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Watch Scott Johnson 's recent MS World Chat on the Promise of Myelin Repair Research

Watch the video recording of the chat session with the founder and president of Myelin Repair Foundation, Scott Johnson.

Full transcript with Q&A on MS World.

Transcript of the MS World Chat.

Carol Menaker:
Good evening and thanks for joining us for the MS World Chat.

And thank you to Ken Day of MS World for setting it up and for inviting us to talk about the Myelin Repair Foundation.

Before we get started if you are interested in watching this chat streamed live on video you can visit http://justin.tv/myelinrepairfoundation.

We are pleased to be here tonight to share with you more about Myelin Repair Foundation and the progress that we are making toward developing a therapeutic for MS through myelin repair.

And equally important is we want to talk to you about how we are doing what we are doing.

Because not only are we focused on developing a therapy for patients but we are also focused on doing it as quickly as we can possibly do it.

We’re getting faster discoveries out of our scientific teams and moving those discoveries into therapies for MS patients.

We also wanted to thank all of the folks who sent questions to us in advance of this chat.

Questions like: When will a therapy be available? What form will the therapy take? What will the therapy cost? And as much as I wish we could answer those questions tonight, we just aren’t there yet.

In the last 5 years we’ve learned a lot about myelin repair. We have learned how to affect myelin repair in tissue assays in animal models,

but that’s just the beginning of a process of understanding whether these therapies will be safe and effective in humans.

So, as we move forward in our process, the answers to your questions will become more obvious. But, today, we are just not there yet.

Even though we can’t answer those questions specifically, we hope that learning more about myelin repair and the promise it offers to MS patients as a next generation therapy will be useful for you. And we hope that you’ll share that with your friends and family.

And with that, I’d like to introduce you to the founder and president of the Myelin Repair Foundation (MRF) – Scott Johnson.

He’ll be talking about Myelin Repair Foundation and the progress that we are making toward therapeutics for MS.

Scott Johnson:
Hi,  my name is Scott Johnson. And like many of you on this chat I have MS.

I was diagnosed with MS 33 years ago, back when I was 20 years old. (1976)

I was living with MS for a very long time, and like many of you looking for a treatment/cure and hoping for something helpful.

Unfortunately, like many of you I have been pretty disappointed over those years with the rate of progress and the treatments that are available.

But, I’m fortunate that I could still walk and get around. I was lucky compared with many with MS. That allowed me to pursue a business career.

(Side note: Scott started up and was CEO for three Silicon Valley companies and worked at Boston Consulting Group)

So, I guess I tell people that there are two things that have been big turning points in my life: the first was when I was first diagnosed with MS, and the second was when I read an article in Business Week (2001) that actually talked about myelin repair for MS.

That was very significant to me. For the 25 years prior to that, all I ever heard was looking for a cure for MS

My understanding was that the Central Nervous System was NOT repairable. That it wasn’t even an option.

So, when I saw that article, obviously it caught my attention, got me interested and I contacted the scientist at Yale who was mentioned.

I asked him a lot of questions about what this meant and what the possibilities were.

He suggested many other scientists for me to talk to.

For the next 8-9 months in my spare time I talked to a large number of scientists.

Two things became very clear:

First, there have been recent discoveries in the late 90s that the CNS was not static. That it’s “plastic” – it can continue to grow and mend itself.

In healthy people, their myelin is replenishing and repairing itself. The repairing of myelin can possibly explain the relapsing remitting nature of MS in the early stages for many people.

What was encouraging was that a myelin repair strategy was potentially not as difficult as you’d think, since you’d be trying to allow your body to do something that MS is preventing – not trying to get your body to do something that is unnatural.

It appeared that myelin repair was an interesting and viable approach. But back in 2001-2002 it was very understudied. It wasn’t receiving much research money from the NIH or from any disease organizations. Very few scientists actually pursuing myelin repair.

As Carol mentioned, the biotech and pharmaceutical companies that could turn something into a treatment were ignoring myelin repair as well.

The 2nd takeaway: the more I spoke to scientists about how their world operated and how medical research was conducted, and as I spoke to people in industry  and learned about how industrial research was done, I found that there was a huge gap between those two.

It’s an incredibly inefficient system, which explains why the process of coming up with effective new treatments is just so slow.

Based on my business experience, it appeared to me that if you applied more of a business model to research and this whole process that you could eventually have a dramatic impact on speeding up things.

The objective of setting up the Myelin Repair Foundation was really two fold. 1) To focus specifically on myelin repair for MS and greatly accelerate that work, and 2) To pioneer and entirely new process that could be used to accelerate research for ALL diseases.

Basically over the past five years, that is exactly what we have done. We have come up with ways to overcome the shortcomings of the process (I’ll talk about those in a few minutes)

and we have interacted with biotech and pharmaceutical companies to help them see that  myelin repair is a viable therapeutic area that they should invest their own funds in to bring treatments to MS patients.

Because the system is relatively unknown to most people, I wanted to use this chat time to talk about how the current system operates and how the MRF operates – how we are different and how we address the shortcomings of the traditional model.

There are several elements of the current system that don’t make sense:

There are thousands of scientists out there, and if you are a scientist that has an idea that might advance research you basically have a question that you want to answer. In order to answer that question you have to design an experiment.

To do that experiment requires time and money. So you need to apply for a grant – (i.e. NIH or the MS Society) proposing for the funds to run your experiments.

In general the time it takes from generating the idea for the experiment, postulating the experiment, applying for funding, receiving the funds, conducting the experiment (couple of years), writing the experiment up, waiting for it to be peer reviewed to be published, and finally publication – takes around 4-6 years.

What happens is that someone else reads that paper. They may have a question that occurs to them. That would then take the next step in moving the research along.

So it’s a very sequential, serial process which is relatively slow. But what slows it down even more is the peer review process.

The peer review process involves a peer review committee which is a small number (3-4) of scientists at NIH, MS Society or other disease research organization who scientifically evaluates the proposals. They decide whether it’s “good science” or not.

Unfortunately, if any one member of that peer review committee doesn’t understand why the science might work, it won’t get funded.

So, if a scientist has an idea for a breakthrough experiment, that scientist will not submit a grant application for that experiment because they know that it will not pass the peer review process.

That means that the experiments that are funded are very incremental (not breakthrough experiments). So not only is this process serial and sequential, but also incremental. These elements slow down the process.

The next issue that we saw is that science is really an individual sport. The only capital for scientists is their ideas.  So, they need to keep those as close to their chests as possible until they publish in order to get credit for them.

In the academic world, it’s all about getting tenure. To get tenure you have to publish. And to publish you have to get grants. Throughout that process you have to keep your ideas to yourself.

Yet to solve a bigger (more complex) problem like myelin repair you need a variety of areas of expertise. Today, there is no mechanism in academia where teams of scientists, with a variety of expertise, can really collaborate and work together as a team.

Plus, what has happened over time is that in order to do “good science” you have to get more and more narrowly focused (more specialized).

So what you get is narrow silos that don’t talk to each other which makes it difficult to solve bigger/ more complex problems. That is a real problem.

As a solution to this problem, the MRF set out to create an environment where brilliant scientists in  different areas of expertise could truly work together as a team.

In 2003-04, we pulled together the best team of brilliant scientists and worked with them as a team.

Another element that we saw was missing in the current system was having a plan. That’s a real problem, because any of you who have been in business know, or actually if you try to accomplish anything, if you don’t have a goal or a plan to achieve that goal you’re likely not going to achieve much, to be honest.

The way that academic research has been set up is as an individual sport. Scientists really can’t really have a larger  goal because there is no way that an individual scientist could accomplish that goal on their own.

We at MRF thought it was not only important to have a specific goal (to do science that would result in therapeutic treatments), but to have a plan to achieve that.

One of the first things we did after pulling together our team of experts was to work with them to put together their research plan.

There are a few other elements that are issues as well, but I don’t have time to get into those. They include intellectual property (IP) – if you don’t patent findings that come out of your experiments then there is no way that companies down the road can justify investing the hundreds of millions dollars necessary to commercialize treatments.

Our model takes care of all the intellectual property protection so that companies can move the discoveries that we do make forward.

Another area that is different in the MRF model is that we actively manage the scientific effort. That’s very important because we want our scientists to focus purely on science. We can do that by taking as many tasks off their plate as possible.

This is something that no other disease organization does, nor does the NIH.

In summary, what the MRF does that is unique is 1) we have a very specific goal – to develop myelin repair treatments for patients, 2) we pull together a team that has all the necessary areas of expertise on it to execute 3)  a research plan that we have put together. The research plan is constantly updated. 4) We also have a process for how that team works collaboratively, 5) we protect the intellectual property, 6) we provide all the resources for the team, not only money but other types of support and 7) we actively manage the process w/ very specific metrics and goals.

I think to that last point, we’ve done something that is incredibly unique. We aren’t aware of any other nonprofit that has stated the goals we had. 

Back in 2004, when we were organizing we had talked to many scientists about how soon a myelin repair drug target would be ready to be licensed to a company to turn it into a treatment.

The consensus was 15-20 years.

As we worked with our team, we challenged them to do that faster. We asked them for a commitment for how fast they thought they could come up with a treatment ready for license.

Being academics, they were very thoughtful with what they came back with. They said, “we think we can do it in 5 years. We think we can shave 10-15 years off of  what the general scientific body expects.”

With that statement or commitment from our scientists, we decided to set that as our specific goal.

Back in 2004, we very publicly said that we would license our first target to a company  within 5 years. This means by the summer of 2009.

We’ve been very public about our goal and we have managed our goal and worked very hard to achieve that goal over the last 4.5 years.

We are very close to achieving that goal. We are very pleased. We believe that we are going to announce that we have achieved it within 5 years.

So, that’s a little bit about our model, how we operate and how different we are from other nonprofits.

Another point I want to make is that in academic medical research a lot of money is spent on that. There’s about $40 billion a year spent on academic medical research.

What I think is interesting is that number has doubled in real terms in the last 10 years. People have recognized that cures are very slow.

The NIH budget has increased dramatically in the early 2000s and disease organizations have increased what they have spent on research and so there has been a lot of money thrown at this problem

On the other end of this, you look at the commercial world and the R&D budgets of pharmaceutical and biotech companies have also doubled in the past 10 years. They are now at about $50 billion/year .

So you have $90 billion being spent to come up with new treatments. Unfortunately, because of the way the current system works and the slowness despite that doubling … the number of molecular entities or new drugs that have been approved have actually been flat over the past 10 years.

So despite doubling the amount of dollars the number of treatments has not increased. What that tells us is that the system is really broken. Part of what is broken about it is that the commercial world does not invest in basic science anymore.

Over the last 50 years, the communication lines between academic researchers who do the basic science and the commercial scientist that turn them into treatments have broken down and fallen apart.

Pre- WWII , there was a lot of interaction and contact between scientists but as the funding sources switched to government and nonprofit money. Those scientists are interacting with the NIH and nonprofits and not with companies.

Unfortunately what that has meant is that this handoff that is really necessary to take the scientific discoveries made in academia  and have companies pick those up and move those forward.

That handoff and transition has become a chasm where there is NO communication. It’s actually referred to as the “Valley of Death”

Very few things cross the “Valley of Death”

In summary what we do:

1. Accelerate the front-end through how we have our academic scientists speed up their research.
2. Take discoveries made in our academic labs, and do the rigorous, repetititive testing to develop data that’s sufficient for a company to make a decision about whether to license something.
3. Talk to companies to make them aware of what we are doing. So that they can take things that we have discovered and end-license them and move them forward.

We are trying to address all the elements of the system: the translational gap, the front end, and also working with the biotech/pharmaceutical industry.

What we do that is different from most organizations upfront is that instead of just funding medical research we thought about the ENTIRE continuum.

We thought about how do we get a drug to patients, to MS patients, to repair their myelin so that we could have a dramatic difference on the disease. And that causes us, from the very beginning, to think differently in terms of a continuum.

Everything, all the steps in the value chain from academic medical research all the way to getting FDA approval of the drug and getting it on the market.

Since we thought of it with the end in mind, we thought of elements like developing tools and new assays that could accelerate all neurological research, we’ve also thought about how we could improve imaging techniques and work with imaging companies, so that when we get to clinical trials we could accelerate clinical trials by having better technology to actually image new myelin.

Another thing that sets the Myelin Repair Foundation apart is our more holistic approach to looking at the problem and to try to come up with solutions to for the entire process.

I hope that serves as kind of an update on how we operate differently from other organizations.

The next thing I’ll talk about briefly is the results.

The effort we’ve had so far over last 4.5 years has really exceeded our wildest expectations in terms of its productivity.

The team has been amazingly productive with its discoveries over the last 4.5 years.

We’ve actually developed 24 new tools that are very helpful for research. And when I say tools, these are new assays or animal models (mice) that help in our understanding of myelin, how it’s formed, being able to measure it.

And these tools can help all neurological research; we will make them available to others to use as well to speed up research.

In terms of tool development the team has been very productive.

Secondly, in terms of identifying potential targets that affect myelination, the team has been productive there as well.

We actually have identified 19 different targets that have an effect on myelination in various models. And 12 of those we have prioritized and are moving forward.

We have filed for 9 patents, 1 of which has been issued. We are writing up an additional 8.

Now, and this is where the real proof is, we are talking to several companies right now that are interested in the work we are pursuing, so as I said coming upfront we’re pretty optimistic that in a short amount of time we will be signing an agreement with a company to move some of the science that we have done in our academic labs, to move that forward into the process of drug development and clinical trials.

We are also involved in two clinical trials that we believe are important not only for myelin repair, but also to better understand the clinical trial process as we get to that with some of our other targets.

We are very pleased with the progress that our team has made. It’s been due to the support from a lot of people across the country.

We had originally estimated that in order to license that first target within 5 years that it would cost $25 million in expenditures.

So back in 2004, we set out to raise $25 million. We have raised that money and are nearing the end of that five year period.

We now know that we will need to license many more targets. Because that attrition rate through bio and pharma is very high. In terms of targets that enter that process compared to the number of treatments that emerge.

We know that over the next 5 years, we will probably need to license another 15 or more targets.

Our objective is to continue to do great basic science. To continue to validate targets that do surface, and to continue to license to companies.

We’re estimating that it will cost $100 million over the next 5 years. We’re clearly setting out to raise that money and we’re already raised $10 million towards that.

So that’s kind of an overview of the Myelin Repair Foundation, about how we are different in terms of how we organize our science,  and some of the results we’ve had to date.

I hope that is helpful and clearly gives you an idea of how different we are in structure, organization and results.

We’d be happy to take questions and answers. Thank you.

Full transcript with Q&A on MS World.