• ALS Crowd Radio Episodes
    • Apr 11, 2016

    ALS Crowd Radio S2:E1 Dr. Merit Cudkowicz on ALS Research: Progress Since the Ice Bucket Challenge

Dr. Merit E. Cudkowicz, MD, MSc
Massachusetts General Hospital
Interview Date: April 12, 2016

Dr. Cudkowicz is a leading expert in the field of ALS and is involved in all aspects of research, clinical trials, and patient care. She gives an update of ALS breakthroughs that have occurred over the last two years, since the Ice Bucket Challenge. The increased awareness and attention brought more resources, especially more doctors, companies and researchers working on the ALS puzzle. Dr. Cudkowicz tells of a new drug called Radicut® (aka Edaravone) which has been approved in Japan to treat ALS. The last two years also brought approval for Nuedexta in treating emotions and can help improve speech and swallowing. iPCS has also taken off, which is collecting blood samples from a patient then making motor neurons specific for that patient. This will lead to better individualized treatments. Answer ALS is currently undertaking a large coordinated effort to collect blood samples. A new company called Origins is creating a prognostic equation to see if they can test drugs in a better and quicker way. Promising stem cell therapy results have been released by Brainstorm and will be released soon from Neurostem. There is hopeful progess in finding biomarkers in three areas: imaging, electrophysiology, and blood & spinal fluid. New technology is also making it possible for outcomes to be measured from home, so patients don’t need to travel to a clinic as often. There are many ways patients can become involved in research and advocacy. Dr. Cudkowicz shares her expertise from being right in the middle of this important time for ALS discoveries.

ALS Crowd Radio Show with Dr. Cudkowicz

Full Transcript:

Seth: Hello and welcome to another episode of ALS Crowd Radio. I’m your host, Seth Christensen. Here with my good friend and a fellow ALS diagnosee, Whitney Hill. Hi Whit.  Good to have you with us. We are honored today to kick off Season 2 of our radio show. Our show began almost two years ago with the Ice Bucket Challenge.  Now, we are honored to have back with us our original guest, Dr. Merit Cudkowicz from Harvard University. I will pause for a moment to have Amy introduce Merit.

Amy: Dr. Merit Cudkowicz is the Professor of Neurology at Harvard Medical School, the Chief of Neurology Services at Massachusetts General Hospital, Director of Massachusetts General Hospital, MDA ALS Clinic, Co-director of the Neurological Clinical Research Institute, and the Co-director of Neuromuscular Division at Massachusetts General Hospital. She is the Julianne Dorn Professor of Neurology at Massachusetts General at Harvard Medical School. Dr. Cudkowicz completed medical training at Harvard Medical School and was a resident in Neurology at MGH. She obtained her master’s degree in Clinical Epidemiology from the Harvard School of Public Health.

Dr. Cudkowicz’s research and clinical activities are dedicated to the study and treatments of patients with neurodegenerative disorders, in particular, ALS. She directs the MGH ALS clinic and the Neurology Clinical Trials Unit. She is one of the founders and co-directors of NEALS, or the Northeast ALS Consortium, a group of 92 clinical sites in the United States and Canada dedicated to performing collaborative academic lead clinical trials in ALS. Dr. Cudkowicz received the American Academy of Neurology 2009 Sheila Essey ALS Award. Dr. Cudkowicz is on the Research Council of the American Academy of Neurology and the Medical Advisory Boards for the Muscular Dystrophy Association.

Seth: Thank you.  Dr. Merit, are you with us?

Dr. Cudkowicz: Yes, and thank you very much for your kind words, Amy, and it’s great to be here. And thank you, Seth, for inviting me.

Seth: Well, I’m honored to have you back. Merit, we’re grateful to have you today and wondered if we could start off by where we left off two years ago. We interviewed you last right before the ice bucket challenge. And know that a lot of our world changed.  Could you tell us at a high level, in your view, has anything changed for ALS since our last interview?

Dr. Cudkowicz: Yeah. So much has happened since our last interview. It says the ALS field is finally on fire with progress and hope and a lot of people studying it. I was thinking about this; whar are the top things that have happened since I last spoke to you.  We’ve have progress in lots of areas. The most exciting, obviously, is on the therpaies.  I’ll definitely talk a little bit about that, but we’ve had progress on therapeutics for the people with ALS. There’s been progress in understanding the underlying biology of the disease and how this might be different in different groups of people. And huge progress in what is really known as big data of making the most of all the data out there on people with ALS and how we can learn about the illness and develop treatments. I think those would be the three areas that have changed a lot since we last spoke two years ago.

Seth: Well, we would love to dive into those areas a little more if you have a minute for each one.  

Dr. Cudkowicz: Oh yes, absolutely. So therapeutic.  There is now another drug approved for ALS since we last spoke, and that drug is approved in Japan but it’s still an approved drug for people with ALS, and it’s called edaravone, also known as Radicut. And I think that’s just a huge news. The last time a drug was approved for ALS to slow down the illness was Riluzole in the mid-‘90s.

It’s really good news to have another drug. This drug now has been reported by the company at international meetings. They’ve shared the data that shows that it slows down the illness, and particularly effective taken kind of early in the illness. So it’s great news. It’s not obviously yet approved in Europe and the U.S. but the hope is that that’s where it’s going to be moving so it’s available to people really all over the world, just like Riluzole is.

That’s I think huge news. And related to that, there’s also another drug that is on the market for ALS called Nuedexta, which is on the market for a symptom of ALS called pseudobulbar affect when sometimes people with ALS might have extra emotions. That can be disabling and we know it works well for that, but in the trial for that symptom, the participants were telling the doctors that their speech and swallowing was better.

Dr. Smith, who was leading that study, Richard Smith, then did another study with NEALS just to see could Nuedexta improve what we call our bulbar symptoms or symptoms with speech, abnormalities or swallowing difficulties?  And he also reported in December his results where he showed that 75% of the people in the study had improvement in their swallowing and speech. That’s huge also because those are symptoms that are really important for patients to try to see if we can find ways to improve them.

I think those are two positive things in the right direction for therapeutics. And also, there is other positive news out there in therapies of reports of a few other treatments that have had initial positive results in what we call the early development trials, the early phase of testing. For example, methylcobalamin was reported to have some positive effects in the phase 2 study. And there was a recent report of Masitinib having some positive effects.

I think we’re finally turning the corner on therapies. We’re finally giving some hits; that’s just phenomenal on my end. So there’s a lot more to do in that but at least it’s going in the right direction.

Whitney: That sounds wonderful. Hi, Dr. Cudkowicz. This is Whitney Hill, Seth’s co-host. We met a couple of years ago when I came to you as a patient.

Dr. Cudkowicz: Good to hear you.

Whitney: Good to talk to you. When you say that those new drugs are showing some promise, how has the research came since the introduction of the money from the ALS ice bucket challenge? Has the focus changed a little bit? Are we seeing things heading in a new direction? Are we hitting olf things and trying to make those work? What do you see?

Dr. Cudkowicz: I think what we’re seeing since the ice bucket is basically global awareness of the illness and ALS research happening in more places than it used to. I think the ice bucket challenge kind of brought the awareness and it certainly brought resources. But it also got people all over the world excited about studying ALS, and other foundations contributing to ALS research. We’re in an unusual time, and I hope it stays for a long time where there are a lot of people interested in funding ALS research.

There are drug companies interested in it, there are investigators all over the world that are opening up parts of their lab to study ALS. The more people you have focused on it from different backgrounds, it’s just I think the more likely that you’re going to have some successes. I really think the ice bucket challenge awareness was almost even bigger than obviously the resources that brought in.

Whitney: I agree. In researching lately, I’ve been noticing that there’s been a lot of news in more of the genetic component of ALS. Certain genetic components that — it kind of seems like the researchers had — we’ve seen a lot more focus on that. Do you agree with that or do you think that there is — that’s only about 5% to 10% of the population, isn’t it, of the ALS patients?

Dr. Cudkowicz: Yeah. I think the genetic form is less than about 10% of the population, and there’s certainly very exciting work going on in genetic forms, but there’s also a ton of work going on in the sporadic forms. In the genetic form, the excitement is that the tools to modify or block genes of mutations from making them mutated protein that causes illness, those tools are now ready to go into people.

There are some trials, for example, patients with SOD1 ALS or C9 ALS, some of the genetic forms. There are trials geared just for those patients and they’re right on target of the cause to try to stop that disease. So you did see a lot of news about that and a lot of research on the models of those diseases, but there’s also a huge emphasis on the other 90%. Some of the work going on in the sporadic ALS is to try to understand, is that one disease or is it ten diseases? Is ALS all one disease or is it a syndrome?

I always give the example of blood pressure. There’s a lot of reasons why people have a high blood pressure and you treat people differently based on their cause of their high blood pressure. We think that that might be the case in ALS too. So where the resource and the researcher going for sporadic ALS is to try to understand that disease heterogeneity. And that was the second area I think has really changed a lot since we spoke two years ago that the tools to understand that disease heterogeneity, exists now where they didn’t exist before. And those are technology tools.

For example, we’re partnering with GE Healthcare and another ALS foundation called ALS Finding a Cure to bring all imaging technology GE has to ALS to see if we could actually image the brain in the MRI and see the underlying biology. If we can, that gives us tools to screen drugs much faster in people and to put the right therapies in the right patients based on their biology. That’s brand new since the last time we talked and looking incredibly hopeful.

The other technology that’s being used to really get at this, is this one disease or is this a syndrome with lots of different biologies,  is a technology called IPSC or Induced Pluripotent Stem Cells. The idea is taking people’s own blood and taking their stem cells in their blood and being able to make from that stem cell the person’s individual motor neuron. It’s a way to study a person’s individual disease. And that technology is relatively recent.

There’s a big study that’s funded by several groups including this ALS Finding a Cure and Team Gleason and Travelers to do big data study where you ask a thousand people with ALS to come in and donate a sample of blood so that we can make people’s motor neurons and study everything there is about it and then really try to get at this question of, is this one disease or many? And once we do that, that’s going to make developing therapies much, much faster for people and much smarter.

Whitney: Dr. Cudkowicz, how do we have access to that process as an ALS patient to have access to the IPSC and to be able to give you our information and have that mapped out? I mean I haven’t even heard of this.

Dr. Cudkowicz: Yeah. Good question. So there is an Answer ALS website that Team Gleason is hosting. And on that website, it’s list of centers that are currently enrolling those five right now, but the hope is that will grow. That will be the first place to start, kind of Googling Answer ALS. And also, the patients ask their ALS neurologists, how can you participate in research? NEALS has really tried to lower those barriers so that any ALS clinic that wants to be part of research and wants to contribute blood samples or clinical data from their patients can do that very easily.

Seth: This is incredible.  We thank you for those answers. I’d like to go to a topic that you mentioned here briefly. It is one of the major outcomes of our original interview. At that time, you talked about big data. A number of these projects are beginning to merge onto that idea.  How far are we out from a true centralized database of ALS patient information?

Dr. Cudkowicz: There’s been huge progress in that as well. I think there’s still more to do but since we spoke, a couple initiatives have taken place to really try to learn from every person with ALS and to be able to share their data with any researcher. One of them is something called PRO-ACT, and that just continues to grow. And what PRO-ACT is, it’s a database of all past clinical trials. The companies have agreed to share the data, particularly, for sure the data from people who are in placebo and sometimes also from people who are on treated drug. And that’s an open source database for anyone to mine, and that continues to grow.

There’s now data from 11,000 people with ALS in PRO-ACT and there have been over 1,000 people who have downloaded the data and about 20 plus papers. But the most, I think, important thing that has come out of that so far is that there are companies actually been formed from it called the Origent to develop a prognostic equation for ALS. Why that’s important is this might be a way to screen drugs faster.

If you could take a few clinical data points, make maybe a prediction of what would happen to someone with this illness, and then see if your drug can change that trajectory. And there have been discussions already with the FDA. Would they accept this type of clinical trial design? These are new people never in the ALS field. They are computer scientists who have come into the field to see if they can help improve and speed up the whole process of how we develop drugs. And that wouldn’t have been possible without this kind of open source huge dataset. So that’s clinical trial data.

The other data we want to really capture is the clinical data because people with ALS are going to the clinics, are giving information to their physicians. Obviously, they’re getting care, but if there was a way to collect that data as well and make it anonymized so it’s safe but available to researchers, that would be fantastic. We are using a system called NeuroBANK for that and working closely with companies that have electronic health record systems like Epic to see if we could make it part of the electronic record that all these hospitals have. There’s been big progress there as well.

Seth: Excellent. Now, are we including non-clinical data in those efforts? For example, environmental data or geographical data?

Dr. Cudkowicz: Yes. The way NeuroBANK is built is that there’s a list of a library of forms and they include all the epidemiology forms that the ALS field has created, as well as forms for other clinical characteristics. The investigator, for example, maybe someone’s doctor, if they want to start collecting information on their patients, they can go to NeuroBANK, it’s free, and download the forms that they want and start entering their patient’s data.  And it becomes part of a much bigger system and can be added to data entered by another doctor in another place. And we’re also building it so that patients can enter their own data, some of their own data in this as well.

I think it’s really important. We should be learning from everybody. That will only speed up the process, if we can find a way and an easy way so there’s not a burden to patients and families to learn from everybody so that their data can be shared and anonymized with the researchers.

Seth: All right. We will pause for a moment to allow callers to get the information they need to ask a question.

Amy: If you have a question for Dr. Cudkowicz, if you could press number 1 on your keypad and that will notify us that you have a question for her. I know with all this information we’ve got a lot of questions out there. And if you need to call in, the number is 516-590-0363 and then press 1 to indicate you have a question.

Seth: Thank you, Amy. Merit, how has the resources you’ve talked about from the ice bucket been applied to reserach today?

Dr. Cudkowicz: A lot of the money has been applied to research, and not just in the U.S. but in all these other countries, Canada and U.K. The number of applications for grants has gone up four-fold in the field, which was just tremendous. There have been a few special initiatives, for example, the ALS Association earmarked some of the funds with this other partner, the ALS Finding a Cure, to fund trials for therapies that were ready to go into what we call Phase 2 study, the initial study to look for preliminary efficacy and that you got the right dose.

They’ve now funded three studies that without the ice bucket money, those studies wouldn’t have been funded. They’ve also funded collaborative groups that studied the basic cause of ALS. They’ve brought in new people to the field and new partnerships, researchers from other areas like Alzheimer’s or Parkinson’s and attracted them to the ALS field.

It’s actually impossible to list all the things that the ice bucket money has helped fund. It’s pretty huge. I think the other key thing is that it forged partnerships. So there are a lot of foundations now working together and saying, “All right, this is a big project that will answer some important questions, how can we work together and pool our funds so that we fund the best science rapidly?” That’s a huge change in the field.

Seth: Thank you. We will now take a question from our first caller. Caller one, go ahead and ask your question.

Caller: Is that me?

Seth: Yeah, it’s you.

Caller: Oh okay, great. Dr. Cudkowicz, in regards to these earlier questions, is there a mouse model being developed for sporadic ALS, a non-SOD1 mouse model being developed? Or part two of the question is, will the iPSC replace mouse model studies altogether?

Dr. Cudkowicz: Those are great questions. Right now, all the mouse models are based on different genetic mutations. And that’s purely because the field does know how to make a mouse model of a non-genetic form. But those models are important because a lot of biology that happens in genetic forms happens in the sporadic form. You can learn a lot about all forms of ALS from those models. People are also making fly and worm models as well.

But I think your second question with where the field is going, especially for sporadic ALS.  Can you use this new technology, this iPSC, induced pluripotent stem cells, where you take someone’s blood or their skin and you can make their own motor neurons? There’s a lot of effort to use that approach to study sporadic ALS. I don’t know yet if it will replace completely mouse models. I think we need to wait to have a few more drugs that work in people where you can then go back and see what was the best predictive model. But that’s the hope is that the iPSC approach will help us screen therapies for groups of people or individuals more effectively.

Caller: Great. Thank you very much. I’m in a study at ALS TDI, in your neighborhood, and it’s been over a year since I gave my sample but it seems that — well, they haven’t gotten my stem cell line created yet.  How long is that typically from your patient to give a skin sample until there would be new cell line generated from the iPSCs?

Dr. Cudkowicz: Very good question. So the time keeps getting shorter and shorter as the technology gets more widespread. It can take 20 weeks or so to go from the skin to the stem cell that the skin cell came from and then to the motor neuron. I mean, it varies by lab but it’s usually in that order of a couple of months. People are trying to make that even faster but it’s still a relatively new technology. It really requires some specialized skill. So there aren’t that many facilities that have the know-how to do this at the moment. I think that will change and it will get faster over time.

Caller: Great. Thanks for your answers.

Dr. Cudkowicz: Sure. Thank you for calling in.

Seth: Thank you, caller. I’m glad that we’ve talked about iPSC. Merit, are we making any progress in stem cell therapy?

Dr. Cudkowicz: Since the last time we spoke; we don’t have a lot of new data yet but we’re close. There’s a study by a company called BrainStorm that has now fully enrolled in the United States and is almost complete. People are waiting for the results of that trial. That’s the trial that uses people’s own stem cells taken from their bone marrow and then injects them in the spinal fluid and the muscle to see if they can deliver proteins that are good for motor neurons that can make motor neurons healthier.

Then there’s Neuralstem that also finished their second trial and they’ve reported out the results of good safety. They actually used some of these datasets that were available through PRO-ACT to compare how people did in their trial to natural history or because they didn’t have a placebo in their group. They reported that 7 out of 15 people in their trial had, at least by one measure, no progression over nine months. And then if you look at PRO-ACT, you would expect to see that more like in 20% of people.

That’s not the same analysis as when you have a concurrent placebo group but it does show the power at least in the early pilot studies of using large datasets, data on people as a comparison group to help you decide whether to go forward with the treatment or not. So that is now in the stage of thinking about what would be the next step that would be needed to get an approved therapy.

Whitney: Wait, Dr. Cudkowicz, is that stem cell therapy available here, and what stage is it in the United States?

Dr. Cudkowicz: Yes. So both Neuralstem and BrainStorm are investigational treatments. Neither today is enrolling participants but they both did studies in the United States. BrainStorm is waiting to have the results of the current U.S. trial to know what the next step would be. Neuralstem, we have their initial pilot results and they are working on what the next step would be, the next trial would be in the United States.

I don’t have any dates today of when this will happen, but hopefully, it will be soon. And a good place to look up about trials are obviously the ALS Association, the MDA listed, and our NEALS Consortium also lists. As soon as we hear a trial is going to start, we list it there.

Whitney: Thank you. We have an online question regarding Nuedexta. I realize that it’s for emotional things and for swallowing; can you use it when you have one and not both? Are they showing that it’s effective to swallowing issues? And then what are the side effects of it?

Dr. Cudkowicz: Very good question. In the clinical trial for swallowing and speech, it worked also in people who didn’t have any of the emotion. So it definitely works on these symptoms separately. If someone didn’t have any of the emotional symptoms and have only swallowing, you could try it for swallowing. It’s a fairly safe drug. There are certain medications you can’t take with it because of the part of Nuedexta that is called quinidine. You definitely have to check with your physician to screen the therapies you’re on to make sure they don’t interact with that part of Nuedexta. Some people get some stomach upset with it, but for the most part, it’s a very safe drug, but definitely one that you need to do with your physician.

Seth: Excellent. On another topic, we spoke in our last interview a lot about biomarkers. Do we have any new biomarkers for ALS in the last few years?

Dr. Cudkowicz: Yes. I think there are several new biomarkers. They’re all under development. It means that they are hopeful but they’re not 100% this is the biomarker for ALS, but there has been a lot of progress. And I’d say they’re in maybe three buckets. One is imaging, and I’d come back to that because in other fields that if you can see the disease, it makes it a whole lot easier to screen therapies. You can think of a cancer treatment. You look and does the tumor shrink or not? In multiple sclerosis, do the lesion shrink or not?

A lot of progress on imaging, and imaging in particular, something called neural inflammation. There are some studies again working with GE to look at inflammation in the brain and spinal cord of people with ALS. That’s looking at the moment like it’s potentially going to be a really good biomarker and a tool to screen drugs that block information. But there are also other efforts really globally to develop tools to the image of spinal cord, which is not an easy task but really important in ALS since a lot of the illness is in the spinal cord.

That’s one category. The other is I would say electrophysiology; measuring how the motor neurons are firing. There’s been some new tools developed largely in Australia called TMS to measure something called hyperexcitability. They’ve shown that sometimes even before symptom onset, you can start to have some hyperexcitability. They do that by looking at people who carry the gene mutation. But that very early in the illness, the motor neurons are firing too much. That gives us a way again to measure the illness, a biomarker of that, and then you can screen drugs against that.

And then the last one I would say is biomarkers in blood and spinal fluid. The most promising at the moment is something called neurofilament heavy chain, where it looks like that’s elevated in the spinal fluid of people with ALS. And it might be correlated to disease progression so that the more someone has of it, the faster the course.

Now a lot of the clinical trials are adding that as a marker to see if their drug can reduce the neurofilament and does that correlate with the improvement in the illness. And if we could find something like that, that means you can screen drugs quickly in a few people. If it lowers neurofilament, then you know that that might be the drug to bring forward fast. So a lot of progress, but I don’t think anyone say we’re done but it’s certainly a lot more than two years ago.

Whitney: It seems to me that there might be a hindrance in getting a lot of this information and people’s willingness and ability to travel to where the clinical trials are being done. Is there more of an effort being made for someone to do it at their ALS clinic and the information be gathered thereand sent to the central location so that information can be gathered and shared but it’s not dependent on person’s ability to travel or to relocate?

Dr. Cudkowicz: Yes. There are a couple of initiatives on this and I think we’re with a field where we need even more ideas. So a couple of things that are being developed are to make it really easy for any center to be part of a trial so if the doctor wants to on trial to lower the barrier. For that, for example, there are protocols that a doctor could just download from NeuroBANK to collect samples from their patients and put them in a repository like blood and spinal fluid and share the clinical data. And that there are some resources provided from this Foundation, ALS Finding a Cure, to make it easier for people anywhere in the country to be part of these biomarker studies. That’s one initiative out there.

The other, for clinical trials is, can we develop tools to measure the illness in the home using mobile devices so that people don’t have to come back to the clinic for their outcome measures to make it a lot easier for people? That’s all a new technology. It’s being done in other illnesses like Parkinson’s, and there’s a big team kind of thinking about how to do this in ALS, what will make sense to measure and how to do that but to bring the visit more to people’s home.  I think ultimately, that’s the way to lower the barriers; it can be done pretty much in your home and you don’t have to travel that far to be part of a study.

Seth: Thank you for those answers. I’ll have Amy remind us of the way to ask the questions.

Amy: If you’re listening online you can dial 516-590-0362 and press 1 to let us know that you have a question for Dr. Cudkowicz. Again, press 1 to indicate you have a question.

Seth: Thank you, Amy. So one follow-up question on the blood and spinal fluid biomarker. Is this being studied as a way of differentiating between quick or fast or a slow progress?

Dr. Cudkowicz: Yeah. The biomarkers can be used in different ways, and one is for kind of prognostication, what you described of, are there markers that are faster or higher or different in people who have a faster course or a slower course? And can that teach you something about the illness or give you a target for a therapy? That’s one use of the biomarker. Another is maybe even diagnostic. Is there a test like, for example, cholesterol for heart disease, something that you can measure in the blood or the spinal fluid that would say this is most likely motor neuron disease? So that’s another type of biomarker.

And then I think the third type would be a biomarker that measures the disease and then when you give a treatment, you can get rid of that biomarker. So that’s what a lot of the effort is focused on. For example, the neurofilament heavy chain I mentioned, the hope is that that might be a marker of the illness, and that if a drug gets rid of it or lowers it that that might be predictive of the clinical effect of the drug.

Seth: So can you lay out specifically, where the spinal fluid study that you mentioned is being led?

Dr. Cudkowicz: I didn’t entirely understand the question. Was it where the spinal fluid is?

Whitney: Where the spinal fluid study is being led. Where is it they direct from; where it’s headed?

Dr. Cudkowicz: I think one of the key things is we need to develop really large bio sample repositories of spinal fluid. There are some studies already ongoing. There is one that NEALS is doing at six centers where over 100 people now have enrolled and are kindly providing spinal fluid about every four months, as well as clinical data. And that’s a really important biorepository that’s being shared with scientists really all over the U.S., as well as in drug companies. But it’s a finite set of sample, so we’re always trying to again build protocols and bring in centers who will approach their patients and ask if they’d be willing to donate a spinal fluid for ALS research. I think it’s a really important sample that can really help the field move forward.

Whitney: That’s great. I think as an ALS patient myself, I’m often saying, “Just take it. Take my spinal fluid. Take my blood. What do you need?” But because of where we are located, we don’t learn about it or know that we should be sending those samples in. So this is great information. What is our greatest need today as an ALS community?

Dr. Cudkowicz: I think we’ve touched on some of them. It’s always hard to say what the one greatest thing is. I think we need to find a way that all patients or 80% of people can be part of research whether it’s giving a blood sample or spinal fluid or being in a clinical trial. That just becomes part of ALS, so we can keep learning and keep developing treatments. We have to figure out what were those, keep lowering those barriers so it’s easy for people to be part of research.

The other is we still need a whole lot of funding. I mean I expect it was tremendous and these other groups that are forming are great, but there are more questions and more ideas of how to conquer this illness then there’s funding available. It’s certainly a lot better than it was two years ago. I mean it’s fantastic now compared to two years ago where there were lot of ideas and was no funding. But I think we have to keep them advocating for money from the government and working together on philanthropy.

We make sure that the funding agencies are funding different things in complementary studies and no duplication. Those groups are all doing that. They are meeting around in circle and talking and comparing and working together to fund research. I think that’s also something that we have seen more in the last two years as well.

Seth: How, again, can we get more involved more actively in the process that you’ve mentioned?

Dr. Cudkowicz:  There are a couple of ways to get involved I think in different levels depending on how much people want to get involved. I think one, the local level of going to your ALS physician, and if they’re not involved in research, asking them to get involved and directing them to the NEALS website, and we can help them on getting these tools to be able to start doing some research. That’s one way.

And then the other, I think advocacy is still really critical. That’s at the government for funding to keep going to ALS. There I would work through the ALS Association or the Muscular Dystrophy Association. They have teams that are going down to the hill and advocating; but lending your voice. There’s nothing better or more moving than a voice of a patient to Congress and the people making the decisions about federal funding.

Seth: Thank you for that. We are thrilled at that. We have a great [0:46:45] [Indiscernible], but we are thrilled to have our season take off with you. Thank you for your ongoing work on our behalf.

Dr. Cudkowicz: Thank you for your role in advocacy. It’s really a pleasure to be on the call.

Whitney: Our next ALS Crowd Radio episode will be in one week, Tuesday April 19th at noon, Mountain Standard Time. Dr. Joseph Beckman from Oregon State University will be our guest about stopping ALS in the lab.

Seth: Okay. We want to thank Dr. Merit Cudkowicz for her time today and we look forward to solving this puzzle we call ALS. Thank you, Merit.

Dr. Cudkowicz: Thank you very much. Have a good day.

Seth: All right. Take care. Bye.

About Author

Seth Christensen

Seth is an ALS patient and founder of ALS Crowd, a division of the CrowdCare Foundation. As host of the ALS Crowd Radio show, he interviews top ALS researchers and focuses his efforts on the aggregation of big data to help researchers and patients find clues that will drive to a cure.


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