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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Serious Work

ARVO is the Association for Research in Vision and Ophthalmology. According to its website it is the largest and most respected eye and vision organization with over 12,000 members from 75 countries.

Basically, ARVO is hot stuff in the vision world.

The 2017 ARVO conference will be at the Baltimore Convention Center starting May 7. If we don’t get this page published for another four months, the 2018 conference is in Honolulu. We could always go there!😎

If you cannot get to Oahu, the ‘Big Island’, for next year, you can always view abstracts of presentations from past ARVO conferences online. There are sections on a variety of eye problems, one of them being retinal concerns.

The first abstract included under retina from 2016 talked about how damaging scar tissue in the eye can be for vision. While anti-VEGF drugs serve to halt the growth of extra blood vessels, they do not prevent the growth of scar tissue. Researchers are working on producing antibodies that will react with the connective tissue growth factor in a protein that contributes to scar tissue formation. This research is still in the (real) rat labs, but may someday be given to wet AMD people with bleeds. Remember bleeds can lead to scars.

The second one mentioned – oops, actually the third – talked about the lack of plasma diagnostic markers for AMD. Plasma diagnostic markers can be found in blood. Someday there may be a blood test that will allow us to determine who is going to start with wet AMD and prevent the growth of new blood vessels even before it starts.

Other abstracts included in the retina section talked about ways of improving ‘bionic eyes’ to provide more details in the images and, yet others, talked about the research being done with stem cells. While the stem cell, clinical trials I am interested in are dry AMD only, there have also been phase 1 clinicals using wet AMD patients. The researcher there, Zheng Qin Yin, reported vision improvement just as was seen in phase 1 clinicals with stem cells and dry AMD. Bonus!

In some studies the stem cell source was embryonic but in other studies they are using bone marrow stem cells. The bone marrow people are also getting promising results.

Never heard of subretinal fibrosis, but apparently someone is studying it! Philipp Roberts to be exact. Not sure why Philipp has two P’s but that is the way he is listed. Anyway……Philipp tells us subretinal fibrosis is the end stage of wet AMD. Apparently you cannot tell the difference between “regular” neovascular tissue and subretinal fibroid tissue with standard imaging techniques. Philipp is working on that.

Basically, there is some serious work being done to hopefully get us out of this mess. I have not said this for a while but I can say it here: now really is the best time in history to be going blind.

And if you are in Baltimore the week of May 7th? Thank a vision researcher for his or her effort. They should be easy to find. The website predicts 11,000 in attendance! Continue reading “Serious Work”

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Clinical Trial Design: Part 1

The Hypothesis

All good experiments start with a hypothesis. A hypothesis is an educated guess about what should happen under a certain set of conditions. For example, “stem cells can grow in the eye and replace worn out RPEs”.

The way I was taught, this statement is then ‘turned around’ to be the negative. “Stem cells cannot grow and replace worn out RPEs”. This ‘turned around’ statement is called the null hypothesis. This is what will or will not be disproven with our experiment. The idea is to see if we can generate a result that will be significantly different from the starting point. The null hypothesis predicts no change. We want to see if we can affect a change that is big enough to matter. Good so far?

The Plan

After deciding what we want to figure out, we need a plan. Our plan is our experimental design. We want to design our experiment so we are reasonably sure there are no stray influences that may have caused the results.

 

The groups

If we are comparing treatments or treatment versus no treatment, we need to make sure our groups are as similar as possible. If we want to see if our new exercise program is better than another training program we really would (ethically) not want 20 year olds in one group and 80 year olds in another.

This is part of the reason good experiments on clinicaltrials.gov list exclusions which means reasons why a person couldn’t be in the trial. They are looking for similar subjects. Another reason for exclusions may be related to the treatments they are using and people’s sensitivities to them. You don’t want a subject who has an allergic reaction to your treatment!

The Sample

Once we have our sample (all possible examples or cases of something is called the population), we should be reasonably sure we have controlled for most of the important variables. Variables are factors that can affect the outcome of our study. However, there are other variables we have to control for in our experimental design.

The Treatments

One of these is the Hawthorne Effect. Anyone who has ever taken intro to psych or industrial/organizational should remember the Hawthorne Effect. In a plant they were trying to increase productivity. The problem became, no matter what they did, production improved! These people could have been working in the dark with the heat turned up to 90F but they were going great guns.

The researchers discovered just the fact that they were noticing the workers and trying to do something for them was enough to increase productivity. It did not matter that working conditions worsened. The workers felt noticed. They wanted to please the researchers.

Placebo effect happens when subjects believe the ‘treatment’ is effective even though it is not. They may show improvement as a result of their belief.

You will see attempted control for the Hawthorne and Placebo Effects in studies that list themselves as ‘double blind’ or ‘double mask’. They will perform fake (sham) procedures on people in the control so they feel noticed and loved, too.

Double blind? The double is because doctors and nurses treat people getting the actual treatment differently. They may not realize it but they may be more encouraging to those actually getting the treatment than they are towards the people who are the controls. When a study is double blind neither the patients nor the people caring for them actually know.

That is my 500+ words for this one. One or two more pages and I should be done. Hang in there!


For more information, click here for Designing Experiments Using the Scientific Method from dummies.com.

Continue reading “Clinical Trial Design: Part 1”

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In a Pig’s Eye

About three hours later and the ‘tapering’ snowstorm is not tapering. Anyone ever read ‘Ghost Story’ by Peter Straub? It scared my socks off on a summer day. On a day like today I would probably be quivering under the covers!

In ‘Ghost Story’ it starts to snow. They cannot keep the roads open and it snows. The electricity goes out and it snows. The phones go out and it snows. Eventually some smart soul figures out there is a malevolent force at work in this small, New York town. Yipes!

‘Ghost Story’ is on page 3 of the Gs in BARD. It is available as an audiobook on Amazon for $17.95. If you are still able to read print, you can get it used for about ⅕ of that price.

Another way to scare your socks off? ‘Turn of the Screw’ by Henry James. This one is a classic and free on Kindle. It is on page 15 of the Ts in BARD.

I will vouch for them both as excellent reads. Anyone else have any recommendations they would like to share? Just because we cannot see so well, doesn’t mean we cannot enjoy a good book. Since we like to think of our group as a cut above, try to avoid recommending trashy novels.  Although for a whole series of semi-trashy novels I would recommend the ‘….in Death’ series by J.D. Robb, also available on BARD.😀 [Lin/Linda: one of my favorites, too, but I don’t think I’d call it ‘trashy’ but definitely R-rated.   Click here for the list in order of publication date.  They’re available at amazon.com, too.  Click here for the first one ‘Naked In Death’.]

Anyway, that was NOT the way I was going to start this page. Not the topic either. I just looked outside and found it all a bit surreal. We are approaching an accumulation of two feet. Not much for some other places but impressive for Pennsylvania.

What I was going to do was tell you about “in a pig’s eye” and how the phrase now has a new meaning. For our international friends, “in a pig’s eye” is an old American expression that implies disbelief. It is the antiquated version of “No way!”

Now, they are finding a way to study drusen in a pig’s eye. Well, actually in a culture medium in which they have placed retinal ‘pig’-ment epithelium cells. (Alright, so it was corny, but I couldn’t resist.) They have found out that pig RPEs are similar in many ways to human RPEs. They have discovered the RPEs in early AMD are actually still functioning and the Bruch’s membrane may have more of a part in the process than previously believed.

This should just be the first of many good discoveries to come out of the pig’s eye experiments. Because they are now able to do a lot of manipulations of pig RPEs being grown in cultures, research can go faster. A lot faster than it would go trying to get people to have all these manipulations done on THEIR eyes.

So there is ever increasing hope here. When you tell people there is a bright future for AMD folks and they say “in a pig’s eye!”, your response can now be “Exactly!”

Keep on keeping on. There is hope.

Now could somebody stop this snow? Enough is enough already! Continue reading “In a Pig’s Eye”

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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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