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Webinar Recap: Research in ALS: What’s Giving Us Hope?

Learn how the latest ALS research has led to groundbreaking treatments that slow this brain disease and may soon be able to prevent it.

The latest ALS clinical trials have produced major advancements in its treatment and have even impacted therapeutics for other neurodegenerative diseases, like Alzheimer’s and Parkinson’s. Board Chair David Dodick, MD, FAAN, hosted a recent webinar with Foundation Board Member Merit Cudkowicz, MD, MSc, one of the world’s foremost experts on ALS. 

Dr. Cudkowicz discusses promising new research, breakthroughs about the underlying biology of ALS, and recent advances in treatment that provide hope for people with ALS and their loved ones. She also explains how these advancements have a ripple effect on the larger field of brain disease research.

What is ALS, and Who is Affected by It?

ALS stands for amyotrophic lateral sclerosis. A rapidly progressing neurodegenerative disease, ALS causes damage to the nerve cells in the brain and spinal cord that control voluntary muscle movement. When these nerve cells (called motor neurons) decline, they stop sending signals to the muscles. This makes the muscles twitch, weaken, and atrophy (lose mass and deteriorate). ALS is a rapidly progressing disease with an average life expectancy of two to five years.

While ALS most commonly affects people between the ages of 40 and 70, Dr. Cudkowicz notes that younger and older people can develop the disease. According to the National Institutes of Health, men are slightly more likely to develop the disease than women—though this difference is reduced as age increases. ALS is considered a rare disease, affecting about five in 100,000 people. “There are about 30,000 people in the United States living with ALS and more than 400,000 globally,” explains Dr. Cudkowicz.

What Causes ALS?

Understanding what causes ALS is the subject of much ongoing research about the disease. “We know that between 10 and 20% of people have a known change in their DNA, what we call genetic mutation, that causes the illness,” says Dr. Cudkowicz. Scientists have identified more than a dozen genetic mutations that can be inherited from a parent and cause the hereditary form of ALS. 

However, the majority of people with the disease have sporadic ALS, meaning it happens without a clear cause or family history of the disease. Researchers believe that this form of ALS is caused by genetic risk factors combined with environmental factors. More research is needed to better understand the development and progression of sporadic ALS.

Exciting Advancements in Treating ALS

A few decades ago, there was only one drug treatment option for ALS. Today, there are three marketed drugs that work for all forms of ALS to slow down disease progression: Riluzole, Radicava, and Relyvrio. The most recently approved drug, Relyvrio, is particularly promising. A combination of two different existing drugs, it works synergistically to target multiple issues caused by ALS. “The combination of these two drugs slowed the loss of function by 25% and also had an effect on longevity,” she says.

Another promising treatment is the gene therapy tofersen (marketed as Qalsody®), which was developed for people with a specific type of hereditary ALS. “This is a treatment that can block the mutation in the gene from making the misfolded or mismade protein called SOD1,” explains Dr. Cudkowicz. The global study showed that the drug slowed disease progression for everyone in the trial. Amazingly, 40% of participants got better and showed improvement in their strength. Researchers are working to understand why the other 60% did not have that improvement, but the findings are still exciting. “This is a huge step forward for ALS,” says Dr. Cudkowicz.

The same study revealed promising news about using biomarkers (biological indicators) with ALS. Researchers used a blood test to measure damage to the nerves. At 12 weeks of treatment, the test showed a 50% decrease in the appearance of an indicator of neurodegeneration called neurofilament. Equally as important, the biomarker also predicted clinical response. “This is a game changer for ALS because that means that there can be possibly a shorter way to screen drugs using this blood test, rather than perhaps needing longer studies looking at clinical outcomes,” explains Dr. Cudkowicz.

Innovative Trials for New ALS Treatments

While ALS studies were difficult to come by in the past, many are now taking place worldwide. Dr. Cudkowicz notes that, at the time of the webinar, there are currently more than 27 late-stage trials. If those trials are positive, the studied treatments could soon be available to the public.

Innovations in how clinical trials are conducted are also expediting the advancement of new treatments. Historically, drugs are tested one at a time, but this is not the most efficient method when there is a long list of them to study. ALS researchers decided to use platform trials, a successful concept used in cancer clinical trials that need to evaluate many different drugs. A platform trial tests multiple drugs in the same infrastructure, adding and removing drugs until researchers find what works. 

Another benefit of platform trials is that the data from a single placebo group can be used for multiple drugs. “This is a way to really cut down the time of drug development in half, cut the costs, and really increase the number of people getting the active drug,” says Dr. Cudkowicz.

How ALS Research Impacts the Larger Field of Brain Disease

ALS research benefits not only those with the disease but also people with other neurodegenerative conditions. Insights from ALS trials are already being applied to several other brain diseases. “The Michael J. Fox Foundation and some of the Parkinson’s researchers came to visit us to learn about platform trials, and they’re now launching a platform trial in Parkinson’s,” explains Dr. Cudkowicz. 

Researchers studying stroke and Alzheimer’s disease are also following suit. “We’re also sharing a lot of the science because some of the biology is overlapping between these diseases,” says Dr. Cudkowicz. This underscores the American Brain Foundation’s philosophy of Cure One, Cure Many: Many brain diseases are interconnected, so a breakthrough for one will lead to advancements for countless more.

Why More Brain Disease Research is Critical

The field of neurology is making progress every day, but there is still much more work to be done. “Ultimately, we want to be able to say we have treatments that stop [people’s] illness. And that’s where the research is so important in attracting young new investigators to the field,” explains Dr. Cudkowicz. She is optimistic about the future of treating ALS and other neurodegenerative diseases. “This is the first time in my career where I’ve seen a drug not only work but actually halt the illness,” she says. “That tells us it’s possible, it’s possible to cure this illness, the more you understand the cause.”

The American Brain Foundation is committed to funding research for ALS and other neurodegenerative diseases. That’s because finding a cure for one brain disease will lead to cures for many others. Join us in our fight against brain disease—donate today to fund life-changing research.