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Ask Dr. Mike: New Treatment Options for ALS

Here you'll find the answers to a wealth of health and wellness questions posed by Healthy Talk fans. Listen in because what you know helps ensure healthy choices you can live with. Today on Healthy Talk, you wanted to know:

My dad was diagnosed with ALS (Lou Gehrig's Disease) two years ago. He's actually doing pretty well, he's on a common drug used for treating ALS, Rilutek (riluzole). I'm wondering if there's anything new?

This drug has been around 15-20 years and is the main drug used to slow the progression down of ALS. However, there are three treatments that you may want to learn about. They are stem cells, TAR DNA-binding protein 43 (TDP-43) and FUS (fused in sarcoma), and the insulin growth factor.

If you want to read the full article on these three promising treatments, read here.

If you have a health question or concern, Dr. Mike encourages you to write him at This email address is being protected from spambots. You need JavaScript enabled to view it. or call in, toll-free, to the LIVE radio show (1.844.305.7800) so he can provide you with support and helpful advice.

RadioMD Presents:Healthy Talk | Original Air Date: March 25, 2015
Host: Michael Smith, MD

RadioMD. It's time to Ask Dr. Mike. Do you have a question about your health? Dr. Mike can answer your questions. Just email: This email address is being protected from spambots. You need JavaScript enabled to view it. or call now: 877-711-5211. The lines are open.

DR. MIKE: That's This email address is being protected from spambots. You need JavaScript enabled to view it. . I love receiving our questions either by phone or email. It's all great. This one came through, so I'm going to probably have to spend a little time with this one, but I thought it was a real good question. To be honest, this is where working for Life Extension really comes to my advantage because of the amount of information we publish at the Foundation.

So, here's the question: "My dad was diagnosed with ALS." Now, that's Lou Gehrig's disease. Very, very devastating neurodegenerative disorder. It has a very poor prognosis overall. So, anyway. So, let' me get back to this.

"So, my dad was diagnosed with ALS two years ago. He's actually doing pretty well. He's on a common drug. I think it's called riluzole. I was wondering if there's anything new on the horizon. Thanks."

So, I think, first of all the drug that he's on, riluzole, that is the drug. It's been around, gosh, I don't know, maybe 15-20 years and it's kind of the mainstay, the main "go to" drug for conventional medicine. It has to do with glutamine and glutamate receptors. I don't remember the whole thing, but it maybe slows the progression down a little bit. It's not anything impressive. So, I just thought this was a great opportunity, in answering this question, and doing some research, going to the Foundation I work at, seeing what we've looked into over the past few years. I came up with a nice little list. By the way, this protocol on ALS is available at So, I wanted to mention three things that we have written about recently. Stem cells for ALS, something called the TAR DNA 43, TDP43 and the insulin-like growth factor and growth hormones. So, I'm going to talk about those in answering this question.

The first thing I think is probably the most exciting and has probably the most potential in treating ALS is stem cells. Basically, a stem cell is an immature cell that can differentiate into a nerve, a heart, a liver cell. You know, whatever it needs to be. As we get older, we have what are call "adult stem cells" that literally are in your blood, in your bone marrow and when needed, they can be released. They can get into the area they're needed. They can, then, differentiate into that more specialized cell if it's needed in the heart, liver, whatever. So, the basic theory is, okay, if there's a destruction of certain brain cells in ALS, is it possible for us to transplant or deliver stem cells into that part of the brain and create new brain cells, a new brain cell environment and would that have benefit? I think there's a lot of potential here.

The problem is over the past maybe 10 years, stem cell research has gone slowly and some of that has to do with the debate about stem cell research, embryonic stem cell research, and so we're not really where we should be, in my opinion, with stem cells in treating all kinds of disorders. By the way, the good news is, you don't have to use embryonic stem cells anymore. There is a way to take skin cells. Dr. Michael West, I want to say he's out in California. Dr. Michael West did a lot of research in creating stem cells from skin cells, totally skipping the embryo, so taking that completely out of the debate and his research is quite exciting and, I think, is going to really change the attitude of stem cells for most people in this country.

So, anyway, bottom line, there's been few trials because of the embryonic stem cell debate, Dr. West's research has not really been known and shared yet. It needs to be, so there's just not a lot of trials using stem cells in ALS. But, there are some. I want to mention a couple here. So, there was one study published in Cytotherapy, I want to say, 2009, maybe 2010, which showed bone marrow derived stem cell transplantation in the motor cortex delays ALS progression and improves quality of life.

So, what they did was, they took stem cells that they grew in a petri dish. Remember, those are immature cells that can pretty much become anything. They injected them into the motor cortex in ALS patients and these patients did quite well. I think there was about 50 people in this study. Again, it's not a conclusive study. It's just the beginning. By the way, before this was done in humans, there were a few animal models that they looked at as well and it was quite successful. So, I think there's a lot of promise there.

Another research study using stem cells was published in Neural Research, 2012, and they did, again, bone marrow injections. So, in this case, they didn't really isolate specific stem cells, they just did a bone marrow aspirate. You know, you go into the bone marrow of a long bone like a femur. You just draw out some of the fluid and they injected it directly into the frontal motor cortex of human ALS patients. It was well-tolerated, safe and there was a quick improvement in symptoms and in cognition and in motor control. Again, these studies were limited, so they weren't able to follow these patients for a long time. So, there's limited information about how long something like that would last. Does it really take hold? Are they really creating new brain cells? I mean, there are still a lot of questions there, but this idea of taking a stem cell, transplanting it or injecting it into the brain, into the motor cortex, is showing a lot of promise. So, personally, I think that this is the best potential we have for treating this very devastating disease, this stem cell research.

Now, there's another one called "TAR DNA binding protein 43". Research has identified the cellular protein TDP43, TAR DNA binding protein as an important factor in the cause of ALS, especially the sporadic forms. TDP43 binds DNA and RNA in cells, including motor neurons. What they find in patients with ALS is that this protein, this TDP43 protein that binds to the DNA and RNA gets kind of clumpy, if you will. You know, like in Alzheimer's, we have the abnormal protein deposits in the brain, but they're called "protein aggregates". It's the same thing here, this protein. So, Alzheimer's has it's abnormal protein. Well, it turns out ALS might have its abnormal protein. So, that's kind of interesting research.

So, this protein, this DNA/RNA binding protein, it just clumps. It doesn't bind properly and that results in some of the pathology that we see and symptoms that we see in ALS. So, what does this mean in the future? Well, it means gene therapy. I mean, if we've isolated a protein, we can, then, find the gene coating that protein and we can see where those mutations are and start trying to correct that, injecting the actual gene, a better gene, a healthy gene into the patient and that might actually allow this DNA binding protein to do what it's supposed to do and not clump. So, there's some research there and then, of course, insulin growth factor and growth hormone have been shown to be beneficial in ALS patients. Still a lot of research to go. Growth hormone, that's a good name for the hormone in kids, but growth hormone is really better known as the "repair and regeneration hormone" in adults, so more research is looking at growth hormone and IGF-1, which is a marker of growth hormone production, using that in ALS patients.

So, stem cells, the TAR DNA binding protein and growth hormone offer some really good opportunities in ALS patients. This is Healthy Talk on RadioMD. I'm Dr. Mike. Stay well.