Research Roundup: Treatments for the Future

BrightFocus Foundation
Research Roundup: Treatments for the Future
Transcript of Teleconference with Brian McKay, Maria Grant, and David Pepperberg
December 21, 2015
1:00 - 2:00 p.m. EDT

Please note: This Chat was edited for clarity and brevity.

GUY EAKIN: Hello everyone, welcome to our monthly BrightFocus Chat, which is presented by the BrightFocus Foundation. I’m Guy Eakin, the Vice President for Scientific Affairs at BrightFocus, and for those of you who have been loyal Chat participants over the last year you’ll notice that our format and our topic are just a little bit different this month.

So many people, as you know, use this time of year for reflection and taking stock of people or things that they are grateful for. On today’s Chat, we are certainly going to do both of those, but we are also going to take a look back at some of the ongoing research that’s happening out there in the world and get a glimpse of a few projects that we think are just particularly innovative and promising. We are very pleased to welcome three BrightFocus-funded researchers who are all involved in making advances in macular degeneration, and we are of course very grateful to them for their dedication to bettering the lives of people with macular degeneration. We are, as I’m sure they are all too, very grateful to everyone who is on the line today and to the donors all over the country who have made it actually possible to fund these researchers that you’re going to be hearing from.

With that, let me go ahead and welcome these speakers. We have Dr. Brian McKay, who is a professor at the University of Arizona, and his work has been in international news quite a bit recently because of a discovery of a drug used to treat Parkinson’s disease that may help people with macular degeneration.

We are also going to hear from Dr. Maria Grant, a clinician scientist from Indiana University, who is looking at how adult stem cells might assist in repairing damaged blood vessel networks in the retina.

We are also very pleased to welcome longtime friend, Dr. David Pepperberg, who researches methods to restore vision lost to diseases like macular degeneration and does so at the University of Illinois at Chicago.

So Brian, Maria, and David, welcome to the Chat.

SPEAKERS: Thank you…thank you.

GUY EAKIN: As you know, we host these calls monthly, and we do collect questions that our participants submit to us, and there are always one or two who ask about the hope for new treatments. Recently we had a question submitted by Melanie, who works with seniors in Colorado, which sums up that theme very well. She asks what research methods for AMD could be helpful to people in the next 5 years, as compared to the treatments that might be 20 years down the road. She says that she works with seniors, and they are doubtful that there will be any breakthroughs in their lifetime.

I would like to start to address that type of question today, and we are going to give each guest 5 to 10 minutes to talk about their own contributions. I would like to start with Dr. McKay, whose technology might be the closest to that 5-year horizon. Brian, you’ve had a busy few weeks, since your paper was published, and that paper describes that potential use of L-DOPA, which many people know as a Parkinson’s disease drug. Could you bring our participants up to speed on what that buzz is all about?

BRIAN MCKAY: Sure. We did a retrospective clinical study with about a quarter of the population of the United States. We had 87 million participants in this. If you’ve been to a doctor in the last 4 years and had a medical insurance plan, you’re actually in the database. It’s all de-identified. We just asked a very simple question. Are people who take L-DOPA protected from AMD?

Intellectually it’s a very simple question. We looked, and it turns out that the average age of onset of AMD is about 71 years old and we can—the people taking L-DOPA who got it, their average age was about 79 years of age. It looks like it’s protective. Then we looked at the odds ratio and the risk factors, and it turns out that many fewer people who take L-DOPA get AMD, so it reduces the risk of the disease. It looks like it can both prevent and delay the onset.

The reason we actually asked the question is because I’ve been studying the puzzle of pigmentation and retinal degeneration. It turns out that about 54 percent of white people, when they lose vision, they lose vision to AMD, but only 4.4 percent of black people lose vision to AMD. It’s very strong racial bias here for this disease, and it extends to Hispanic people too, so it looks like pigmentation and race have something to do with the disease. And it also turns out that glaucoma goes the other way: 6.4 percent of white people lose vision to glaucoma, but 26 percent of black people lose vision to glaucoma. So the number one and number two causes of blindness in our country are somehow tied to race and pigmentation, but how, we don’t really know.

That’s what my lab has been doing, and we know that actually pigmentation is important even earlier on during retinal development. If you have albinism, the lack of pigmentation in the tissue that supports the retina—called the retinal pigment epithelium RPE—lack of pigmentation there, you don’t really get proper retinal development, and what they actually lose most of the time is variable, but is actually the macula. It’s almost like a genetic macular degeneration. Albinos frequently don’t actually get that structure in their retina, so we know that somehow pigmentation is tied to this.

What my lab has done is we actually made a kind of an interesting model system where we made pigmented cells from the retinal pigment epithelium and then compared them to non-pigmented cells from the same donor, same tissue, and we investigated how they interacted with neurons. We discovered that the pigmented cells make a lot more of a very important protein called pigment epithelium-derived factor (PEDF), one of the most potent neurotrophic factors in the eye, but also in the body. It’s also a very potent antiangiogenic, so it turns off new blood vessel growth while supporting the retina.

We’ve discovered that that is tied somehow to the pigmentation pathway, so we went after that and tried to figure out how. We discovered that there is actually a receptor in the retinal pigment epithelia that binds to an intermediate of the pigmentation pathway, which turns out is L-DOPA. So there is actually an L-DOPA receptor.

When people take L-DOPA for Parkinson’s disease, it’s actually given to them because it can cross the blood brain barrier and it gets dopaminergic neurons where it’s actually converted to dopamine. But in the eye, that’s probably not how this is working. There is actually a receptor for this molecule that we discovered, and when we interact that receptor with the ligand, we control PEDF. If I take that ligand and I add it to retinal pigment epithelia cells, I can control their ability to support the retina.

This may actually tie back to albinism, which we have in our second stage of a clinical trial. It may go back to being able to prevent AMD—this morning I was working on putting together a clinical trial for that. It may actually even tie somehow to glaucoma in the end. We don’t know that one quite as clearly yet, but by looking at this, we actually found in three different data sets from the Marshfield Clinic to two nonintersecting data sets there, and then the market insurance claim database. We got kind of the same answer in all of it. We get AMD—if you take L-DOPA, AMD shows up at 79.3 years of age; without L-DOPA, it’s showing up at 71.4 years of age.

An interesting thing that we also found was that people who take dopamine agonists, they usually largely are kind of selective for the D2 receptor family of dopamine receptors. They were actually protected—not as strongly—but they were protected, and this could be because the receptor we found that binds L-DOPA, it actually interacts with dopamine too. So the selective agonists that we’ve created that hit D2, they also hit the GPCR that is the receptor for L-DOPA.

GUY EAKIN: As you tested this, one of the astounding things to me is that you’re in an area where something that has been a basic science—as that 15, 20 years ago, might have taken us 10 or more years to even think about getting into a clinical trial—you were able to really rapidly move into asking questions about humans using these medical records. What was that process like? How many medical records did you hit in that process?

BRIAN MCKAY: 87 million, a quarter of the population. It’s not a small study. When you look at—I’m looking at the graphs—you can’t even see the SD bars, they are so small, so yeah, it’s big. These huge data sets allow you to mine for things where overall in the data sets, 5.4 percent of the population in the data set had AMD or had a record of AMD and only 1.5 percent had Parkinson’s disease. So it takes a tremendous number of individuals to actually see 1.5 percent of 5.4 percent and then ask for age.

GUY EAKIN: At the same time I hear that one in 20 people in our medical system have AMD, which speaks to just how prevalent a disease it is.

BRIAN MCKAY: That’s very true. It’s a very prevalent disease, but when you try to mix two diseases together, it gets…Parkinson’s, although it’s very common, it’s only 1.6 percent of the population. When you take one in 20 and then you take 1.6 percent of that, it takes a large data set in order to see anything.

GUY EAKIN: Well, that’s really exciting. What are the next steps specifically about this idea that L-DOPA—well, it might be doing something in terms of delaying or even preventing macular degeneration, so what are the next steps for you and your research group?

BRIAN MCKAY: I was on the telephone this morning with Murray and some other people; we are working on putting together the clinical trial. It needs the prospective clinical trial. I think that with Murray’s genetic information and his ability to select the people that are at the greatest risk, we can shorten up the timeframe for the trial: rather than being 7 years, which is kind of what NIH and NEI think is right, we should be able to shorten this up to many fewer people genetically by saying, “These people are at very high risk of developing this disease and so are these,” and then put those two groups either plus L-DOPA or minus L-DOPA and get through the clinical trial faster.

GUY EAKIN: Well, obviously it’s just a really exciting line of work. I wish you the best on that as you begin that clinical trial.

At this time I want to shift the conversation over to Dr. Grant. Maria, do we have you on the line?

MARIA GRANT: Thank you. I would like to just mention a few things about the work that we are doing. Everyone’s body has an amazing regenerative capacity and, unfortunately, as we age, that capacity is reduced. At my lab, we are focusing on strategies to try to enhance the reparative capacity—to enhance the ability of the body to heal itself and compensate for factors such as aging and stress.

What we have studied and been able to show is that the bone marrow, which is a source of reparative cells, has tremendous capacity to release cells following certain types of stimulation. By putting these cells into circulation, then they can go and repair damaged tissues.

These damaged tissues can represent the blood vessels of the retina, they can represent cells such a retinal pigment epithelial cells—cells that are the target of what Brian was just talking about—the receptor for L-DOPA. These cells have a tremendous ability to help the reparative process and they do this by primarily providing factors that support injured or compromised cells. These factors will show that cell that it needs to become healthy again.

To take many years of research and condense it and make it more understandable, there are certain things—even exercise—have a way of mobilizing these cells into circulation. It also shows the importance of things like exercise, as one gets older, to maintain the natural comparative capacity. We are currently doing a study using acupuncture that stimulates certain parts of the central nervous system to release these cells into circulation. There are ways that are not invasive or particularly harmful, or cost as much money as drugs. We are not using pharmacological approaches but more natural approaches such as acupuncture and exercise.

What we are able to show through exercise and acupuncture studies in rodents is that these cells can increase and can leave the bone marrow and leave other sources where these cells all live dormant. They are released into the blood to be able to go out and repair. What we have been able to show is that you can increase, by 300 percent, the levels of these cells in the blood by these simple things such as acupuncture and exercise. But it is intense exercise. The idea here, once again, is that if we can restore the regenerative capacity then we can potentially prevent diseases such as age-related macular degeneration or diabetes retinopathy.

GUY EAKIN: Maria, your work is really far-ranging. You mentioned the macular degeneration and diabetic retinopathy, and you sort of bridged from working in this world of adult stem cells. So, these are stem cells that one finds in the bone marrow as a possible therapy or solution to these visual disturbances, but you bridged that with the really interesting things around acupuncture and exercise.

We have one question coming in, Jackie from New Jersey, asking about saffron, and you know you’re a clinician, I’m sure you get asked about nutrition any day of the week, and maybe saffron is not something I’ve heard so much about in terms of macular degeneration, but from the standpoint of when you look at the literature around acupuncture or around nutrition or exercise, as you mentioned, what do you think people should keep in mind as they are looking at those studies? How do you decide what’s—when you hear something out there in the news, what should be your red flags, what should be your green flags on whether to move forward with those?

MARIA GRANT: That is a great question. I think there needs to be some level of rigor. Has there been a clinical trial, or has there been basic research to support the actual benefits? It can be very difficult, as Brian alluded to, that these trials can take a long time. There are animal models that can emulate the key aspects of a human trial. If there is a particular study, or if there isn’t a study, then that may be a point to put the materials in context. Is there research to substantiate the claims that are being made by the person writing the article or by the group that is putting that concept forward?

I think a really important idea to keep in mind with regards to interpreting studies is, are they done by researchers that are funded? By agencies like BrightFocus or the NIH? And have they been through the process of what we call peer review? The research, even before it started, has been reviewed by colleagues to assess if it is reasonable. In addition, if there is a paper that comes out substantiating the concept, if it is beneficial for AMD, that is important too. Once again, that is the process of peer review, where other scientists have critically looked at the work and assessed it. To be able to get funded, to be able to do the research or to get a manuscript or paper, it means that that work has gone through that process, which is really important.

The other thing is that if you are reading the article, does it make sense? Does it capture some thought process that is logical? I think that with all of these resources that are popular right now, there may not be a study. There may not be research that substantiates it. My own lab has been interested in acupuncture. That, interestingly, has been around for centuries, but the mechanism has not been well established. One thing that we have been able to do is show that behind all of the history, behind all of the centuries, is show that there are actual mechanisms, that there are certain points we can use.

For example, the certain points that we use in acupuncture to stimulate the release of stem cells are points that activate the sympathetic nervous system. We know from other studies that the sympathetic nervous system is responsible for the release of stem cells. It is sort of a logical progression. I think when you read papers, whether they are in the lay press or in scientific venues, the logical progression is an important thing to keep in mind. 

GUY EAKIN: That’s amazing, and I don’t think that before speaking with you today that very many people would link acupuncture to the release of stem cells. These are things that certainly we hear a lot about in our own private lives, but to know that there is a science going on behind that is really quite extraordinary, I appreciate you taking the time to describe that to us. You started to get into the process of science, and that’s important. I know that at BrightFocus we helped you with an ambitious grant to look at some of these early stem cells, and you went on to take some of that data and take those bone marrow stem cells and go and get a NIH grant. Can you tell us about what that is and where that NIH grant is taking you next?

MARIA GRANT: Ok, sure. What we are doing is we are taking human cells from individuals that have age-related macular degeneration—and, so, patients that have the disease—and we are looking at their stem cells to see how vital they are; their vitality. Is there a difference in individuals that have age-related macular degeneration versus healthier adults? The idea is to look for an intrinsic defect.

That is one question. The other is that we are able to, by genetically modifying the bone marrow stem cells, we are able to guide its direction and prompt it to become a certain type of cell. What we have been studying—this is all animal model work—is we have been able to show that with the right type of modification, they can become retinal pigment epithelial cells. But they do it in a very interesting way. The stem cell is programmed while it travels from the blood toward the eye, toward the back of the eye, it is kind of thinking about becoming the cell. When it gets to the injured area in the eye that the environment in the stem cell is attracted to, then it helps differentiate. It helps the cell become the retinal pigment epithelial cell in a staged process. The cell has a genetic cue to become that cell that we artificially gave it, and then the environment of the injured retina takes the cell directly away.

It is a combination approach, where the context in where the cell needs to go helps provide the stem cell with the information it needs to become a retinal pigment epithelial cell. This is the process that we have been examining in the NIH grant, and the idea is we use mice that are humanized, in the sense that they don’t reject the human cells. We are trying to make them closer to what could potentially in the future be done in patients. This is obviously nothing that would happen soon, but the type of thing that in the future, by using the mice that can accept the human cells, would put us just a little bit closer to the idea because we are actually taking human cells and manipulating them and driving them to become a new cell and replace a cell that is injured in the back of the eye—the retinal pigment epithelial cells.

GUY EAKIN: Thank you so much. And I think we hear so much today in the news about personalized medicine. This is one of those places where we are looking at an idea of taking a patient’s own adult stem cells and nudging them in the direction of becoming a new tissue that would be therapeutic and would be a replacement for the cells that are being lost in macular degeneration. Thank you so much, Maria, for giving us some time to talk about these ideas.

We’ve been going through a wild world of L-DOPA, a Parkinson’s drug. We talked about adult stem cells and exercise and acupuncture. I want to move on to hear from Dr. David Pepperberg. We are excited to hear about his work that is also in the news in different ways. We hear the word “prosthesis” and we talk about artificial legs and artificial joints, and we have this concept that they might be electronic or made up of sophisticated plastics or metals. But David, your project is one of the ones that we supported, and it was to develop something that I’ve heard you call a chemical prosthesis. That fascinated me, so can you give us an idea of what chemical prosthesis is and how that’s going to work in the eye?

DAVID PEPPERBERG: Sure. I would be glad to briefly summarize what we are up to in that project.

Before I give my little summary—I’m sure I speak for Maria, and Brian, and all of our colleagues in the research area—what a wonderful job BrightFocus is doing in networking occasions like this. I mean, I think it’s obvious to the whole world what our passion is, in driving this science forward. BrightFocus is really head and shoulders above so many other operations in really being so proactive to bring together those of us who are in the science and those of us who are hoping to develop these new cures. It’s just terrific. I just want to thank you.

This project you are referring to, Guy, is one that, yes, we can think of this as a chemical prosthesis or other context. When we hear the term “nanotechnology”—that is the construct of molecular-sized structures that we and others are using, in this case to develop what we hope will be therapies for restoring vision that’s lost in advanced stage macular degeneration.

You mentioned, Guy, electronic prosthesis—that your listeners know are already in their first generation, and are available. Patients have been treated. But the great limitations of these electronic prosthesis is simply that you cannot get what we call the spatial resolution—the ability to achieve the sharpness, the acuity that the normally functioning retina has, due to the limitations of electronic circuitry. What I and my colleagues have been working on is to design, to create new molecular-sized structures that we can specifically interface with what we call inner cells of the retina.

Now, what do I mean by that? Macular degeneration primarily targets—as we’ve heard already in today’s conversation—primarily targets the retinal pigment epithelium, an important tissue that specifically supports the function of the photoreceptors of the retina (the rods and cones). And when one or both of those two tissues is under attack and deteriorating and dying, you have a situation, as we now know, where cells of the retina to which the photoreceptors ordinarily communicate their visual signals in a healthy retina often remain potentially capable of function, but because of the macular degeneration disease, what we call the upstream—the start of the visual process—can no longer send appropriate signals to those inner cells of the retina.

What we are doing is to develop molecular-sized structures that we can simultaneously target to specific cell types of the inner retina—retinal ganglion cells are the key target cell type here, because it’s the ganglion cells that represent the output of the retina—which ordinarily send visual signals to the brain. What we are trying to achieve is to be able, with a suitable introduction of these agents into the eye, to target them to bond to key proteins on the retinal ganglion cells. These proteins make those ganglion cells directly, in response to light, able to fire or to generate their electrophysiological signals that we hope can mimic those that, in the healthy retina, would be coming from the rod and cone photoreceptors.

The overall idea, in other words, is to be able to—if you think of a bypass on the highway where there is an accident on the road, the workers have arranged a detour to leave the highway and then come back on at a later point—the idea is to bypass the problem, the dead and dying photoreceptors in the retina and to bypass functionally the visual process by making the downstream—that is, going around the problem and making the downstream output cells of the retina, the retinal ganglion cells, directly responsive to light.

Our work is at a stage—we are very excited about it, and we’ve had what we think is good progress in the cell types that we have been testing these structures. We are at the point now of applying this in animal studies to retinal cells themselves where the photoreceptors have been by genetic means or other means impaired, and which can serve as a good model for the disease.

So, as I say, we are very excited about this. If we can make this work, we think it offers, potentially, the hope for the restoration of at least a good degree of vision by making those retinal ganglion cells that themselves are not at all directly involved in light absorption directly responsive to light and able to generate signals that the brain can interpret as a visual thing.

GUY EAKIN: That’s really interesting. You have a situation where we have a disease where photoreceptors are dying, and you in some sense have said, well, maybe we don’t need those photoreceptors. Maybe we can replace them with this chemical that you’ve built. I think, one of these things—of the three technologies that we’ve heard about today, perhaps this one might be the most distant in the future, but one of the questions is, what do you think of when you think of the route of administration? How would this chemical get into the eye? Have you started to give thought to what this would look like when converted into not just an engineering project in your laboratory, but when it’s converted into that medicine that we actually think will be going forward in being in patients one day.

DAVID PEPPERBERG: Sure. That’s a very important question, and it’s one that we are directly working on. By analogy, let me say what I think is our hypothesized desirable route. That would be a direct injection into the eye of the patient of a small quantity of this active molecular structure that can seek out and bind to the ganglion cells as they need to do. There is plenty of precedent for injecting test agents in therapies into the eye as many of your listeners may know, for so-called wet age-related macular degeneration. The standard of treatment these days is injection by the ophthalmologist of one of several types of what we call Anti-VEGF therapies, and we are talking simply about the kind of route that we suspect may be workable of the agents that we are developing.

This is to say that these days patients come in all the time for such treatments, typically on a monthly, every other month, or several months basis. And of course there is active work to be further developing the efficacy of those drugs so that fewer delivery procedures are needed. But as you’re asking what can we envision right now as the starting point for introducing and delivering those materials, and there’s great precedence from the fact that safely and efficaciously for therapies like—therapies that are Anti-VEGF therapies are being done all the time for many, many patients.

GUY EAKIN: If you could say something briefly, one of the other areas of your laboratory—this isn’t the only hat you wear, but you also have some interest in areas of work that draw on knowledge from the field of Alzheimer’s disease, which is incidentally another interest of BrightFocus Foundation. You’ve been drawing on Alzheimer’s disease to see if that field and macular degeneration might be able to share some insights with one another to make some advances in both fields. Could you say briefly what’s going on there?

DAVID PEPPERBERG: Sure, exactly. This is the second of our two arms of focus here and is, as with the first, extremely exciting to us in a very recently initiated project that BrightFocus has been so generous in helping us to get off the ground. As many of your listeners may know, a molecule that we call amyloid beta, one that we abbreviate as Abeta—amyloid beta is a protein that for many, many years has been investigated in the context of diseases of the central nervous system, of brain diseases, notably Alzheimer’s disease as well as other brain neurodegenerative diseases. Now, there is a huge literature investigating the role of Abeta in brain diseases like Alzheimer’s, and it’s fair to say while much remains unknown today, there is little doubt that the dysregulation of this protein, when regulation of the levels of that protein in the brain becomes dysregulated and not carefully controlled by other processes going on in the nerve tissue, you have problems that most people believe are contributing substantially to the development of the disease in the brain—that is, Alzheimer’s.

Now, in recent years, and so far having been investigated only a tiny extent compared to the extensive work in the brain, there are indications in several respects that amyloid beta in the eye tissues may be contributing in certain ways to the development of a key neurodegenerative disease of the retina, namely age-related macular degeneration.

There is no doubt that amyloid beta exists in the eye tissue, the retina, and the retinal pigment epithelium. Again, we don’t know yet very much about how it works, but there have been suggestions from several lines of experiment that a deregulation of that important protein may be a player in very early progression of the problems that lead to age-related macular degeneration.

What we are doing in the BrightFocus-supported project is testing a specific enzyme, an enzyme that exists naturally in many tissues, but has not really been investigated for its potential therapeutic action when supplemented externally. This enzyme we call Neprilysin. When introduced into the eye, in our case of our experimental animals that have a problem with the dysregulation of amyloid beta, it may be able, by reducing the buildup of Abeta—that is, bringing back into an okay state, a situation that has gotten out of whack with respect to Abeta—that it may be able to delay or retard what otherwise may be the development of the disease due to problems of the dysregulation of the Abeta.

So in a nutshell, using quantities, very tiny quantities of the enzyme that is known as Neprilysin, and introducing this into the eye tissue, we are just in the process of looking to see, can we, by reducing by essentially engineering a reduction in what would otherwise be the buildup of amyloid beta, delay or retard the deterioration of the eye tissues in a model of age-related macular degeneration?

We are very excited about this. It’s at a very early stage, but if we learn from our experiments that this is indeed improving, or I should say retarding, the deterioration of the eye tissues, we obviously—this would be a very substantial advance in knowledge with potential therapeutic capability.

GUY EAKIN: Well, thank you so much, and everybody stay tuned because of those connections between lots of neurodegenerative conditions we’ve been exploring today. We’ve heard about Parkinson’s and macular degeneration. We’ve heard about diabetic retinopathy and now possibly connecting Alzheimer’s disease with macular degeneration.

So we would like to segue over to a couple of the questions. We don’t have terribly much time remaining, but we have some questions, most notably—if we have Dr. McKay still on the line we have some questions about L-DOPA. One of the questions comes in asking—and I don’t have the name of the person, I think it was if I recall correctly, it was Jerry from Michigan—asking if L DOPA might help prevent dry AMD advancing further to the more advanced or wet forms of the disease. Certainly we heard about, in the records we looked at, we heard about prevention and delay of diagnosis of AMD, but what do we know about progression of the disease? Did you see that in your research?

BRIAN MCKAY: That is what the clinical trial is for. We can’t see it with the records because they are static. So I can’t see movement. Do I think it’s the right way to go? Yes. Do I think it will work? Of course I do. Can I prove it? No, not yet. We need to do a clinical trial. Clinical trial is the only way to answer the question.

GUY EAKIN: I know that we’ve talked ahead of time, and there is a registry for this clinical trial, so if there are people who would like to participate, my understanding is that University of Arizona is collecting those names. If anybody would like to stay on after the call, then we’ve been given some of that information from the university and can share that with anyone else.

There’s a question that comes from Leonard from New York saying, “Okay, in advance of that clinical trial, is it desirable for an elderly person to try the L-DOPA approach?” You have an approved drug and you have a suggestion that it might help, so outside of that clinical trial, what are the risks, what are the typical side effects of L-DOPA?

BRIAN MCKAY: I’m not an M.D., I have a Ph.D. I can’t practice medicine, so I can’t tell people what to take and what not to take. On the other hand, as a scientist I can tell you that for normal people without Parkinson’s disease, L-DOPA doesn’t appear to be particularly dangerous. All drugs can have effects, but this has been since the ’60s, and it appears to be well tolerated in most people. That doesn’t mean everyone, so I would say talk to your doctor about it.

GUY EAKIN: That’s typically the advice that the BrightFocus Foundation gives: that we do wait for the evidentiary basis of all of these decisions because clinical trials are very important to how we find out that drugs work. When the results of that clinical trial come out, you will be the first to know through BrightFocus and many other places. Let’s see, do we have any other, any other calls coming in? Do we still have Maria on the line?

MARIA GRANT: Still here.

GUY EAKIN: There was a similar question from Burton from California, whose eyesight is too far gone for most of the projects that we’ve talked about, but he asks about acupuncture from the standpoint of late-stage disease. Similar to the L-DOPA question, what do we know about acupuncture and macular degeneration? Can you say anything about the literature in that area?

MARIA GRANT: I can’t. There isn’t any yet. Sadly, I probably should explain that the acupuncture points that we’ve been studying for the last 5 years that we feel that are conducive to the release of the natural stem cells from the niches, not just the blood vessels, not just the bone marrow, but throughout the body. Those particular points have been used for centuries to treat other types of conditions, like arthritis and other types of conditions that are associated with inflammation. What we can say is that there is a considerable amount of data supporting the use of these particular points to reduce inflammation. One function that we know about these stem cells, when they go out into circulation, is that they produce factors that are anti-inflammatory. These anti-inflammatory factors have been associated with cures. The cure of arthritis, the cure of asthma, and the cure of diseases associated with inflammation. So, we do indirectly have evidence. It is just that we kind of redefined what has been known about these acupuncture points. Indeed, they have been likely releasing stem cells for many different conditions that are proinflammatory, and they have been successful. It is very indirect evidence.

I am not sure that I made myself clear. We are currently doing a study to examine this, and we have done a similar study in horses and in rats. Now we are going to move forward in patients. It is definitely important to realize that there is a lot of knowledge about acupuncture and how effective it can be for certain conditions. But how and why it is effective is kind of the gap that we are filling in, and these particular points are very easy.

The trial we are initiating is basically 2 weeks, because there is evidence to suggest that 2 weeks is a good increment. These cells are released into the blood, they lower the levels of proinflammatory factors down to normal, and then the body is stimulated again and it is good for 2 weeks.

Conceptually, there is evidence that it is deleterious to the eye and maybe a contributor to age-related macular degeneration, so it could be beneficial. Clearly, it is safe and is not harmful. As a physician and scientist that is our first goal, to not hurt. This is a very natural way to release stem cells. It is the way the body does it. Not the way certain stem cell therapies work, taking stem cells out and putting them at the site of injury. That is a bit of a different strategy. This strategy that we are using is much gentler and pretty simplistic. It is more the way the natural process or repair occurs physiologically.

GUY EAKIN: Well, thank you so much. The reason we invited you all onto the call as our guests today is each of you really are at the cusp of some really amazing discoveries, and here at the end of the year thinking of everything that we are thankful for, really is all of the work that is going on by the researchers, you and those around you, who are making inroads into this really horrible disease.

Certainly our thoughts are going out to the people who are living with this disease, some of you are on the call today, and we hope that this has been a really helpful call to understand that we are at the interface of what has been science fiction becoming science fact. I’m just very appreciative of everybody on the call today for helping to give us a sense of the state.

It is about all the time we’ve had to talk about possible treatments of the future, so we hope that you found it helpful, we hope that you found it inspiring. And thank you again to all of our guests for taking the time to speak with us today.

Thank you for everyone for joining us today. Within about a week we will be posting a recording and a transcript of the call on our website, and of course you can also listen and download past Chats on iTunes and SoundCloud.

Our next Chat is going to be, “AMD: Answers to All Your Questions,” and that’s going to happen on January 27, 2016. It will be a 100 percent question-and-answer Chat with you asking the questions and one of our friends from the ophthalmology community taking the time to give you some answers.

We encourage you to register now and submit those questions in advance, and we will also be sending you a reminder email if you’re registered for this call.

Of course you can always call BrightFocus at 1-800-437-2423 or find any of our resources at our website, www.brightfocus.org.

Once again, thank you Brian, Maria, David for reassuring us that there’s so many wonderful people dedicated to finding better outcomes with people with macular degeneration, and thank you everyone for listening to the call today. If you would like to leave a comment after the call, just stay on the line.

Happy holidays and happy New Year, and thank you from all of us at the BrightFocus Foundation. 

BrightFocus Foundation: 1-800-437-2423 or visit us at www.brightfocus.org. Available resources include:

• Information on these, and other research projects funded by BrightFocus.
• Resource list for people with macular degeneration.
• Resource list for people and their families with glaucoma.

Clinical trials information: www.clinicaltrials.gov.

Possible treatments discussed:

•  L-DOPA, a drug for Parkinson's disease that seems to reduce the incidence of AMD in Parkinson's patients
• Acupuncture and adult stem cells
•  Nanotechnology/chemical prosthesis
•  Anti-VEGF therapies

The information provided in this transcription is a public service of BrightFocus Foundation and is not intended to constitute medical advice. Please consult your physician for personalized medical, dietary, and/or exercise advice. Any medications or supplements should be taken only under medical supervision. BrightFocus Foundation does not endorse any medical products or therapies.