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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Electrician’s Nightmare

Rods and cones. I learned those words back in the sixties in science class. We were studying the eye.

Rod cells are concentrated in the peripheral sections of the retina. They work well in dim light. They play a key role in night vision. While we need rod cells for good, overall vision, they have their limits. Rod cells are lacking in sharp vision and color perception.

That brings us to the discussion of cones. Cones are located most densely in the center of the eye. Their jobs are to see sharply in bright light and to easily perceive color.

Considering many of us have trouble with sharp, central vision and ‘wash-out’ in bright light, as well as have trouble with color perception, it comes as no surprise that we, those with AMD, are a little short on cone cells. If you have seen images of the ‘divot’ in your macula, that cone-shaped hole should be full of cone cells. Mine is not.

Because we are a bit short on cone cells, cone cells were what researchers were trying to grow for us. After a number of years the breakthrough came at the University of Montreal and was published in 2015. The head researcher was Gilbert Bernier. Merci, Gilbert!

Dr. Bernier came to the idea there must be something that helps to grow all those cone cells in the macula. After all, the embryonic cells are pluripotent. That means they are capable of becoming any one of several different cells. What makes it so these particular cells became cone cells?

Bernier discovered a protein that limited the stem cells to becoming pretty much only cone cells. He actually achieved about 80% purity, a pretty much unheard of accomplishment before this.

Even more exciting, Bernier’s cells organized themselves into nice, pretty sheets of retinal tissue. Not a disorganized mess.

And if that were not enough, when his cells were injected into the eyes of healthy mice, they migrated to exactly where they were supposed to be!!!! Stem cells with the homing instinct! Pretty cool. They were doing the happy dance in Montreal.

This is a huge step but not yet the answer to replacing cone cells, the photoreceptors we lack, and ultimately our vision. As I have said before, the cone cells grown in the lab are like cell phones without a tower. They do what they should do but have no way to send the signal to the brain.

According to the Discovery Eye article on the optic nerve and how it links to the brain, there are approximately 125 million photoreceptors, rods and cones, in the human eye. These connect to two, different intermediate neuron types and 23 different kinds of other retinal ganglion cells. Some of the retinal ganglion cells communicate with as few as five photoreceptors.

In short, the eye would be an electrician’s nightmare! It is no wonder no one has been able to figure it out yet.

There is, however, a way for it to be done. The knowledge of the body – remember these cells both organize themselves and migrate accurately – is miraculous. The next step is finding out how to give the connect order. I suspect the Montreal crew is working on it even now.

Bonne chance, Gilbert, bonne chance. [“Good luck, Gilbert, good luck” for those of us who don’t know French.] Continue reading “Electrician’s Nightmare”

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Kids and Zombies

This page is going to be another mixed up affair. I don’t have a full page on either of a couple of totally unrelated topics. Hope you don’t mind if I cobble them together.

First thought: I have talked about loving my big people and have not said anything  about my little people. Let me correct that.   I love my kids!

We may have an exceptionally tolerant and loving group of kids at our school, but I don’t think so. I think kids are just naturally adaptable and loving. It is the nature of the beast.

Anyway, my kids are wonderful. They take my telescopic glasses and CCTV in stride and think they are cool. My disability? Yeah, she doesn’t see well. What else is new? So what? Can we move on now?

The other day I was waiting for my ride, sitting in the office with all my gear. A little girl about seven was waiting for her mother. The little girl asked about my stuff and I first told her I have an ‘old lady’ eye disease. I think I am going to take this little darling home with me! Her response was “you are not an old lady!”

Then I told her I used the equipment to help me so I could continue to work with the kids at school just like I like. Her response? “Thank you”. OMG. I love my kids.

Second topic: Back to stem cells. Lin gave me an investment article. It was dated July, 2016. Mark Collins writing for marketexclusive.com suggested people invest in….drum roll, please….Astellas Pharma!

For those of you who just walked in, this is the company sponsoring one of the stem cell trials I am signed up for. I am thrilled to hear that this company is being touted as a good investment opportunity. It means the company is seen as a moneymaker. And how are they going to make money? By bringing the stem cell treatment to market, that is how!

Collins cautions this is a somewhat long-term investment. It may be two or three years until the product is brought to market, but the financial analysts are very positive about its future.

I am signed up for phase 2 clinical trials. If Collins is accurate in saying they should come to market by 2020, phases 2, 3 and 4 will have to come about in pretty short order. Good.

One more quick note: please remember RPE stem cells may stabilize vision. That means things won’t get worse but they won’t get better either. There is a bigger maybe for restoring some vision. Subjects in phase 1 got a few letters (not lines) on the eye chart back. RPE stem cells do not cure AMD.

Geographic atrophy, aka advanced dry AMD, means I have dead photoreceptors. Dead is dead. No zombies are produced in this procedure. That means it is not a cure. They are working on a cure but it is not expected for at least 10 years.

[For a review of what RPEs and photoreceptors do, see Sue’s page The Science Stuff.]

As always, I often do not know what I am talking about, so please check the stock tip with your broker….but if you make a billion dollars? Remember me! 😎 Continue reading “Kids and Zombies”

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The Cockeyed Optimist

One of the books nephew #2 considered necessary for educating Aunt Susie was Steven Johnson’s Where Good Ideas Come From. I listened to it and it came to mind today when someone insinuated I was perhaps naive to have such faith in stem cell research.

I will eternally be a cockeyed optimist but I don’t believe I am unrealistic. Johnson has a concept I call “you can’t get there from here” and he refers to as the adjacent possible. Have you ever wound your way through a large museum? You cannot get from the marine invertebrate section to the land vertebrate section. They don’t connect. However by traveling through marine vertebrates, you find your way. Same concept. We are not in the stem cell cure ‘room’ and we cannot get there from here. But if I can help the researchers get into the next room, maybe even the one next to the stem cell cure room, I want to help.

Truth of the matter is, astounding leaps and eureka moments are rare. It is important to support the people finding the slightly closer rooms, one room at a time.

A reason to be optimistic? The bigger the network the faster the innovation. The more people working on a project, the faster dead-end lines of inquiry are abandoned. One person’s findings spark an idea in someone else. We are climbing onto each other’s shoulders to reach higher and farther than any other time in history. Open exchange of ideas is spurring us ahead at an incredible rate.

Johnson even reports error is necessary for growth. This goes way back to genetic mutations. How boring the world would be if everyone were identical.

Variation through error allows for variations in thinking and even serendipitous events. Knowledge advances through errors almost just as much as it does through linear, accurate discoveries.

I appreciate people are concerned about me. I really do. I like when people want to protect me. It is a nice, warm fuzzy thing. But I am not going into this with crazy, pie-in-the-sky expectations.

My hope is to stop the progression of the disease in one eye. The phase 1 subjects got some acuity back because dying photoreceptors were revived. That would be nice.

After that I am on the 10 year plan. Vision in 10 years is my tentative goal. Might happen. Might not. I am cautiously optimistic.

Did I choose wrong with stem cells? No. There is another author my nephew recommended, Malcolm Gladwell. The book Blink by Malcolm Gladwell refers to a concept known as the adaptive unconscious. The adaptive unconscious makes decisions without conscious contemplation. It is knowing without knowing how you know. Some people call it intuition. Gladwell has discovered the collective unconscious is very frequently right. The adaptive unconscious was how I first came up with my plan.

Hold it to the scrutiny of the conscious? Of course! Looking at the pros and cons of everything – repeatedly as situations change – is necessary. After all, I am not naive 😜.

Have a great day!

Love, the cockeyed optimist Continue reading “The Cockeyed Optimist”

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The Waiting Game

Yesterday was my third appointment with Regillo. Quite frankly I was hoping for great things. Hoping I would get a definitive answer and it would be positive! No such luck. My ‘answer’ was another “maybe”.

What criteria are they using? No clue. I was told my eye condition certainly qualifies me. Beyond that, I got no inkling of what I need to do to move up the list.

After a year and a half, I am getting more and more frustrated and antsy. If there is a way to become a prime candidate, I don’t know what it is.

Anatomy of the eye-click on the image for more information

I did learn a few things. Contrary to what I read, the good doctor says everyone has a suprachoroidal space (SCS). [Lin/Linda here: the SCS is the space between the sclera (outer part of eye) and choroid (space below RPEs).  It’s important in both clinical studies Sue is referred to because both insert the stem cells into this space; more about the clinical trials below). Not sure why the difference between what I read and what he said. I know I read something about ‘forcing’ (my term) an SCS in guinea pig eyes. They did it by injecting saline solution between the appropriate layers. Maybe the difference is between having a space and having a medically useful space? I might be wrong but I got the impression the delivery system works better when there is fluid in the SCS. Maybe not. Anyway, everyone has one. I am just not sure if you need the fluid to accommodate the delivery system. If you really want to know, check with your eye doctor. I am still trying to piece this all together.

The next thing I found out was the Ocata/Astellas study may resurrect sooner than I was originally told. I had heard two years or more and now I am being told 2017. Sweet.

I was asked which one I preferred. The one I would prefer is the first one to come to fruition! I will be dancing in the streets to be asked to participate in either one of them.

So that is where I stand now. I have been given two strong maybes. Is that a guarantee I will get something or do two nothings equal nothing? It is driving me crazy!

So back to practicing my distress tolerance skills. I have to ACCEPTS my situation. Engage in activities and contribute to others. I have to compare my situation to those of others and be grateful; things could be worse. Doing things to laugh will help me to have opposite emotions and I can push away problems I cannot solve at present. I can also have pleasant thoughts and intense sensations that distract me from my frustrations. It can be done. I have done it for a year and a half. I can keep on.

written 12/16/2016

Continue reading “The Waiting Game”

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Beware Snake Oil

Happy Sunday to you! Lin and I have been emailing back and forth. Since I have another glimmer of hope about the clinical trials, we have been talking stem cells.

I am very optimistic about stem cells. I have known from the instant I saw the Wills studies on clinicaltrials.gov that it was the way I would be going.

I have not wavered in nearly a year and a half and I am not wavering now.

That said, do you remember when I talked about the three states of mind? I try very hard to stay in wise mind on this issue. Wise mind is the melding of emotional mind and reasonable mind. Emotional mind fuels but reasonable mind guides.

Going online we have noticed a lot of what I can only call testimonials to amazing new treatments. These popular press articles all talk about one person – that was one, 1, uno – person who has recovered his or her sight due to this miraculous, revolutionary new procedure, whatever it might be. To me, it seems stem cells have become the new snake oil.

OK. Now some of you just got your hackles up. Calm down. Sometimes snake oil worked. Not saying it did not. However, until it was evaluated pretty thoroughly we did not know a great deal about what it was, what it could do including harm, etc. Lots of harm has been done in the name of treatment.

For example? Blood letting. A president no one ever thinks of, Rush, was killed by bloodletting. It was an acceptable practice. I don’t imagine many of you have had blood removed to cure a disease but I am guessing you may know one person who has. Bloodletting is a proven treatment for Polycythemia Vera, a condition in which there are too many red blood cells. A treatment may have a beneficial use. We just need to find out what that use is. And we need to do it scientifically…at least in my not so humble opinion.

I am cautiously optimistic about stem cells. I have an internationally known doctor at an internationally known facility. The two studies I am signed up for are funded by large, reputable corporations. I even have the informed consent documents already!

What about you? First of all, I would like to think I could influence you to only go someplace with the credentials Wills Eye Hospital has. If that is not possible, ask questions and do your homework without committing to anything. Remember desperate people are vulnerable people. Also, even if the people you are dealing with are decent human beings, their theories or procedures could be faulty or simply not right for you.

What questions to ask? The American Speech, Language and Hearing Association – of all places – published a very nice list (with extensive links; gotta love those speech teachers!) in What to Ask When Evaluating Any Procedure, Product or Program. Read this. Take a copy with you and ask the questions. If you don’t get decent answers, turn around and walk out. Use your desperation, fears, hopes as the fuel but let your reasonable mind do the steering. Stay in wise mind on this one, guys.


Lin/Linda here:  Sue wrote this in December, 2016.  Yesterday (March 16, 2017) an article was published in the New England Journal of Medicine about 3 women who became blind after stem cell injections for which they paid $5,000 from an unnamed clinic in Florida.  The news spread fast and articles were published and widely shared by large news organizations (NPR, CNN, the major TV stations ABC, CBS, and NBC and the New York Times), by macular degeneration organizations (The Macular Degeneration Partnership, Macular Degeneration Association and The Macular Society in the UK) and by several professional organizations for those in the field of vision & eye care.

What went wrong?  This article Three people left blind by Florida clinic’s unproven stem cell therapy says it best from what I’ve read:

  • First there is almost no evidence that the fat/blood stem cell combination the clinic used could help repair the photoreceptor cells in the eye that are attacked in macular degeneration.
  • The clinic charged the women $5,000 for the procedure. Usually in FDA-approved trials the clinical trial sponsor will cover the cost of the therapy being tested.
  • Both eyes were injected at the same time. Most clinical trials would only treat one eye at a time and allow up to 30 days between patients to ensure the approach was safe.
  • Even though the treatment was listed on the clinicaltrials.gov website there is no evidence that this was part of a clinical trial, and certainly not one approved by the Food and Drug Administration (FDA) which regulates stem cell therapies.

 

Continue reading “Beware Snake Oil”

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