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What’s Being Done Locally To Study And Treat ALS?


Amyotrophic lateral sclerosis, or ALS, is a devastating disease that is almost always fatal and has no known cure. We discuss the challenges the scientific and medical communities face in their efforts to fight the disease. We'll learn about some innovative research taking place in San Diego, and meet a man who is living with ALS.

For more information about ALS, got to the ALS Association Greater San Diego Chapter site.

MAUREEN CAVANAUGH (Host): I'm Maureen Cavanaugh, and you're listening to These Days on KPBS. America became familiar with ALS when New York Yankees first baseman Lou Gehrig was forced to retire from the game after coming down with the disease. That was back in 1939. Then, as now, there is no cure for this neuro-degenerative disease and very few treatments available. ALS has baffled doctors and researchers because the disease appears to affect all sorts of different people for no known reason. And it's also hard to diagnose, especially in its early stages, but research continues. This week scientists from UCSD and the Salk Institute will make a presentation to the California Institute of Regenerative Medicine about their research into stem cell therapy for ALS. Joining us this morning with an update on ALS and its effects on patients and their families are my guests. Dr. Don Cleveland, he’s professor and chair of Cellular and Molecular Medicine at the UC San Diego School of Medicine, and head of the Laboratory of Cell Biology at the Ludwig Institute for Cancer Research. And good morning, Dr. Cleveland. Thank you for being here.

DR. DON CLEVELAND (Chair, Cellular and Molecular Medicine, University of California San Diego): My pleasure. Thanks for having me.

CAVANAUGH: And Dr. Geoffrey Sheean is director of the Neuromuscular Division in UCSD's Department of Neurosciences. He’s a practicing physician who helped establish the ALS Center at the UCSD Medical Center in Hillcrest. Dr. Sheean, good morning. Dr. Sheean, are you with us?

DR. GEOFFREY SHEEAN (Director, Neuromuscular Division, Department of Neurosciences, University of California San Diego): Yes.

CAVANAUGH: Good morning to you. Thanks for being here.

DR. SHEEAN: Yes, I am. Good morning.

CAVANAUGH: And Dan Desmond is my third guest. He’s a retired Navy veteran who lives in El Cajon, and has battled ALS for four years. Dan, good morning. Thank you for coming in.

DAN DESMOND (ALS Patient): Good morning.

CAVANAUGH: Now we invite our listeners to join the conversation. If you or a loved one is living with ALS, give us a call, please, to share your story. Or if you have questions or comments, call us. The number is 1-888-895-5727. Dr. Sheean, let me start with you and let me ask you, what is amyotrophic lateral sclerosis, it’s also known as ALS.

DR. SHEEAN: Yes, well, amyotrophic lateral sclerosis or ALS, also commonly referred to as Lou Gehrig’s disease, is one of these conditions in which there is progressive degeneration of some parts of the nervous system which control the movement of muscles. So patients become progressively weaker and weaker in various parts of their body. And it’s a, what we class as, a neurodegenerative disease which is to say that there is degeneration of some of the neurons or brain cells but also in this case some of the cells in the spinal cord, too. Other examples of neurodegenerative diseases include the Alzheimer’s disease and Parkinson’s disease and so this is another one of those. It’s part of a family of conditions in which these cells deteriorate but, unfortunately, this is the – generally considered the worst of this group of conditions.

CAVANAUGH: Now, Dr. Sheean, what actually happens to a person who has ALS. As I said in the introduction, it seems that it’s rather difficult to diagnose in its early stages. Why is that?

DR. SHEEAN: Well, the onset of the condition can be quite subtle at first and difficult to actually realize what’s going on for both the patient and for their doctor. So the symptoms might be something as simple as some cramping or stiffness in a hand or a leg which then maybe progresses to some weakness of that leg and some of the muscles become a little thin. And this can happen relatively slowly. And there are more common causes of this type of problem than ALS itself. In fact, most of the time that – those symptoms are actually going to be due to something much less serious and so those conditions, other conditions, are looked for initially. It’s really difficult in the early stages until it becomes clear that no other explanation is being found for what’s going on and the condition progresses to the point where it becomes clearer that it is actually ALS.

CAVANAUGH: Now, Dr. Don Cleveland, as the research continues into this disease, I understand that we’ve gotten down to the molecular level in trying to understand what happens to the body. So what happens on that very small scale inside the body when a person has ALS?

DR. CLEVELAND: So the landmark discoveries made in the nineties and then two additional ones in the last two years have given us the first molecular clues to what really provokes the premature death of the neurons, as Dr. Sheean described, the neurons that trigger the muscles to contract. And for many years, we thought, well, these special cells, the ones that start in the spinal cord and then go out and connect with the muscles, innervate the muscles, that these cells – that there’s something that causes them to die and it’s something that they do wrong. And what we’ve learned is that it’s actually a simple lesson that we all know from everyday life. The quality of your life is not dependent just upon you, so it’s not just the motor neuron, it’s the whole environment, the cellular environment in which the motor neuron lives that determines its fate. And, indeed, that is one of the new and most important lessons: The neighborhood matters if you’re going to affect the course of disease, affect the life of those motor neurons. You can do so by targeting them directly but you can also do so by targeting their neighboring non-neuronal cells, and that’s an approach which we will be taking forward in clinical practice and I’m sure we’ll discuss more about it with funding from the California Institute of Regenerative Medicine, that to try to improve the neighborhood so that these neurons—you’re born with them.


DR. CLEVELAND: The number that you have is the number that you have in early development and they have to last the lifetime of the individual. So when you lose them, you can’t get them back. And what we’re – the approaches now will be to try to slow the loss by improving the neighborhood in which they – in which those neurons survive.

CAVANAUGH: Right. Now, I’ll tell you, one thing that is fascinating and I know one thing that’s baffled researchers for years about ALS, Dr. Cleveland, is the fact that there are a small number of people affected by ALS who have gotten it genetically, who – but the vast majority of people have no such genetic risk factor for this disease.

DR. CLEVELAND: Yes, that’s absolutely the case. About 10% of the cases have a clear genetic component but the 90% do not. So we – the current thinking is that there is very likely that for the 90% where there’s no clear genetic component that disease arises from a combination of probably some genetic risk factors and some kind of environmental exposure. Unhappily, that’s exactly the description today: some kind of environmental exposure. And for those who doubted an environmental component, we, as a country, were taught that from the veterans of the first Gulf War in the early nineties. That’s still a young population. This is a disease that typically afflicts people in their forties or fifties, and we now recognize that the veterans of the first Gulf War have an increased incidence of ALS, almost certainly due to something to which they were exposed during that first war. The problem is, what was it?


DR. CLEVELAND: And we do not know.

CAVANAUGH: I’m speaking with Dr. Don Cleveland. He’s one of my guests, also Dr. Geoffrey Sheean. About to bring Dan Desmond into the conversation. We’re talking about research into ALS and also about people living with the disease. 1-888-895-5727 is the number if you’d like to join our conversation. Dan, as I said that you’re a retired Navy veteran, lived in El Cajon. Tell us, if you would, how did you first realize that something might be going wrong with your body.

DESMOND: Well, I know that I’ve been very active all my life. I opened up and started a nonprofit counseling center in El Cajon, which is still going. And we live in El Cajon. We have five and a half acres on the side of a road, a cliff almost. And in the process of putting trees in and everything else, which I found very beneficial to me because I could plant stuff and I didn’t have to argue with a patient, let’s say, and it was very enjoyable. But I found as the process, I was doing that, I was using my hoe more and more, it seemed like, as a cane. Started doing some things. We were walking on the beach and I find my wife’s walking faster than I can. The biggest thing was we had gone on a vacation with a couple to Sedona and the gentleman and I went ahead and we went for a walk, it was a 10-mile hike, and we were about a mile and a half, two miles into it, and my legs just gave out. I sat down and he and I spent about two hours just sitting right there. When I hobbled back and got back to the car and, you know, I just couldn’t figure it out.


DESMOND: The first doctor I saw was Dr. Armstrong and I remember walking into his office. He saw me walking down the hall and I sat down and he said we’re going to do some testing, he goes, but I already think I know what’s wrong. And he did the testing. He said he’s 90-some percent sure that I have ALS. And he goes, you’ve got a short time to live. I remember him saying that. And he looked at my wife and I and he goes, you probably have four years. And we said, okay. And he goes, no, no, he goes, you have four years to live.


DESMOND: And I said, yes, I said, I heard that. I – Sounds like I’m dying. And he goes, yes. And I say but you’re dying, too, it’s just I know when. There’s a little difference. But being in the military, the military has taken this one disease because over 60% of the people who come down with this disease were in a branch of the military for at least 90 days. And so they’ve come alongside. That’s been a wonderful blessing for me.

CAVANAUGH: Let me ask you something, Dan. Obviously, that first diagnosis, after it hits you, after you get that in you and you know that this is something that you’re going to have to deal with, how has your life changed as a result of this disease?

DESMOND: Well, a little after ten years of doing counseling—and I really enjoyed it—my patients, clients would come in and they start talking about my health because I’m using – I went from a cane to a walker to a wheelchair in about six months time because my legs just went. And counseling is not about me, it has to be about them.


DESMOND: So we had set up a nonprofit corporation. There was a wonderful gentleman there. And I said, this counseling practice is yours now. I’ll gladly serve on your board but it’s yours. And he has about 10 people on staff, and it’s a wonderful thing. So I had to stop that. That was number one. I’ve lost – With this disease, you lose different parts of your body. It could start in your throat, it could start on a hand, it could start on your leg. What happens is you lose the limb. You still feel. If there was a fire and my foot was on top of it, it will burn. It will hurt. I just can’t move it up. And the little things of – well, fasciculations. Fasciculations is like a muscle spasm.


DESMOND: I normally have those going in about 20 areas of my body at any one time.


DESMOND: And I think within about a five month period of time, I lost about 30% of my lung capacity. Now the lung didn’t get impacted, the muscle around the lung…

CAVANAUGH: The muscle around it, right.

DESMOND: And so at times my hands freeze up, different little parts of my body. I’m doing less. But I have been very, very blessed. I’ve had a full life. Got all my family here, all my children, tons of friends. And so we will still do things. We were joking out in the waiting room, when I was – just before I went in this wheelchair, a friend of mine said, Dan, you doing anything on Friday? No. He took me over to Torrey Pines and hooked me up and I went sailing off the cliff. And, you know, it was a wonderful thing. And me, I’m just – I’m Dan, type A, I’m trying to be a C right now for my wife, but that doesn’t work. But having a disease is a terrible thing. Having this disease is probably not my first choice.


DESMOND: But I have today and I live today, and I enjoy today.

CAVANAUGH: We have to take a break, Dan. We’re going to hear so much more from you, though, and thank you for sharing that story. It’s a very – You gave people a very good idea of what it is to get a diagnosis like this and to live with it. We are going to take a short break. When we return, I’m going to speak more with my guests. We have a number of people who want to join the conversation at 1-888-895-5727. You’re listening to These Days on KPBS.

CAVANAUGH: Welcome back. I'm Maureen Cavanaugh. You're listening to These Days on KPBS. We are talking about ALS, research going into the disease and the effects on patients and their families. My guests are Dr. Don Cleveland. He is a researcher and he does research on ALS. He’s one of the lead investigators for the California Institute of Regenerative Medicine grant on ALS stem cell research. Dr. Geoffrey Sheean is director of the Neuromuscular Division in UCSD's Department of Neurosciences and a practicing physician who’s helped establish the ALS Center at the UCSD Medical Center in Hillcrest. And Dan Desmond is here. He lives in El Cajon and he’s battled ALS for four years. We’re taking your calls at 1-888-895-5727. Before we take a call, Dr. Sheean, I want to ask you, one of the things that is baffling about this baffling disease is the time frame that’s given for people who are diagnosed with this disease. We know some people who are diagnosed with ALS and who die quite rapidly after that diagnosis and yet other people, most notably Stephen Hawking, has been dealing – living with ALS for over 20 years now, I believe. What is that difference, do we know?

DR. SHEEAN: Well, I think that’s exactly what you point out is correct that, you know, ordinarily the average survival, as Dan was told, is somewhere between three and five years or so. But there are some cases that go much more rapidly or much more slowly and that is very, very surprising because the patients all seem to be otherwise very similar in the areas that are affected. So we don’t really know, actually, why that’s the case. There are certain things that can help us predict how rapidly it’s going to go but really the pace is generally determined by the disease and the actual reason for these differences, I think, are largely unknown, although Dr. Cleveland might have some…


DR. SHEEAN: …ideas about that.

CAVANAUGH: Is there anything that we know about how quickly this disease is going to progress.

DR. CLEVELAND: We know that in some of the genetic cases where we know that the primary gene whose defect causes disease, there the predictions on the speed with which the disease will progress can be made much more accurately. Unhappily, for about half the American patients who have the most prominent genetic cause of disease, that – the disease actually progresses quite rapidly so those patients typically have only a year of – from onset to death. But in the other cases, no, we do not know what are the key components that drive more rapid disease or the quite unusual cases like Stephen Hawking where the disease has progressed remarkably slowly compared to the typical.

CAVANAUGH: Let’s take a call. We are taking your calls at 1-888-895-5727. Inez is calling from San Diego. Good morning, Inez. Welcome to These Days.

INEZ (Caller, San Diego): Well, good morning. Yes, I was – I’ve had ALS since 1996 and I wasn’t diagnosed until about 2004. And I’ve been battling it ever since. And it really takes a toll. It’s very hard but I feel the same way as your other caller. I feel very blessed that I know and finally found out what was wrong with me. I went for quite a few years not knowing what was happening to my body. I find it difficult now to talk. My arms and my hands are constantly moving. My legs also, but I still have a lot of strength in my legs and I’m still able to do some of my artwork but it’s very, very hard. And in the beginning, I remember my hands were moving. I could see little movements in my hand and couldn’t figure out what it was and then I would try to hang up my clothes and I found it difficult to raise my arms and to hang skirts, you know, and button buttons, little things like that. And I started going to the doctors, they could never figure out what it was. I went for about 3 years to Kaiser. Finally, I was told to go to a neurologist so that was when they found out that was what it was. And I have a wonderful doctor now, Dr. Hawkins here in San Diego and I go to him about every four months just to let him know what’s happening so he can help other patients with how to deal with it and all.

CAVANAUGH: Inez, thank you so much for calling. I appreciate that phone call. Nancy is calling us now from Carlsbad. Good morning, Nancy. Welcome to These Days.

NANCY (Caller, Carlsbad): Good morning. My mother had ALS and I’m wondering if there’s a hereditary component here.

CAVANAUGH: Let’s – Nancy, let’s try to find out about that. Dr. Cleveland, is there a test that Nancy or someone in her position could get?

DR. CLEVELAND: So, the first quest – so, Nancy, was there anyone else that you know of in your family who had ALS besides your mother?

NANCY: Not that I know of. I don’t know of – I don’t know – Not that I know of.


DR. CLEVELAND: So do you have an extended family or are you a more modern American family that – whose roots are not quite as deep as – So if there’s only one, then the high likelihood is that your mother had what we would refer to as sporadic disease without a strong genetic component. And there really is not a useful test to – for looking for one of the known genetic causes. They – the most prominent cause, genetic cause, only accounts for 2% of overall cases, so it just wouldn’t be a sense – I don’t think it would be a very sensible plan.


DR. CLEVELAND: And the very high likelihood is that it’s – that it doesn’t have a strong genetic component.

CAVANAUGH: I see. But even if there were a number of people in her family who had ALS, there really is no way to test if she would have that genetic component.

DR. CLEVELAND: Well, there – So, again, the – about 20% of the patients who do have clearly inherited disease have mutation – have a defect in a known gene and another 10% have a defect in a second and then in a third gene. So we could – if there was a clear evidence of an inherited form, yes, you could begin to – we could score for that.


DESMOND: I was going to say, if her mom was the only one, yeah, see her regular doctor but don’t live with a fear.


DESMOND: Just move ahead with your life and enjoy today.

CAVANAUGH: Right. Dr. Sheean, I wanted to ask you, we heard from Inez and we have Dan here, who are living with ALS. What kind of treatments are available? I know that you describe your clinic at UCSD as multi-disciplinary. So what kind of – what things do you offer patients there?

DR. SHEEAN: Right, well, I think it’s, first, yes, it’s not a disease that we can cure for sure. There is one medication that has a fairly limited role in slowing down the progressions of ALS a little. It’s not a very strong effect but it is the only thing that we have that’s proven to actually alter the course of the disease but only by prolonging the inevitable by several months at best. So – But that doesn’t mean that we can’t do an awful lot to help people. We can. And what’s called the multidisciplinary clinic at ALS is actually a group of people, specialists, and that includes people like therapists, including speech, occupational, physical, respiratory therapists, dieticians. We have a nurse. We have a number of people there who can attend to the patient’s needs. And we can do an awful lot to make people more functional. In the early stages, we can help strengthen them as well, but help them make better use of their abilities and that will make them more functional, improve the quality of life. And the other things that we can do there are to – very early on, we can attend to their breathing difficulties as well, initially by using facial masks or similar to those masks that are worn by patients with sleep apnea to help support their breathing and their diaphragm to make their, initially, breathing at nighttime much more comfortable and then potentially in the day as well. We’re also very aggressive with the use of nutritional therapy including the dietician as I mentioned but we can also, when the time arises, we can use feeding tubes artificially inserted into the stomach to help support nutrition because that will, we believe, although, unfortunately, not yet proven, help prolong survival, too. So there’s an awful lot we can do to make life better and to prolong life as well.

CAVANAUGH: Dr. Sheean you make a very strong point in an earlier conversation that people used to think, well, ALS is a death sentence so why should I bother with treatment? But you make a very, very strong point that it’s important for people not to give up.

DR. SHEEAN: Yes, absolutely, because, yes, I think there is this unfortunate nihilism out there where people think, okay, there’s nothing that can be done and then there’s – they do just give up. But that’s not entirely true. We can make the quality of life much, much better for the patient as well as their family, too. We mustn’t forget that this is not just a person, it’s a part of a person who’s a part of a family. And we can do an awful lot to make their life easier, too.

CAVANAUGH: Dan, I believe that you use the ALS Center at UCSD. How has that helped you?

DESMOND: Well, first of all, the – As the doctor had said, the individuals who are there are very upbeat, very positive. The individuals go in, the patients, and there’s going to be x-number of us seen at one time. So let’s say half a dozen. Each of us goes into a room. Well, then the doctors, the specialists from each of the different fields, are going to come into that room so the patient isn’t moved from room to room. And that’s easier on the patients. A lot of us come in in wheelchairs. The other thing is it’s a team approach. I think that is so good. And, myself, I know the dietician has worked with me and I’ve changed some of my eating habits because a lot of individuals end up getting food in their lungs, or water, and that causes all kinds of side effects. It can be a pneumonia, etcetera. And this is the best of my knowledge, you don’t die of the disease, you die of the effects from the disease because the body just doesn’t work.


DESMOND: And – But having the UCSD come in alongside the individual patients and if there’s something wrong, we just pick up the phone and we phone them.


DESMOND: And that is – that’s so much easier. The medicines they’re going to give are going to fit for the individual person.

CAVANAUGH: Let me take a phone call. Chris is calling us from Carlsbad. Good morning, Chris. Welcome to These Days.

CHRIS (Caller, Carlsbad): Thank you for taking my call. You know, we had a very close family friend diagnosed with ALS last fall. He was progressing pretty rapidly with his symptoms and just really going downhill. And his ultimate diagnosis was ALS and then he did some research and found out he was bitten by a tick several years ago and I guess on the off chance he had some testing done to see if, you know, it was Lyme disease and he eventually tested positive for this and has since undergone some intensive antibiotic treatment, I think, is what they gave him. (audio dropout) since completely halted the symptoms of ALS as well as actually been improving, walking around and doing things he couldn’t do three or four months ago.


CHRIS: So I’d be interested to hear – It sounds like there’s some controversy around it but I’d be interested to hear the correlation between ALS and Lyme disease and whether that’s a possible diagnosis and along those lines.

CAVANAUGH: Gotcha, Chris. Thank you. Dr. Cleveland? Any correlation between ALS and Lyme disease?

DR. CLEVELAND: Actually, I think that’s a question best directed to Dr. Sheean.

CAVANAUGH: Oh, all right.

DR. SHEEAN: Yeah, well, I think that that is an interesting story and it raises the possibility that the patient actually does have Lyme disease and it does underline the difficulty that it can – that a patient can experience in actually getting a diagnosis of ALS. Well, firstly, ALS by itself does not have a test that we can run that comes back positive or negative. There is no test at present for ALS…


DR. SHEEAN: …so it’s really one of exclusion. In other words, we have to exclude alternatives, things that we know can look like ALS and Lyme disease is certainly one of those. And we are very careful to check for Lyme disease and we take that seriously but it can be a difficult diagnosis to prove. But – so it’s possible that the patient does have Lyme disease. As far as I know, there is no connection between the Lyme disease itself and what we would otherwise call ALS. In other words, you don’t get ALS from a tick bite that gives you…


DR. SHEEAN: …Lyme disease, you get Lyme disease that might look like ALS.

CAVANAUGH: I see. One of our other callers wanted to know what the difference is between MS and ALS. Anyone like to take that one?

DR. SHEEAN: Well, perhaps I should take that one.

CAVANAUGH: Okay, Dr. Sheean.

DR. SHEEAN: Yeah, well, MS is multiple sclerosis.


DR. SHEEAN: And ALS is also a central nervous system disease like multiple sclerosis but multiple sclerosis we strongly believe is an immune system attack on parts of the nervous system in a completely different way to what goes on in ALS. And there are other differences. Firstly, MS can affect pretty much any part of the nervous system but ALS generally afflicts only those areas of the nervous system that control muscles that we actually have control over ourselves, like breathing and moving our arms and legs and it doesn’t affect things like bladder control, for the most part, or – and it doesn’t affect sensation, as Dan pointed out. He’s got perfectly normal feeling…


DR. SHEEAN: …and sensation. So there are big differences in the way it appears, and MS is, in many ways, much more readily treatable these days than it was as well. So the distinction between the two is not usually difficult in the full blown cases but they can look similar in some ways early on.

CAVANAUGH: I want to ask you, Dr. Cleveland, I know that you’re going to be making a presentation about your research proposal at the meeting taking place, the CIRM meeting, taking place in San Diego this week. Where are you in the process of this research?

DR. CLEVELAND: So the California Institute of Regenerative Medicine, this was Prop 71 that the Californians voted on in 2004 has launched, this last year and our project just the first of this month, has made substantial funding available to try to use a stem cell therapy approach for treating disease, in our case ALS. So we have – we know the proof in principle that by supplying patients with good neighborhood cells, if you remember my conversation earlier in our…

CAVANAUGH: Yes, right.

DR. CLEVELAND: …program where we now know it’s not just the damage derived within the neuron, the motor neuron, the one that innervates the muscle. It’s not just damage within that cell that leads to the dysfunction and the failure of that motor neuron, it’s actually the failure of the neighborhood, and that’s how the disease spreads from a focal start. And so what our approach will be to use stem cell approaches to provide normal supporting cells to these motor neurons. We know it can work, now the – the real question is now can we, using FDA approvable methods, can we really do that in the human setting? The Proposition 71 has really stimulated the scientific community and the medical community, can we really develop methods that will work. Oh, this is the good news and the bad news.


DR. CLEVELAND: The good news is we really – there’s a very serious effort to test whether this will really be a positive therapy in ALS but it’s a question. We will know only by trying to take the approaches forward to real clinical practice and our – we have a timeline. We’ve started this month. The timeline is to be in clinical trials in the fourth year. So that sounds long to patients. I have to say it’s extremely aggressive from the research standpoint but we’re going to give it a very serious effort.

CAVANAUGH: We only have about 30 seconds left, Dan, and I’m wondering as you hear this, how closely do you follow the research into finding a cure for ALS?

DESMOND: To be very honest, I’m not right on top of it. I applaud it. I’m in favor of it. I probably won’t be around in four years and I know that. But the issue is, there will be a multitude of other people who will be around and so the research is going to help them. And it takes time, there’s just no way around it. The only thing I can say is the individuals who have the disease today, live today. You’ve got today. Live today as a good quality of life. Mentally affects your physical being. And if you give up, then the fight is over, and there’s too much to live for. Enjoy today and the research is going to come along. They’re going to find a cure at a point in time.

CAVANAUGH: Dan Desmond, thank you so much. I appreciate everything that you’ve said today. Thank you. And Dr. Don Cleveland, thank you. And Dr. Geoffrey Sheean, thanks so much for all of you. And all the people who called we didn’t get on the air, please go online with your questions and your comments, Coming up, memories from the World War II Battle of Iwo Jima as These Days continues here on KPBS.

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