was a ‘geographic undesirable’? I will have to do a little research after hip hop. (Some things do take precedence!?)

OK. Here we go. I looked up PRELUDE, etc and got routed to Clinical trials.gov. It is only going into phase 2 but the good news is they are recruiting. The better news is they are recruiting at Mid Atlantic Retina, Regillo’s place. Now I am getting stoked. “Watson!…The game is afoot!”

Shoot off an email later and get ready to accost Regillo when I see him in December. One other person who is only starting to realize the depths of my tenacity! (Cue Vincent Price laughter!)

OK. Enough nonsense….for now?. From the looks of things, PRELUDE was a study checking, for at least one thing, the feasibility of a delivery system. If you recall, we talked about ways of delivering stem cells to the back of the eye where they belong before. No just shooting them into the vitreous fluid where they roll around like loose cannon balls and end up who knows where. No, no, no! They must be placed. The way they are placed is to thread them through the subretinal space.

Now I am thinking the suprachoroidal space and the subretinal space are the same thing but I really don’t know. [The title of the article I linked to above is “Subretinal Delivery of Cells via the Suprachoroidal Space: Janssen Trial”.] How many ‘secret passages’ can an eyeball have? Be that as it may, the whole idea is to snake through this space (or spaces?) and get to the back of the eyeball without violating the integrity of the vitreous and opening it to infection.

The progress of the implanted cells in this study was checked with microperimetry. This appears to be an up and coming imagining technique we also talked about. It is a technique that tests which parts of the retina are working and how well they are actually working.

Which brings me to my word limit and bedtime. I have been to hip hop and back and need to take a bath and get ready for bed.

I did find what I think is the original journal article for this as well as the clinicaltrials.gov listing. I will follow up….tomorrow.

written October 16th, 2017 Continue reading “Secret Passages in the Eyeball”

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I Am Not a Mutant

This continues where page I Want to Be a Mutant ends.

I stand corrected. The genetic testing we had done by ArcticDx DID screen for complement factor I. I did not remember correctly. Mea culpa. I was in error.

Now the problem becomes this: I don’t have it. Everything else – OK, almost everything else – has one or two stars next to it. Next to complement factor I is a negative sign.

Unless I am also misinterpreting that, I would be a non-responder to ‘lamp stuff’. I guess someone else gets to be Storm. I am apparently not a mutant.

Not sure if that is good news or bad news. Reasonably sure being part of the lampalizumab study would keep me out of the stem cell study. I have always wanted the stem cells. I am also not excited about having a needle in my eye every month . Remember when I was talking about primal fears? One of them is bodily integrity. That is one of my biggies.

On the other hand, the stem cell study is taking forever to get off the ground. I could be effectively blind before they get their stuff together.

‘Tis a dilemma. Not sure why Regillo said he wants me on lampalizumab without knowing if I would be a responder. Not sure how much people without complement factor I actually responded. Not thinking it was much. Maybe he is just being a scientist and looking to experiment. I would not mind except for the needle part.

I really am not as brave as you wet folks; not at all. And that is especially true when I suspect I will get no benefit from it.

That said, you people who have had genetic testing, check your charts. The way Lin and I are reading them, the gene you are looking at is CFI, fourth from the bottom. If you have little stars, you should be a responder to lampalizumab. Take the chart to your doctor and inquire if I am accurate. Remember I have been wrong before, so check it out.

Of course, since ‘lamp stuff’ is only for slowing GA, you people with early AMD or wet AMD, won’t be candidates for it either. However, knowledge is power. If you have the gene and do progress to GA, you can respond quickly to get the right treatment.

You who have not had a genetic screening, hold on a bit. This is the first treatment for GA available and it is showing what looks like a relatively large difference in effectiveness across genotypes. I suspect insurance companies will start paying for genetic testing out of self-defense. [Lin/Linda here: Medicare HAS approved the ArcticDX testing.] I could not find frequency of the I allele quoted anywhere (and my eyes were crossing trying to read the genetics info, so I quit). However, I found the frequency of some other alleles associated with AMD to be about one in three. With two chances out of three the injections will not be effective in any given person, insurance companies will pretty much insist on genetic testing in the very near future. Meaning, my dears, they pay!

And that is that. I intend to have a talk with my local retinologist in August and Regillo in December. Those of you with research capabilities, keep an eye out for the phase 3 study journal article. I would like to read it.

And the journey continues.

written June 28th, 2017

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Reading Modern Retina

Never thought I would be skimming back issues of Modern Retina, but here I am! Let us get back to some of the science stuff.

Amyloid beta is a major component of plague found in the brains of those with Alzheimer’s. There has been some suspicion AMD and Alzheimer’s are related at a genetic level. A recent study completed by Cheryl Guttman Krader failed to show any positive effects of injecting an antibody that targets amyloid beta into the eyes of those of us with geographic atrophy.

For the time being this means this line of inquiry will be abandoned or re-worked. Proof of concept did not occur and these researchers might go on to investigate something else.

Why are negative findings good news? One less blind alley to investigate! Since we don’t know which ideas may bear fruit, they all have to be investigated. Eventually we get to only the ones that have the most promise. Scientific method.

And another reason I think this finding is good news? It sort of suggests the Alzheimer’s and AMD connection may not be so cut and dry. Phew!

Here is another failure in proof of concept. Aflibercept is called Eylea when it is used as an inhibitor of vascular endothelial growth factor (VEGF – read “one of the things that makes the extra veins grow in AMD”). Michelle Dalton tried implanting stem cells in the eyes of patients who had been getting Aflibercept. She hoped the stem cell would produce the natural vascular endothelial growth factor and make the shots unneeded.

Unfortunately, many more patients than she had hoped required rescue doses of the drug. However, she also had people who kept the stem cells alive and these imported new stem cells did produce some of the Anti-VEGF molecule. Quantities were just too far below a therapeutic dose.

While this may be a failed experiment on the face of things, it is not all bad. Knowing there was some production of the desired molecules means this procedure may be very helpful once they figure out why it worked the little bit it did. Magnifying that effect may lead to fewer injections.

Last one, David S. Boyer wrote a review on multiple strategies being investigated for treating dry AMD. While many protective strategies for our photoreceptors and RPEs have failed, one they are still looking at with interest is brimonidine, brand name Allergen. Allergen is once again an intravitreally administered drug. (That is needle in the eye. We appear to be destined to join our wet AMD friends in that fate!) Coming out of phase 1 trials, brimonidine looks good. Next for it is phase 2, proof of concept. Will it perform as hoped?

Glatiramer acetate is looking good for reducing drusen. Glatiramer is used to treat multiple sclerosis, a disease in which the immune system wears away at the covering on the nerves. The theory is that glatiramer acts as a decoy to mitigate the autoimmune reaction. This treatment is based on the idea AMD really is an autoimmune disease.

There has been some evidence glatiramer reduces drusen, but Dr. Boyer warned us drusen can become fewer on their own. Drusen regression.

And that is a topic for another page.

written June 26th, 2017

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BIG News!

Woke up with a start at 2 am last night. Probably several things.

First thing that happened was a call from one of my contracts. She had called my third place of employment to schedule an evaluation and was told I did not work there anymore!

News to me! Now, I don’t get there a lot but the plan was for me to go and do a case or two when called. Maybe something like once every six weeks or so. I was never told I was being fired!

Of course it turns out someone got something wrong but it did get me to thinking. Once again, how does one graciously bow out or – hopefully equally graciously – be shown the door? Inquiring minds.

The second thing that has me a little anxious is my big ‘field trip’ tomorrow. I am going to do some sightseeing on Manhattan with an acquaintance from school. First time that far away from home without my husband since my sight loss. I know it can be done, but it is still a little scary.

Third thing: I saw Regillo yesterday. My eyes are getting worse slowly. (I am not so sure about the slowly part!) He confirmed scotomata (aka blind spots) get darker but did not necessarily say they go black. He said that he would not expect a central vision loss to cover 60 degrees of arc. That wide a loss would be ‘extreme’. Those two answers at least get us slightly closer to settling two of my burning questions from this Spring.

The big news, though, is he wants to try me on lampalizumab next winter. It appears the phase 3 clinicals are going to wind down by the end of the year and phase 4 trials will be starting.

People, the numbers of subjects in phase 4 trials is BIG. HUGE! Phase 4 trials take place after the FDA approved the marketing of a new drug. The drug is made available to the public through local physicians. They look for effects and side effects in diverse populations.

What this means for you is simply this: the first actual TREATMENT for geographic atrophy may only be six months away! This is the first breakthrough!

Lampalizumab is an injectible drug. It has been proven to slow the progression of geographic atrophy and to “reduce the area of geographic atrophy” by 20%. Dosing occurs monthly or every six weeks.

Will I do it? Probably. I really believe stem cell replacement of RPEs is the way for me to go, but it is taking forever and I don’t have time for forever. Lampalizumab can be administered locally and would avoid lots of trips to Philly. I don’t like the idea of intravenous injections but I don’t like the idea of a vitrectomy either! A 20% decrease in disease progression might win me enough time (and macula!) to have a more successful intervention later.

If you have dry AMD and geographic atrophy, it might be worth your while to broach the subject of lampalizumab with your retinologist. Let him know you are interested. This could just be the start of something big for all of us.?

Continue reading “BIG News!”

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Sisters Are Doin’ It For Themselves

I have paid a lot of attention to the male movers and shakers in vision research. Perhaps it is time to note the contributes of the women. Recently I have come upon short articles about the research of two.

Sally Temple, SUNY-Albany, and her colleagues recently published a paper on how nicotinamide can suppress the progression of AMD. Nicotinamide is a vitamin B3 derivative.

Dr. Temple took pluripotent cells, that is stem cells, from people who had AMD and those who did not. She manipulated the stem cells to become retinal pigmentation epithelial cells and grew them in her lab.

One of the first things Temple and her team noted was the cells from the AMD people acted differently from the RPEs grown from healthy subjects’ cells. The cells from people with AMD produced different chemicals. The chemicals were the same ones that figure in the production of drusen and contribute to inflammation.

These were RPEs growing on a culture medium in a glass dish. There was nothing else to contribute to the formation of the chemicals. The chemicals had to be coming from the RPEs. And, with no other possible influences, the cause for the production of these chemicals pretty much had to be genetic.

The fault, dear readers, is not in ourselves but in our genes. One more tally in the genes are destiny column.

But the good news is, when they squirted (or whatever) nicotinamide on the offending RPEs, things improved. Chemicals that are responsible for the bad things were less and the RPEs survived longer.

Perhaps if we find a way to get nicotinamide directly into eyes, we will get the same results in vivo as in vitro. Worth a try.

Masayo Takahashi is a Japanese researcher. Takahashi has been experimenting using pluripotent cells taken from the same people they are going back into. No embryonic cells required.

There is excitement about this new procedure not only because of ethical issues. There are indications this procedure will be cheaper and faster to implement. In additional, they are thinking people can ‘bank’ their stem cells. These can be used either for ‘repairs’ in the original cell ‘owner’ or they can be given to other people who are immune matched. (Sort of like blood type matching. Don’t want the body getting up in arms over the ‘invading’ materials.)

Bottom line is the ladies are out there rocking it just like the men. They continue to come up with great new findings and each one takes us a little bit closer to effective treatments and maybe – just maybe – even a cure.

To copy Lin’s use of old song titles, “sisters are doing’ it for themselves”. And they are doing it for us, too! Continue reading “Sisters Are Doin’ It For Themselves”

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