Scott Kauffman on Doing Whatever It Takes to Create a World Without ALS

Scott and Stephen

Earlier this year Scott Kauffman, a business executive and long-time leader in the fight against ALS, began his tenure as chairman of The ALS Association Board of Trustees. We recently caught up with Scott on Connecting ALS, where he talked about his connection to the disease, raising awareness and his vision for creating a world without ALS.

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The transcript below has been lightly edited.

Can you introduce yourself and let us know a little bit about your connection to the ALS community?
I'm Scott Kauffman coming to you from sunny Palo Alto, California. And I joined the board of The ALS Association six years ago. I've chaired the Audit Committee and the Communications Committee, and I've just completed my second month as the Chair of the national Board of Trustees of The ALS Association. But as I like to say, my most important role is as a caregiver to my son, Stephen, who was diagnosed 10 years ago at the age of 27. In fact, he will celebrate, if that's a term we can even apply here, his 10 year anniversary of the diagnosis, this June 6.

Now, when I'm describing what has happened to our family, I like to leave people on a high note. So I mention that my son lives five doors down from me with my daughter-in-law, who fell from the heavens eight years ago, and my grandson. They made me a grandfather four years ago.

And then sometimes I see the quizzical look on people's faces, those that understand what it means to have ALS, and I explain how there are two major systems in the human body, the motor neuron system, which ALS impacts dramatically and the cardiovascular system, which it does not. So even though Stephen is late stage, meaning a machine breathes for him and a tube feeds him and he, if on an ALSFRS-R score probably is about a one. He has a little motion in his neck. We put a switch right behind his ear, he is able to command all of his vast multimedia arrays -- watch all the sports there is. The Golden State Warriors are giving us a lot to cheer for. We were season ticket holders for 20 years. And of course being a typical ALS patient with a sound mind, he is a living testament to our mantra these days of making ALS a livable disease.

What have you seen over your years of service in your time as a caregiver that suggests to you that there's momentum, that we're moving in the right direction, that we're seeing progress or that progress is on the horizon?
I bring a healthy skepticism to the table. You know, it's been eight plus decades since Lou Gehrig called himself the luckiest man on the face of the earth. Ten years ago when Stephen was diagnosed, I mean, the first reaction a family has is, you kind of curl up in a corner and cry. And then I realized that wasn't going to move the needle. So I started looking around, and I was somewhat dismayed at how disjointed and fragmented the community, as you've described it, was. So much so that I was frustrated and decided to create my own foundation. And with the appropriate level of naivete and arrogance, I went about founding a 501(c)(3).

I got permission from Lou Gehrig's estate to call it the Iron Horse Foundation. And Stephen was born in New York City. He grew up a Yankee fan. Lou Gehrig's nicknamed the Iron Horse -- until Cal Ripken came along, he held the record for the most number of consecutive games played until he realized that there was something going wrong with his body. And at the time I was running a global advertising agency holding company. So we had tremendous resources, and they helped me set up the organization, the beautiful website and starting to talk to some of our clients about cause-related marketing. And then the Ice Bucket Challenge hit, and all of the sudden there was a lot of money coming into the community, the majority of it going to The ALS Association. And so I decided that I could be of more value there.

And over the six years since I joined The ALS Association -- and because of some of the pro bono work that our agencies had done on behalf of a wide swath of the major players, including ALS TDI, Answer ALS, Team Gleason, and the like -- I have felt a closer galvanizing of the community, a desire to share resources, particularly data.

And there are those among us that believe that the cure is here, it's on this planet with us, but probably an exercise in big data, machine learning, artificial intelligence. And the only way you're going to get there faster is if you pool all of the resources and all of the data and that you kind of divide and conquer. And so I've seen a greater willingness now for the organizations to collaborate, whether it's on different research initiatives or on different care programs. And I have to believe that if we sort of lower the barriers of what might be perceived as competition among the groups, that the entire community will be better served.

And then on the other side on the pharmacological side, which I'm less well versed in, and so I listen carefully to what our research team is saying and what the FDA is doing, and what pharma is organizing to help do, is that there seems to be a lot of energy and momentum behind therapeutics. And there are a couple in particular that have demonstrated an ability to extend life by a certain percentage of time, which then broadens the community. We think assistive technology and better knowledge and care are also extending lifespans. As I said, my son is now at 10 years. And with that will come a growing population. And so therefore the need for more services in more places, which in all likelihood will create an even greater demand for these therapeutics. If we can get someone's life extended by six months, that could literally be the timeframe in which the "cure" happens. And so we're hopeful that we'll continue to move the progress along of the length and the extension of the disease.

When you get diagnosed today, and I don't know where these numbers come from, the standard line is the average life expectancy is three to five years. Which I've never understood how you could have a 24 month average expectancy span. But that being said, I'm well aware of many people that have gone beyond five years now. And so hopefully those numbers will get revised and hopefully at some point this will become a chronic disease, not a fatal disease. And hopefully at some point after that this will become a preventable disease. So that's how we're thinking about the steps of making it livable while searching for a cure.

You have been active in the We Can't Wait Meeting and the testimony before FDA several weeks ago before the advisory committee. As FDA goes into its next phase of deliberation on the new drug application, what do you hope is their takeaway from the conversations that the community has had with them? What do you hope is guiding their deliberations today and over the next several weeks?
I'm hoping that some combination of common sense and a pledge that the FDA has made to this community about exercising, what they deemed regulatory flexibility when it comes to our community, given how clearly our community has risen up and said, "If it is safe, we will incur the risk of whether or not it is effective." And part of why it makes perfect sense is that, I hate to use terminology like this, but our community realizes they have little to lose. Obviously nobody wants to advocate for drugs that could be potentially dangerous, but once a drug has been deemed safe and in the case of AMX0035, you're talking about two previously approved drugs that have a proud track record of safety. The cocktail combination does not appear to in any way impede that safety.

Now the question is, is it effective? We all know how heterogeneous this disease is. So in any form of clinical trial, unless you have great knowledge of what version of ALS the patient population has and there's no way to know that right now, because we have so little understanding of the 90% of the community that incurs sporadic onset. You're basically climbing up a hill. AMX0035 may be literally the cure, who knows. I mean, I'm the least knowledgeable person to speak on this, but it may be the cure for version 6 of ALS. We don't know what we'll call it then. But in the same way that you wouldn't treat pancreatic cancer the way you would treat skin cancer the way you would treat breast cancer the way you would treat testicular cancer, we believe that there are many different forms of ALS. There's upper motor neuron onset, lower motor neuron onset, bulbar onset, people that can walk but don't have motion in their arms, people that can speak but can't walk and vice versa.

And so using the norms of the standard statistical process to determine whether a drug is effective, is almost a false logic when it comes to our community. So once it's safe and once our community has expressed a willingness to consume a drug that may not be effective -- and by the way, even if it's only effective because of a placebo effect in my mind, that's effective. I'm hoping that the 6-4 vote will be meaningless coming out of the advisory committee -- and we could all spend a whole show talking about the composition of that advisory committee and how those decisions were made and how the question was posed, and then how the language was slightly altered when the question was finally posed, and how nobody challenged the change and how the question was posed -- we could go through all of that, but that's the minutia. That's losing the forest for the trees.

And I am hopeful, back to my opening sentence, that the powers that be, having already expressed a desire to bring urgency to this process, having already coined a phrase, regulatory flexibility, will make the -- I won't call it an exception -- will make the appropriate decision given what our community faces today.

Talk to me a little bit about your vision, where you see the fight going from here, and some of the important steps in the next few months and years.
We think about our mission as really three pillars of advocacy, research and care services. And with the extended life expectancy that we're anticipating, we want to be in more places, serving more people. So that's a lot about just looking at the map and understanding where families are underserved today. I live in Palo Alto, California. As I'm fond of saying, it is one of the most resource rich environments in the world. My son was diagnosed at the Forbes Norris Clinic. It's about a 25 minute drive from our home. A few years later, Stanford opens up a world class multidisciplinary clinic. That's two blocks from our home. But I can't imagine what it's like to be battling this disease without those kinds of resources. So we're going to take a very hard look at where we are underrepresented and underserved in the expectation that our community is going to grow over a period of time and then hopefully shrinks again because we've cured it and this insidious disease goes away.

I'm also very big, as I said at the outset, on collaboration. So it's working with the other organizations. Yes, we compete for funding dollars, but there are a whole bunch of things we can be doing better together on a whole number of fronts, including, most importantly right now, advocacy. So the Act for ALS and the $500 million deal that Joe Biden signed that has a $100 million for five years and how do we spend those dollars between new drug discovery versus early access programs for our current community. And doing a better job of getting the research organizations to share information. I mean, I truly don't care where the cure comes from, but I don't want it locked up in a silo somewhere waiting for a university or a pharmaceutical company or an independent researcher to take credit for it. I just want to get it out into the field, tested, proved, and then into people's bodies so that we can rid this world of ALS.

During ALS Awareness Month, a lot of people are paying attention to our fight maybe for the first time, or maybe for the first time since they took the Ice Bucket Challenge all those years ago. So for folks who maybe aren't living and breathing this every day, what do you want them to know about ALS, about the fight and about ways that they can contribute to the progress that you've been talking about?
I want people to know that this is one of the most devastating diagnoses a human being can can receive. I want people to know that the support of the community is paramount. I want people to know that the family battling this disease -- and that's just not the person living with it, but the caregiver, the children or the parents, whatever it may be -- all live this disease. And you live it 24/7. And in the case of my son, who's been alive for 10 years, that's a choice you make at some point when your diaphragm gives out. And that's a choice that I wouldn't wish on anybody, but very fortunate that our son decided to get a tracheostomy and has that machine now breathing for him.

But the community support is really important. And we're coming out of the pandemic now and a lot of the fundraising that we do is event based at the community level. And so, even if you don't have a direct relation to the disease, you will meet some of the finest people you would ever hope to encounter if you chip in and pledge and show up and participate with us. And at the end of the day, like any community that bands together to support its populous, these families really depend on that passion and compassion in grappling with this. It also, I think, it gave me a whole new awareness of humanity and what it means, what is fundamental to a human being. When I think about the things that my son now has to rely on other people to do for him, and how graciously he has accepted that reality, and then how normalized that all becomes, because those things aren't really important.

Things that would be embarrassing in normal walks of life, but they aren't the core essence of you as a human being or what humanity means. And I think we have seen the best instances of people reaching out to help people when it comes to ALS. If there is a silver lining here, it is that everybody around Stephen now fully appreciates what it means to be alive. It's not just a case of stop and smell the roses every once in a while. It's every time you scratch your face because you have an itch, be thankful you can scratch your face because you have an itch.

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