Advancing ALS Care and Research: Introducing ALS Association’s Inaugural Conference

John Novak, MD

Amyotrophic lateral sclerosis (ALS), is a progressive neurodegenerative disease that affects nerve cells in the brain and spinal cord. Most people who develop ALS are between the ages of 40 and 70, with an average age of 55 at the time of diagnosis. Treating the disease has been a challenge thus far, as several medications have shown efficacy in animal models of ALS but fail to increase longevity or improve the quality of life for the disease. Riluzole, an anti-glutamatergic substance, and edaravone (Radicava; MT Pharma), an antioxidant and free radical scavenger, have been typically prescribed within the clinical settings.

For the first time ever this summer, the ALS Association will host its inaugural ALS Nexus Conference, an opportunity to bring together leaders in the ALS community to connect, collaborate, and change the future of the disease. The event which takes place July 14-17 in Dallas, Texas, features a variety of sessions that focus on the latest developments and opportunities in research, care, and advocacy. Throughout the event, clinicians can learn about ALS drug development, palliative care for ALS, ways to improve clinical trial efficiency, innovative technology and artificial intelligence, assistive technology, and access to genetic therapies, among other topics.

Prior to the meeting, NeurologyLive^® sat down with John Novak, MD, MS, director of the OhioHealth ALS Clinic, to provide some insight on the upcoming meeting and what clinicians can expect. Novak, who also serves as a vice chair of the ALS Association Care Services Committee, gave commentary on how the themes of the meeting align with where research is currently, the different types of clinicians who should attend, and updates in biomarker and genetics for ALS. For more information and registration to the event, click here.

NeurologyLive: Provide an overview of the ALS Nexus, what does it bring to the clinical community?

John Novak, MD, MS: Well, I think the Nexus being in its first year is kind of exciting because it's an open book; we don't know what to expect. Looking at the agenda, it’s a real mix of the science of ALS. We hit on drug development, targeted gene therapies, and similar talks. But we're also focusing on how we reach underserved communities. How do we make care affordable and equitable? How do we fund the research? I’m excited because we’re not just focusing on one aspect of ALS care. We’ll look at the science and discuss how to deliver this care effectively. It’s unique compared to many of our more scientifically granular conferences. We’ll have people who do the science and those who deliver the care, which is great.

Who should attend the meeting?

We hope to attract a wide array of clinicians. Therapists are extremely helpful in caring for ALS, and their attendance would be great because they look at things differently. They focus on how patients get through their day, which is always helpful. Having palliative care involved is important too. There’s a specific discussion on involving palliative care, which focuses on the quality of life. Neurologists need to work closely with them. We’re excited about having them involved and hope to get other clinicians besides neurologists to attend, talk, have lunch, and rub elbows. That’s how we advance ALS care.

How do the themes being discussed at this meeting represent where we are in ALS research/care?

Talks on drug development are always exciting. Everyone wants to know what’s cutting edge in science, where we are going, and how we target therapies. Our current therapies are broad-spectrum, aiming to slow ALS, but now we’re focusing more on specific genes. The science is exciting, and we’ve learned a lot in the last two years. But we also need to look at how we deliver care and maximize the benefit of science. How do we move past the science and get it into clinicians' hands? This aspect is unique to this meeting and will be exciting.

Outside of drug development, how are we working towards improving the symptomatic aspects of ALS?

One big topic is the cost of ALS care. It's a huge problem. Medications can be expensive, but symptomatic care, like non-invasive ventilators and durable medical equipment, are also costly. We need to work on solving this problem across the spectrum of care, from mild to severe end-stage care. Discussing this openly and working on these problems is important. Patients should be in multidisciplinary clinics where this can be approached. It would be great to have clinicians who don’t do this daily attend and learn about the benefits of multidisciplinary care.

Where do we currently stand on the prevention of ALS?

Our first major move was identifying who’s at risk. This is limited to our genetic population, which is estimated at 15-20% of cases. Most cases are sporadic, and we don’t have good ways of predicting those. We've made positive strides in identifying carriers and figuring out what we can do before they become symptomatic. We have one therapy for a specific mutation in the SOD1 gene. The focus now is on treating carriers of that gene before they become symptomatic. We’ll see this approach with other mutations soon. Unfortunately, we don’t have a great way of doing this in sporadic cases yet, but we’ll learn a lot in the next few years.

Where do we stand with finding effective biomarkers?

I’ve started using neurofilament much more, and I think other physicians have too. It's partly about getting people to use it daily. It’s one thing to use it in trials, but another for clinicians nationwide to become comfortable with it and understand its clinical significance. If thousands of clinicians use it, it gains traction. Neurofilament is the most used biomarker now, but we’ll probably need more. A detailed look at physiology will advance our capabilities. Monitoring multiple biomarkers in the same person while giving different medications will be huge. We're getting there, probably a few years away, but this approach is being talked about a lot.

Transcript edited for clarity.