How Do You Spell Progress in CMT Research? STAR!

Gilles Bouchard’s passion to advance CMT research started when his son, Yohan, was diagnosed with CMT.

On the 10th Year Anniversary of the launch of STAR (Strategy to Accelerate Research), CMTA Chairman, Gilles Bouchard, gives a webinar explaining the current status and remarkable advances made in CMT research. This webinar was so informative and well-done, I transcribed it for you. Spread the word folks. Pretty amazing work being done right now.

The actual webinar can be heard here: https://www.cmtausa.org/resource-center/learn/cmta-webinars/

 

Never before has the CMTA had so much treatment-driven research going on, and we continue to grow, expand and reach new people and partners. Please read up and I’ll give updates as the come down the pipeline. So thankful for our clinicians, scientists, volunteers for making this work possible. Thanks to all our generous supporters who make this vital work possible.

Webinar Transcription:

I’m the chairman of CMTA, and our family’s been involved with CMT and CMTA for almost 15 years. Our son Yohan was diagnosed with CMT about 15 years ago. So it’s been quite a journey for us, as for many of you, I’m sure. What I want to do today is give you an overview of the STAR program.

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As you can see from the logo, we are celebrating the 10-year anniversary of STAR.  STAR has been very successful, offering promise and hope for the 3 million people worldwide with CMT. Nevertheless, for people like you and for me, 10 years feels like an eternity when there are still no viable treatments for CMT. On the other hand, 10 years is actually a short period of time when it comes to developing drugs.

About 10 years ago, we had a CMTA board meeting to discuss our approach to research. We realized that while there was some good research and a few really brilliant researchers working on CMT, there wasn’t really a lot going on in terms of research that would bring drugs to market. And we asked ourselves, how do you accelerate research?

It’s extremely challenging to develop new drugs. On average, it takes 10.5 years from the time a drug is identified to get approval. The clinical part of this 10-year process takes about 8 years. Even scarier, 90% of the drugs fail during the approval process. And it costs hundreds of millions of dollars ……

 

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So we asked ourselves, from a business point of view, how to accelerate this process?

From a research point of view, let’s look at the some of the positives about CMT:

  • The causes for the most common types of CMT are very well known, and more types are discovered every year, which is not the case in many diseases, especially for the nervous system. We can create animal models. We can create assays or cells in Petri dishes so we can create stem cells. Now we can test and create models of CMT, which are very valuable to our partners.
  • The other positive is that CMT is classified as a rare disease, which gives companies some advantages in the market. Although CMT is rare, it’s still fairly common and there are a large number of people with CMT,  which is helpful for companies because obviously it’s still a big market and it allows us to have enough patients for clinical trials..
  • Since the CMTA has these models, we can take advantage of the latest developments in genetic and neurological therapies. As you know, since the genome was sequenced, there’s just been an explosion of technologies and applications, and many of those actually apply very well to CMT. So that’s very positive for us.

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What makes it interesting for a pharmaceutical company to work on CMT?

  • Testing Infrastructure – We have  developed a very extensive testing infrastructure. We created animal models, assays (tests). We created stem cells when they were needed. We also work with companies who specialize in testing animal models and analyzing tests. It’s what we call the STAR testing infrastructure, and it’s a very powerful infrastructure. In fact, most companies approach us because they’ve heard about this testing infrastructure, and they want to work with us on testing their compounds or their drugs or their ideas. And you’ll see, as I go through the details by each disease, I’ll give you examples of the things we’ve done there.
  • Key Opinion Leaders (KOL):  Companies know their drugs and technology well, but they might not be experts in CMT, so they won’t have access to key opinion leaders, who are experts in CMT. This is why we built such a strong scientific advisory board – they collaborate with our partners and advise them on CMT. This creates strong teamwork and complementarity between their knowledge and the expertise of the companies.
  • Community Access. I always say that the CMT community is our biggest asset. Companies want to talk to the community to better understand the disease. They want to understand what’s called the burden of disease, how it affects people, how it affects people’s lives.

Almost every company tends to have dedicated resources to engage the community. Our CMT Association is a huge asset for us as we reach many people worldwide. And again, we thank you all for just being part of that.

 

  • Clinical Infrastructure: Once companies are done with the testing in animals or in assays, they want to test in humans and bring drugs to market. And this is where drug development becomes very expensive. Again, hundreds of millions of dollars are spent in clinical trials. And the more people you need, and the longer it takes, the more expensive it is, and that can often be an inhibitor for companies to work on the disease. It is especially challenging in CMT, because it’s a slowly evolving disease.

 

So how do you tell that a drug is working in a slowly evolving disease without spending years and years following a large number of people?

We work with the INC (Inherited Neuropathies Consortium), started by Dr. Mike Shy, who has involved many world-renowned clinicians and scientists. They had the vision to create this infrastructure like the Centers of Excellence (COE), where they see people with CMT. They do what’s called natural history, where we see how the disease evolves over time with patients, and then we develop biomarkers or ways to measure the progression of the disease.

 

The whole idea behind biomarkers is that we want to run clinical trials in just a few months with a handful of people, and get a good quality read whether the drug is working. And we’ve made tremendous progress here. And that’s a really attractive part for companies now.

 

Financial Support – We’ve learned that we have to be very flexible, and we have to provide the kind of support that fits their business models. So we work together on joint projects or  even give them access to some funding through investors.  We’ve learned to be very flexible on how we can help companies where needed.

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STAR is based upon five core business principles:

Partnerships: We have announced five partnerships. The one thing we look at in terms of the measure of success is how many partners are we able to attract, how many phone calls we get a month about companies interested in working on CMT. And frankly, the last two years, it’s just exploded. So, besides the five that we’ve announced, there are  actually eight more companies that we have not announced because they want to remain discrete right now. Again, they want to wait until their results are published to go public.

And we have a pipeline, meaning that we are in early discussion with another 15 more companies. Three or four years ago, there were only a handful of companies who were working on CMT. So this is probably the most exciting part as it shows that STAR’s starting to work because there is a long list of companies actually doing some work on CMT, and an even longer list of companies interested in trying to figure out how to work on CMT.

 

For reference, we also have five wonderful partners who help us work with pharma companies through all the testing procedures.

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Sanofi Genzyme – is a very big company, and we started by screening their whole library, and basically, we’re down to one compound now, and we’re in the process of testing it, and we should know very soon whether it works successfully, and then we’ll decide what is the next step on this one.

But even more exciting is that we’ve actually grown that relationship, and we have now three other projects with Sanofi. There’s another family of compounds that they’re very interested in, and we’re actually testing those as well in CMT right now. They’ve also done some really good work with us on biomarkers, which hopefully will be published soon.

Lastly, we’ve done some work on what’s called target discovery where we have worked together on finding other potential drug discoveries or other compounds that targets  CMT, and this has led to additional projects.

 

Ionis – You may have heard about the breakthrough with Ionis last year. They used components called ASOs, which go straight to the gene and really counteract the negative effect of having a duplicated PMP22 gene. That was a major breakthrough because they were able to stop and sometimes even improve the situation in two different animal models. This was probably the best example of showing that when you reduce PMP22, it actually helps the behavior of the animals – a breakthrough!

Now, they are basically working very hard in trying to design a drug for humans. Even though they have the drugs for animals, it’s obviously a lot more complex and challenging to develop the drugs for humans. They want a drug that’s very efficient, so that it can get to the nerves. We’re also in discussion with them about clinical planning.

 

InFlectis – We’ll talk in the context of 1B and 1A. Inflectis is a company in France that has developed a very interesting compound that has shown some exciting results in 1B and also in 1A.

Acceleron – You might have heard of Acceleron because we’re actually recruiting for clinical trials right now. They have a drug that helps rebuild muscles, so while it’s not a direct cure for CMT, it could really help people with muscle wasting caused by CMT.

Regenacy – one of several companies that’s working on what’s called axon degeneration. It’s a field that’s seen a lot of development the last couple years, where people try to find out ways to stop the degeneration of nerves.

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To be the most effective with our resources, with our money, we find initiatives that cut across all diseases. Personally, I think it’s strategically something that is very important, and guess what? There are five of them.

Gene Therapy – You may have heard about CRISPR and gene editing. It’s a field that’s been around for quite a while, but it’s completely exploded over the last two or three years. There are close to 1,000 clinical trials in gene therapy around the world.

Currently, these efforts have only produced 2 new drugs; one of which which was approved in the U.S. less than a year ago for Spinal Muscular Atrophy (SMA). As we continue gene therapy research inside the lab, our ultimate goal is to transform it into effective treatments for people living with CMT. I encourage you to watch the PBS documentary: “The Gene Doctors” to understand my excitement, as it is clearly going to be a big part of our future.

Gene Replacement therapy also will be applicable to CMT. With gene therapy, scientists  take genetic material, put it in a virus, and the virus goes in the nerves, and this genetic material kind of compensates for the gene that’s either missing or deficient. This suits CMT very well because we know the genes that are deficient, and we have models where we can test it. So people are very excited about applying gene therapy to CMT.

Now, a new branch of gene therapy that’s exploding and is relevant to CMT is CRISPR also known as gene editing. Here you don’t just put an additional gene material in the cell, but you actually go in there and change it – change the genome. 

Axon degeneration –  it turns out that there are a couple of really big markets that are driving this. One is chemotherapy, which creates a lot of neuropathies for people, and the other one is Diabetes. So a lot of companies are working on these diseases, and they are very interested in applying it to CMT.

And remember, from a business point of view, it’s always very interesting for a company to have a rare disease indication for a medication because they get the advantage of the rare disease while having the bigger market. So there are at least five companies we’re working with right now on different approaches to axon degeneration, and we’ve also launched several specific studies that we’ll talk about per the various diseases. And one of the beauties of axon degeneration is it could apply across CMT types, even the ones they are not diagnosed.

Models: It’s incredibly important to have quality animal models, usually mice, sometimes rats, and cell models or assays and stem cell. We can take people’s skin samples and make stem cells, and then we can make neurons or Schwann cells (Schwann cells make myelin) out of them.

Gene discovery: As you know, we’ve discovered a lot of CMT-causing genes. I think we’re up to 120, but there are still many types of CMT which have not been discovered, especially Type 2’s. It’s very important to continue that, especially in the context I mentioned earlier, that more and more of the new therapies are very gene-specific. So we need to continue to discover more genes.

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Biomarkers – We are collaborating with the INC, which CMTA supports directly. Most of the funding for the INC comes through the NIH, but both the MDA and the CMTA provide additional funding, and then we have also funded several specific projects on top of it.

Biomarkers are important to track disease progression over time. Scientists are looking at elements in your blood. For example, Dr. Mary Reilly’s lab is looking at neurofilaments and her promising work is being published.

And then another project, again, in the blood, looking at what’s called microRNAs. This was funded by CMTA with Drs. John Svaren and Mike Shy and another company that we’re working with on this.

We are also looking at skin biopsies because you can actually detect levels of PMP22 in the skin. The CMTA is funding this encouraging project, spearheaded by Drs. Svaren and Shy.

We are also looking at MRIs of the calf muscle and looking at the fat content in the calf muscle, which correlates to disease progression. So, when you put all the three approaches together, we now think that we can run clinical trials over a shorter period, six months, with maybe as few people as 50, which is huge progress from the past when we needed hundreds and hundreds of people.

 

We’ve done a lot of work in CMT1A, and now we are funding efforts to address the other most common types, so you’ll see a lot of this for 1B, 1X, 2A, and other types. Now those biomarkers are again, elements that we look at in the blood or in the skin, but also, the FDA wants to see what’s called functional outcome measures. They really want to see the patients getting better. For example, with treatment, are people walking better, getting better use of your hands, etc….. So the team has been really busy coming up with the scores, which are functional in nature and which try to capture how much better people are or worse people are as their disease evolves, and try to do it in a way where we can see small changes, and also in a way that correlates with those biomarkers. The good news is there’s a major grant that was just approved by the NIH for the next two or three years to continue this work, so I think we’re going to be in very good shape.

We are also measuring activity in people’s homes with wearables.  We’re looking at a proposal right now to look at some sort of wearable device where we could measure people’s gait, et cetera, when they’re home. This would give us an indication about  diseases progression while at home which would give us a longer timeline and more information. .

 

Disease by Disease

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CMT 1A –  So, as you know, CMT1A is created by the duplication of the PMP22 gene, so the most obvious thing to do is to try to decrease the level of PMP22 in the body. We have the small molecule screen going on with Sanofi. We have the ASO approach with Ionis. We are now working with another company who actually wants to use CRISPR to decrease PMP22, so that’s very exciting, and actually, those tests are just about to start. And then also we’re looking at a very interesting proposal to kind of combine a gene therapy approach with an ASO approach where you would use a virus to send a little piece of RNA into the cells to try to interfere with the PMP22.

Now, besides the pure focus on PMP22, there’s been a lot of work done over the years to see this with other targets in the body that the new drugs could go after to help with CMT1A.

One in particular that’s shown some promising results is called P2X7, and we are working with two companies right now with drugs that target this. And they actually both are in testing right now.

We’ve done work to identify new targets, and we are actually funding a project that’s in full speed right now to look and see if there are other elements that we can identify that could help decrease PMP22. And then we’ve mentioned axon degeneration. This applies to all CMT types. If the work progresses well with other types of CMT, it could be very applicable to CMT1A.

We are doing the muscle regeneration work with Acceleron.

 

About two or three weeks ago, a company called Pharnext announced a Phase 3 clinical trial for CMT. The early results of their tests were encouraging. They showed that in some patients the disease improved, in others it was stable, and, sure, in others it got worse. But it was clearly better than the control group and the placebo. I think the general consensus around scientists is that this is interesting, and we want to see a lot more of the data as it becomes available throughout next year.

They will have to do this, obviously, as they engage the FDA, so it will be very interesting also to see how the FDA engagement happens. And we think all this will happen through 2019, so again, very interesting announcement.

So we all have to really help them in a way because they are defining how the FDA and the agencies think about CMT. Again, nobody’s been there before, so we’re working very closely with them, and all the patient advocacy groups are really involved.

It’s our first chance in a way to make the voice of the CMT patients heard to the FDA or to the European agencies, so it’s very important for us to all work together and basically make the voice of the patient heard. So this very first drug is going through this right now.

CMT1X

 

 

 

 

 

 

By the way, on the right is the CMT1X gene, and I think there are over 400 different mutations of the CMT1X gene.

There was a study on natural history that was just published. I encourage everybody to go to the Centers of Excellence and to go back because then our scientists can not only study your CMT, but see how it evolves over time, and that’s what’s called natural history. And this is one of the most important things that companies and pharmaceutical companies want to see, to really help them figure out how the disease evolves after the drugs get administered to people. So, there’s good data on 1X, and I think we’ll continue to see it happen in also in other diseases, but we need more and more patients to do that.

We just approved this year the funding to develop new mouse models of CMT1X. Pretty much all the work on 1X has been done with a mouse where the whole gene was removed, and it’s actually a very good model. But we feel it’s time to get more precise models with specific mutations, especially as new  genetic technologies are coming into play. So we’ll need better models that we can just look at the specific gene mutations. So this is being funded, and it’s in progress.

We are also co-funding a Dr. Kleopa’s gene therapy project with the MDA . He has shown some really good results in mice, where he was able to get the gene material to the Schwann cells in the mice, and it did have an effect. So we continue to push on this project, and we try to find ways to make it more translational, meaning – how do we bring this to humans as quickly as possible.

 The role of inflammation for CMT1X was highlighted by Dr. Martini in Germany. And there are several companies that we’ve talked to that have drugs that target the element that he identified as being part of the inflammation. The challenge there is that it’s hard to find a drug that doesn’t also affect your immune system, so we are in discussions with drug companies to see if we can find a drug that does go after this inflammation factor but doesn’t also wreak havoc in your immune system.

And last but not least, axon degeneration, is very important for 1X, as it is one of the types of CMT where we see fairly severe axon degeneration, so we are evaluating right now a proposal to actually do a specific experiment to try to highlight the role created by axon degeneration with a specific target.

 

Now let’s move to 1B which has about 200 mutations which fall into 3 categories, and we have good mouse models for each one of the categories of CMT1B – early onset, mid onset, and late onset. We  continuing to do some testing with InFlectis at the animal level with them, and we are in the early stages of clinical planning.

 

By the way, if you look at the picture on the bottom left, you see three little pictures. The left one shows the myelin around the axons for a normal mouse. The middle one is a 1B mouse, so you see it’s really degraded. And on the right side is a 1B mouse treated with Sephin, so that’s why people got really excited about Sephin because it showed some really good results in the mice. And the other good news is InFlectis with their drug  Sephin were recently approved for the Phase 1 trial in Europe. Phase 1 is when you actually go to healthy patients and just test the drug for safety, so if you combine this with the animal model trial we’re doing with them, pretty soon after that, it’ll be late next year, if it’s all positive, they might be able then to move to clinical trials in humans, which would be very exciting.

The other part of the puzzle is called UPR, the unfolded protein response, and because Sephin seems to be acting on this UPR, (gene makes the protein form in a different way). Dr.  Mike Shy analyzed all the CMT1B mutations and figured out the ones that were affected by UPR. So those were the ones that would be most likely candidates to work with Sephin.

There is encouraging results using Sephin on CMT1A. We could potentially have clinical trials with both diseases if all goes well.

Axon degeneration is also very applicable for 1B. In fact, we funded a project which is active right now, just like in 1X, to see basically if the specific target has an effect with CMT1B. We should have the results on this probably early next year.

CMT 2

I put all the CMT2’s together because we find a lot of technologies now apply across CMT2s. So why is this so exciting? Basically, a few years ago there wasn’t much going on in CMT2, and all of a sudden we see a lot of activity. And this is because two interesting things are happening. The first one is all those new genetic technologies  tend to apply really well for CMT2 because, if you recall, CMT2 affects neurons, and the genetic material of neurons is actually located in the spine. 90% of your neurons are in your central nervous system, so the people who are working on those technologies for the brain or the spine are very familiar with neurons, and also they’re very easy to get to and to deliver a drug to. So they really want to start to try that on CMT2s.

And the other part, based on our strategy, is that we have developed some really good models of CMT2. We have two excellent rat models which our partners really love. There’s a good mouse model of CMT2E that’s been around, and we also have some good stem cells. So all this coming together shows that there’s a lot of interest in CMT Type 2. A lot of those technologies apply across CMT2 and will carry over to CMT1, but it’s a good place to start with a lot of those new technologies, so let’s go through it.

Gene therapy. So a lot of focus on gene therapy, especially for CMT2. What we did is we put together a summit in Baltimore this summer, and it was really incredible because we asked the world experts in gene therapy to join us to help us basically not only educate ourselves but to also help us build our strategy. In the room, we had a dozen of the world’s leading experts in gene therapy. It was really amazing to see the quality of the people, their willingness to come work with us, and also the openness. They were sharing very openly, and it was incredibly productive. And basically, what we’ve been doing since then is we’ve been working with them to define a very specific gene therapy strategy, which we’re not quite ready to communicate in detail, but there’s a lot of activities, a lot of partners involved, and it’s something that’s really, really exciting to us because it will probably start in a couple of CMT2 types and may have broad applications over time across all CMT types.

And also I want to mention that there’s a family that’s done some really amazing work on CMT2D, and they are pushing a single-patient initiative. And they’ve approached us to work together with them, and we’ll support them wholeheartedly, because they’re also helping pushing the envelope.

Axon degeneration –  we have an experiment going on with a partner on CMT2A that holds promise and we also have other companies that are willing to work with us on this, so very exciting.

Now, CRISPR, that’s another really interesting area. And I’m sure you’ve seen it on TV, you’ve heard a lot of the discussions about it. We’ve been approached by UCSF, and you might have seen their video about CMT, but they are really interested in working on CMT, and they really want to work with us. And they were part of our last STAR meeting. We have STAR meetings twice a year. The last one was in San Diego in October, 2018 and it was just an incredible meeting. I think all the scientists there were just glowing about how this was the best meeting ever and the quality of the new people coming in, and the CRISPR folks were there, and they really lit up the room with their ideas.

Still a bit exploratory, but fast-moving, is a technology called CRISPR surgery where you take blood samples from a given patient and then from those blood samples, they will grow them into stem cells. Those stem cells they will grow into neurons, and then they will then edit those neurons with CRISPR to remove the defective gene or fix it. And then they will reintroduce this into the body.

We also talked about the need to continue to discover new genes, especially those CMT2s, so we actually increased our funding for Dr. Zuchner in Miami, who’s doing an incredible job with his database, and he’s discovering genes almost on a weekly basis.

And then a couple of disease-specific initiatives. We actually did a screen of approved drugs for CMT2A. Unfortunately, the hits were not that convincing, so I’m not sure we’re going to follow up with that, but at least we did it, and we have a good assay to do that. And then there was a paper that came out last year that showed some specific drugs could really help with the activity of mitofusin, and we are basically doing a pilot program with them to try to dig into this further, so this could be very exciting for CMT2A. And in CMT2E, thanks to the work that was done on creating the stem cells, we are just starting to do some drug screening using those stem cells, which is very exciting.

 

Some people asked questions about CMT2C because the gene is the same as on some version of SMA. They were asking about the SMA drugs that has been marketed. But, again, and I’m not a specialist here, but the SMA drugs that are on the market are for a different gene than the one that is just CMT2C now. There’s some good work going on in CMT2C in a couple of centers in the U.S., and some of those things we’re talking about would apply across all of the CMT2s.

 

CMT4. Now, CMT4 is very well suited for gene therapy because it’s monogenic, but also because, remember, CMT4 is recessive, so both genes are affected, so there is what’s called loss of function, and this should be very attractive for people working on gene therapy because they can replace all the genes.

 

So we’ve had this project with Dr. Kleopa co-funded with the MDA that was actually showing some promising results in CMT4C that’s been included, obviously, in our Baltimore discussion. And then, again, there’s a family doing a wonderful job on CMT4J pushing also a gene therapy approach, and we are in discussion with them how we can work together and support them. So a lot of exciting things for CMT4 on gene therapy for CMT4, and of course, axon degeneration would apply there as well, then gene discovery as well.

 

We’re not done yet. We have a lot more work ahead of us than what we’ve done, so it’s really important to support STAR. And I just want to explain to you, from our perspective, why it’s so important and more important than ever to support STAR.

 

Leading Program – It’s a really exciting program, and a lot of partners are coming to it. Again, people speak by joining us, and the list of companies keeps growing, and there’s no better measure of success and credibility for me than this. But besides this, we’re also very careful about our resources. If you look at our financials, they’re really best in class, and we continue to improve them.

 

Best-In-Class Financials – If you look at most organizations, they tend to have overhead levels in the upper 20’s. CMTA historically was around 20%, which is really good, if you look on Charity Navigator, it’s actually quite good. But we’ve actually improved it. We made a lot of tough decisions the last two years because we want to continue to improve that. And if you look at last year’s financial, we were down to 12% as overhead, which is absolutely world-class, and I’m really proud of the team. And we actually think we can sustain kind of this low-teens level looking forward, so a very, very world-class performance here.

 

Decision Makers – And the other thing that is very special that I really am proud of about the organization is the people who are making decisions, the board members, are also major donors. So in a way, people put their money where their mouth is. About 20% of revenue comes from the board itself, so the point there is we’re going to take care of your donations very well because ours is there as well. So we are all major stakeholders here. We’re all in. We believe in it, and again, we speak with our own money and own fundraisers, and this, I think, is very unique about this organization that is very special. And I can’t say enough about my colleagues on the board and their dedication and the amazing work they do. And it’s recognized, and we work very hard for this.

 

Recognized: We just got upgraded to platinum by GuideStar. With Charity Navigator we’ve been in a three-to-four star range for the last few years, even though they keep raising the bar. And last year, for the first time, they gave us a perfect 100% rating on transparency and governance. We’re very proud of that, so it’s very important to look at those third-party evaluations.

 

Multiplier Effect – Because we work with partners, the heart of our strategy is that when we spend one dollar, it’s really to incite partners to come in and spend 10 times more at least. So when you give a dollar to STAR, obviously, we take good care of it, it’s spent very wisely, but also it brings in partners that spend the big money, and our partners spend tens of millions of dollars on CMT, and this multiplier effect is really important.

Remember, we don’t have the hundreds of millions of dollars that it takes to develop the drugs.

This is a very challenging but also very personal journey me. That’s why I wanted to show a picture of Yohan here, my little hero. He didn’t choose to have CMT, but he’s the reason why I’m involved, why we’re involved, and why we have a passion around this. And I think for all of you on the phone, I’m sure it’s the same thing. You all have a personal journey, so the point I want to make is, 10 years ago when we looked at the situation, it was bleak. It was like staring into the abyss. There wasn’t much to do. It’s not the case anymore. There’s a lot everybody can do.

 

Everyone can join the movement. Please get involved. Everybody has a role to play. These are exciting times, but there’s a lot of work to do, and we need everybody on board to help us.

 

The CMTA has branches, we have fundraisers, we have walks. Just please make CMT your cause and help us. And also, help yourself. Make sure to join the INC Patient Registry so you’ll be there when clinical trials come along. Go visit a Center of Excellence, and go again. This is how we get a natural history. It’s really important, especially for the less common types of CMT. We need more and more patients so we’ll be ready for clinical trials.

 

And then when I started working on nonprofits, people told me about the three W’s, and I think that applies to everybody. So we can all help through work, through wealth, and through wisdom, and hopefully all three or a combination thereof. And then all together, we’ll move this forward.

CMTA RESOURCES

 

 

 

 

 

 

The work the CMTA is doing right now on all fronts is nothing short of impressive. Please get involved!

http://www.cmtausa.org

 

3 thoughts on “How Do You Spell Progress in CMT Research? STAR!

  1. I read this blog on CMT this afternoon and as a new supporter to finding a treatment for my nephew Yohan and the thousands of other’s afflicted by this disease I am proud to be a part of initiative to give hope to others around the world. I learned this summer of the power of spreading the news and asking for others to donate to the cause. It certainly came as a pleasant surptprise to me when I had to up my goal twice. It wasn’t a huge amount but my point is by getting others to step up that as a community this battle can be won together. Thanks you Elizabeth and Gilles for your leadership. I’m looking forward to our next CYCLE 4 CMT event in Vermont this year. ( http://www.cycle4cmt.org )

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    1. Love You. Love your commitment, George. Thank you for sharing and spreading the word. Amazing what we are accomplishing! And, the new scientific technology available now will serve only to enhance and advance treatment driven research!
      xoxoxoxo

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