Turning a Diagnosis into a Movement to Find a Cure

Imagine one day, you feel a twitch in your hand. Weeks or months later, your hand gets weaker, and you can’t turn the key in your front door. The diagnosis: Amyotrophic lateral sclerosis (ALS), a rare neurological disease that impacts muscle control. ALS robs people of the ability to speak, eat, move — and even breathe, typically in under five years. The causes of ALS remain largely unknown, and there are no effective treatment options.

I AM ALS empowers patients to lead the search for a cure. CZI supports I AM ALS to develop tools for building a movement across the ALS community that connects patients, caregivers, doctors, scientists, foundations, corporations, and the public in the fight against ALS. This support is part of CZI’s broader work to help patients and their families accelerate research on rare diseases, which affect approximately 400 million people globally. CZI’s Rare As One Project is committed to supporting and uniting these communities in their quest for cures.

Tania Simoncelli, CZI Science Policy Director and Rare As One Project lead, sat down with ALS patient and I AM ALS co-founder Brian Wallach to talk about his quest to find a cure.

I AM ALS co-founders Brian Wallach and Sandra Abrevaya. Photo by I AM ALS.

Why did you start I AM ALS and how are you hoping to change advocacy for this rare disease?

We started I AM ALS after my diagnosis in November 2017 to answer the central question: “How do we find a way to empower people who are living with ALS to be a part of finding the solution for their disease?” And not just ALS, but really every disease out there — because too often, when you get diagnosed, you become reactive and depend on others to help you figure out what to do and how to do it.

Another big part of our effort is bringing together different ALS groups around a shared set of goals. There are over 25 ALS groups, and we’re lucky in that respect. Unlike many other rare diseases where there isn’t even a single group, we have a lot of people already fighting. Yet we end up having a lot of people doing the same things, and only finding out about duplicate efforts three years down the road, which in the fight against ALS is not okay — many of us only live two to five years.

With I AM ALS, we spent nearly a year prior to launching learning about the ALS space, meeting with ALS groups, patients, doctors, and beyond to see how and if a new group could help augment the fight. What we found was that there was a true need for a patient-centric, patient-focused group built around the idea that by truly engaging and empowering those living with and fighting ALS, we could build a movement to capitalize on and expand these amazing resources and finally win the fight against ALS. Our goal is to build tools that will benefit everyone, connect everyone, and unify the amazing actors in this fight to drive towards cures as fast as is humanly possible.

How do you see I AM ALS helping other ALS or rare disease groups make progress on their disease?

One of the things that I am most excited about with I AM ALS is our chance to really build an open source model that other disease groups can use.

We have a favorite saying within our foundation, which is, “What’s done in secret is done in vain.”

We hope that we can partner with CZI and other groups to not just build a model that can reimagine how we fight ALS, but reimagine how people advocate and fight every rare disease.

Our hope is that the technology and advocacy platforms we build can empower others to do their work far faster and not have to reinvent the wheel, and thereby allow them to do other things that are needed to accelerate their fights.

Why do you think it’s important for patients to actively engage with researchers and help find treatments and cures for their disease?

Having patients in the room with researchers and clinicians to be a part of the discussion changes those discussions. It makes it real and grounds it in experience. When you look at a scientific idea for a treatment it can be a beautiful concept, but the question is, “How do we actually take that idea and make that real for people? Not 25 years from now, but now?”

Patients ask questions that others may never ask, and their presence empowers researchers and clinicians to remember that their work has a real impact on millions of us across the globe — about 400 million people are living with a rare disease. Think about that for a second. And every one of those people has family members and friends.

An enormous number of people are impacted by these diseases, and if patients and their loved ones are part of this effort to find solutions, we will see outcomes way faster than we ever dreamed.

How are you using your expertise in understanding the power of community organizing to accelerate progress against ALS?

I was lucky enough to meet my amazing wife Sandra on the 2008 Obama campaign. I spent 18 months with the campaign, and from that campaign Sandra went on to found two different nonprofits after we worked together in the White House. Those experiences taught us the power of community, and they also taught us the importance of being able to listen to everyone and not be reactive to any single idea.

It taught us to hear people in a real way, to slow down and listen to their hopes and dreams, and then to build something that reflects those desires in a way that people can see and be a part of. We’re helping not just ALS patients, but anyone who is interested in this fight to find ways to make a difference — if you send an email, if you join us in any way, every little action matters and brings us closer to the outcome that we all want.

Left: I AM ALS co-founders Brian Wallach and Sandra Abrevaya. Right: ALS patient and I AM ALS co-founder Brian Wallach testifies in Congress. Photos by I AM ALS.

What advice would for a patient who’s recently been diagnosed on how to start driving progress forward?

The first advice is grieve. Be with your family, and then figure out how you want to be involved in moving things forward. Not everyone has to be on Twitter every day talking about ALS, but I hope that you will find a way to be empowered, and I hope that you will realize that you are never walking this fight alone.

There are thousands of others out there, whether they have ALS, whether they have a rare genetic disorder, whether they have Alzheimer’s, that are with you every step of the way and want to see you win this fight. When you know that, it gives you the capacity to wake up every morning and say “I may not be in a position to carry this on my own, but I’m going to be a part of that larger effort that will make it real.”

You were at the kickoff meeting for the CZI Neurodegeneration Challenge Network, which brings together experimental scientists, computational biologists, and physicians to understand the fundamental biology of neurodegenerative disorders like Alzheimer’s and ALS. How do you hope the Challenge Network will change the approach to finding treatments and cures for neurodegenerative diseases?

I left that meeting smiling from ear to ear because in many respects, that meeting realized the hope that we will no longer research in a silo — we will research together. We don’t have to have a discovery in ALS that takes 10 years to get to get to a patient; instead, it will be shared right away. That kind of synergy will allow us to take the pace of discovery and really hypercharge it.

That kind of connectivity doesn’t happen until a philanthropy like CZI says “Hey, by the way, why aren’t we doing this? Why isn’t there a better way?” Unless you question the system, the system just goes on. Seeing the Challenge Network begin to break down barriers in the neurodegeneration field is amazing, and I’m excited to be a part of that.

How do you see technology playing a role in solving neurodegenerative diseases?

There is an amazing chance now to be able to do things that we never thought possible before, and if we don’t tap into those resources, if we don’t look at how we’re using them, we miss a huge chance to be a 21st-century problem solver. We have to adapt and use new tools at our disposal to really accelerate research findings.

We have the tools at our disposal now — with connectivity like social media and the internet — that we didn’t have 20 years ago. There’s no reason to have barriers between research silos anymore, and breaking them down means getting patients involved across the entire healthcare system.

What would you want researchers, clinicians, and others working on neurodegenerative diseases to understand from the patient perspective?

We are here to work with you. We want to be a part of finding cures. We may not speak the same language, we may not know every single term or every single chemical compound, but that doesn’t mean that we are not a part of solving this problem with you. We want to accelerate your discoveries because they will help save our lives. That means that we want to help in every way we can: raising more funds, raising more awareness, being a part of your clinical trials. Help us help you, and we think together we can make amazing things happen.

More and more patients and their families are accelerating research and driving treatments for their rare diseases. Share your #RareAsOne story.

Chan Zuckerberg Science Initiative

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Supporting the science and technology that will make it possible to cure, prevent, or manage all diseases by the end of the century.

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