From Ice Buckets to Breakthroughs: A Conversation with Calaneet Balas, President and CEO of The ALS Association

For many, awareness of ALS (amyotrophic lateral sclerosis) grew through the Ice Bucket Challenge of 2014, a viral social media movement that raised unprecedented funds and attention for research. ALS remains one of the most devastating neurodegenerative diseases, gradually stripping individuals of their ability to move, speak, and breathe while leaving their cognitive functions intact. A decade later, we sit down with Calaneet Balas, CEO and President of The ALS Association, to reflect on the progress made since that pivotal moment. She shares insights on the evolving landscape of ALS research, the lasting impact of the Ice Bucket Challenge, and the urgent need for innovation and collaboration to drive the next breakthroughs in treatment.

Calaneet, a decade ago the Ice Bucket Challenge raised unprecedented awareness and funds for ALS research. How has that movement shaped the trajectory of ALS research and public engagement since then?

Calaneet Balas: I get that question a lot, and I always say the Ice Bucket Challenge truly transformed the ALS space—and neurodegenerative research more broadly. In just six weeks, it raised around $250 million around the world. A decade later, we’ve seen that every dollar invested in research has leveraged an additional $7 to $11 in funding across the globe.

This influx of resources has driven major outcomes. We now have several new drugs on the market, including one that’s emerging as a game changer. Additionally, the funds helped expand multidisciplinary clinics from about 30-40 centers across the U.S. to over 200. These clinics put testing, nutritionist and physical therapist together in the same place, providing one-stop comprehensive care. It has been shown to extend patients' lives by up to a year—a significant improvement for those struggling with the logistical challenges of accessing care.

Ultimately, the Ice Bucket Challenge not only sparked an immediate funding surge but also set off a cascade of advancements in research, treatment, and patient care. It’s incredible to think that something as simple as dumping a bucket of ice on your head could ultimately help save people’s lives in such a profound way.

You have ongoing investments and research initiatives in ALS treatment. Could you share some progress?

Calaneet Balas: Certainly. One example is a treatment called Qalsody, which was approved about two years ago. We first invested in it 20 years ago when it was considered a “crazy idea”. After investing roughly $2 million in early research, it progressed through subsequent trial phases and now approved to treat patients associated with a specific genetic mutation, SOD1.

What's truly remarkable is the real-world data published in the United States and two other countries. For some patients, Qalsody not only halts disease progression—it appears to restore function, a result that was previously unthinkable in neurodegenerative care. Our goal was initially to stop or delay progression in individuals with the genetic mutation, so witnessing functional recovery is astonishing. This breakthrough has paved the way for additional clinical trials using similar technology for other genetic mutations and rare diseases. We’re closely monitoring its impact as it continues to evolve.

Compared to other rare diseases, does ALS present unique challenges, or do you find that the overall approach is similar across the rare disease community?

Calaneet Balas: No, they're not the same. Most of my colleagues and counterparts in other diseases recognize that we have a lot of similarities, but ALS is distinct. If you think about the concept that over a certain amount of time, you lose your ability to move your muscles, speak, breathe, communicate, eat, and yet your mind is still solid. It's an unbelievably cruel disease, and one that a lot of people don't understand, and one that a lot of people are scared of. And the community itself is a very unique community because they are scared.

It reminds me of the height of HIV, and the anger, the "help me," the desperation. All the needs as individuals progress through this terrible disease are so high and so demanding. And watching someone that you love to turn into someone who can't even communicate to you at all is really unfortunately very unique. I've never seen anything like it. I say that once you see ALS, you can't unsee it. You can't look away from it because it's so hard.

Building on these advances, what does a “miracle drug” may look like to ALS patients in your view?

Calaneet Balas: Many people living with ALS often say, “If I could just stop it now.” For instance, someone might say, “I've lost my ability to walk, but I can live with that if the disease stops progressing.” Does everyone want a cure? Absolutely. But a therapy that halts progression at any stage would be a silver bullet for our community.

How can we accelerate research in ALS and bring these therapies to patients faster? What’s the role of biomarkers in helping diagnose and enroll patients earlier?

Calaneet Balas: You're speaking my language. Getting patients diagnosed earlier is a huge initiative for us. When somebody with cancer gets diagnosed earlier, their outcomes are usually better. We're trying to do the same thing in neurology. Currently, however, we lack a reliable biomarker for ALS, which means diagnosis is often a process of elimination that can take up to two years. During this time, patients continue to progress, which is a significant challenge.

We're investing heavily in biomarker development and collaborating with other organizations in neurodegenerative diseases to share insights and resources. This collaborative approach not only accelerates the diagnostic process but also enhances our overall R&D productivity for ALS therapies.

What does collaboration mean to ALS Association? How is your organization partnering with all sectors in the ecosystem to drive innovation in ALS?

Calaneet Balas: We work with our partners in a variety of ways. For instance, we fund early-stage clinical trials and preclinical work at smaller biotech companies—efforts that have contributed to treatments approved in recent years. We also collaborate with pharmaceutical and assistive technology companies to bring together diverse expertise.

One of our key initiatives is hosting two annual roundtables focused on specific topics, such as genetic testing and counseling. In these sessions, we gather around 50 experts from different sectors to discuss strategies for early diagnosis and investment in genetic research. This collaborative approach ensures that our direction is shaped by the entire ecosystem rather than by our organization alone.

Additionally, we've recently launched a small venture capital arm aimed at de-risking emerging technologies and therapies. While we’re not positioned to invest in large-scale Phase 3 trials, our goal is to help move promising innovations to the next funding round, thereby attracting larger investments from industry. This model has been successful in other areas, like cystic fibrosis, and we're confident it will accelerate progress in ALS research as well.

Building on that collaborative model, what challenges do you still see in advancing ALS research and treatment?

Calaneet Balas: It's a great question, and it's a very complex one. I have the privilege of being the chair of the International Alliance for ALS and MND Organizations globally. One major concern is the heterogeneity in access to clinical trials—many regions, including parts of the U.S., lack diverse patient populations, limiting the data and insights we can gather. To address this, we've built several international databases. For example, Project MinE emerged from the Ice Bucket Challenge funding, enabling us to collect genetic data worldwide and mine it for insights.

We are working with our partners in Canada and Europe to encourage regulatory agencies to exercise greater flexibility when evaluating therapies. Access is another critical component. Additionally, there is a pressing need to educate stakeholders; I've seen cases where insurers recommend trying one therapy first, delaying crucial treatment. In ALS, delays can be fatal.

We also learn a great deal by exchanging information about different health systems. By sharing experiences with agencies like Health Canada and the EMA (European Medicines Agency), we can advocate more effectively and accelerate drug approval and access in our own regions. These coordinated efforts are essential to making meaningful progress in treatment availability.

Calaneet, your description of ALS is a stark reminder of the urgency for change. With Rare Disease Day marking the end of February, if you had the full attention of every pharma and biotech CEO, investor, and policymaker, what would be your call to action?

Calaneet Balas: Don't walk away from risky spaces—step forward instead. These areas need the most investment, and once you hit it out of the park, you'll be the only one standing there. We have a lot of drug development happening in areas that maybe don't need it as much. I do think that there's an opportunity to look at rare diseases in a way that they're not as rare as you think. And once we can get these therapies into market, we need your partnership in that.

I say this phrase a lot because we talk about patients. We talk about rare disease. We talk about these communities. And the way we talk about it is always like they're other people. But they're not other people. They're us. And so don't shy away from it. That would be my call to action.

Thank you so very much.

Calaneet Balas: Thank you. It was nice to speak with you.