How a new partnership aims to tackle ALS

ALS concept

DDW Editor Reece Armstrong speaks to Alessio Travaglia, PhD, Director, Translational Science, Neuroscience, Foundation for the National Institutes of Health (FNIH) about challenges in treating amyotrophic lateral sclerosis (ALS) and why a new public-private partnership launched by the FNIH is hoping to advance new drugs and diagnostics for the disease.  

RA: What are some of the traditional challenges in treating ALS?  

AT: ALS is a brutal disease, and treating it is an ongoing battle. We have medications to manage symptoms and improve quality of life, but we can’t stop the disease itself. The disease affects everyone differently, making it hard to develop a universal treatment. To further complicate matters, there is a lack of reliable markers to predict the course of a patient’s disease or measure treatment success. 

RA: Why are treatments so limited for this condition?  

AT: ALS presents a unique challenge when it comes to treatment development. We are still piecing together the puzzle of what causes ALS. While genetics play a role in some cases, the exact mechanisms behind most cases remain a mystery. This lack of understanding makes it difficult to develop targeted treatments that address the root cause of the disease. 

The progressive nature of ALS makes it a moving target. As the disease progresses, the underlying pathology changes. This means that a treatment that might be effective in early stages may not work as well in later stages.  

RA: 2024 is the 10-year anniversary since the viral Ice Bucket Challenge. How much awareness did the challenge bring to ALS and have treatments advanced much within that time?   

AT: The Ice Bucket Challenge definitely increased ALS awareness. It was a global phenomenon that put this disease on the map for many people. Also, the financial boost from the donations has been critical. More funding means more research, which is leading to a better understanding of ALS and the development of new treatments.  

RA: Could you tell us about the new Accelerating Medicines Partnership in ALS? 

AT: The Accelerating Medicines Partnership in Amyotrophic Lateral Sclerosis (AMP ALS) is a truly innovative initiative launched in May 2024. It’s a public-private partnership that brings together the resources and expertise of the National Institute of Neurological Disorders and Stroke at the National Institutes of Health, the Food and Drug Administration, Critical Path Institute, and other stakeholders from academia, life science companies, foundations, and patient-focused groups to tackle the tremendous challenges of diagnosing, understanding, and treating the disease. 

This partnership tackles ALS from multiple angles with the ultimate goal of speeding up both diagnosis and treatment development. One key focus is to build a centralised database that will allow scientists to analyse vast amounts of information from various sources, ultimately accelerating discoveries and breakthroughs in ALS research.  

Another critical area is earlier diagnosis. AMP ALS is heavily invested in developing reliable biomarkers. Earlier diagnosis is crucial because it allows patients to access treatment sooner, potentially improving their quality of life. 

Finally, AMP ALS is tackling the challenge of standardising how we measure how a patient feels, functions, or survives. Currently, it can be difficult to compare results across different studies. By using consistent assessment tools, researchers can get a clearer understanding of whether new treatments are working. This will ultimately lead to faster development of effective therapies for patients with ALS. 

RA: How important is the interplay between public & private institutions in building data to better understand, diagnose and treat ALS? Following that, how useful will centralised datasets be in enabling research into ALS? 

AT: Collaboration between public and private institutions is crucial to breaking down data silos and building comprehensive data sets for ALS research. By sharing data and expertise, public and private institutions can accelerate progress: it’s about leveraging each other’s strengths. Centralised datasets are game-changers. They allow researchers to analyse vast amounts of information from various sources, all in one place. This can lead to faster breakthroughs in understanding ALS and identifying new treatment targets. 

For example, with a centralised dataset, researchers can easily compare genetic data from different patients to identify potential genetic links to the disease. Centralised data gives researchers a much bigger picture and serves as a powerful tool with the potential to revolutionise ALS research. 

RA: Could you discuss the importance of identifying biomarkers for ALS for both diagnoses and the discovery of new therapeutics / impact on clinical trial design?  

AT: Right now, diagnosing ALS can be a lengthy process. Some people wait years before receiving an accurate diagnosis after experiencing their first symptoms. Validated biomarkers could allow for earlier detection, potentially through a simple blood test or scan. This is crucial because it allows patients to get treatment sooner and potentially improve their quality of life. 

Biomarkers can significantly improve clinical trials, as well. Currently, measuring treatment effectiveness in ALS is challenging due to the variability in disease progression. Some patients experience rapid decline, while others progress more slowly. This makes it difficult to establish a clear baseline for measuring a treatment’s impact. 

Biomarkers that track disease progression can provide a more objective way to assess how well a new treatment is working. For example, a biomarker might show if a new drug is slowing down the progression of ALS. This would be a much more reliable way to assess a treatment’s effectiveness than simply relying on changes in symptoms, and it would be a crucial step forward in the fight against this devastating disease. 

RA: The initial five years of this project will see perspectives given from those with lived experiences of ALS. How essential are these perspectives for helping drive research into the disease?  

AT: The AMP ALS program is built on the foundation of collaboration, and a key group of collaborators includes the people most affected by this disease — people with lived experience. These individuals bring a unique and invaluable perspective and guidance to the program’s direction and ensure it remains patient-centric. By directly involving them in discussions about research goals and initiatives, AMP ALS ensures the program addresses issues most relevant to their needs and well-being. Their feedback ensures that clinical studies are practical and minimise burdens on participants.  

RA: In terms of drug discovery, what are you hoping to see emerge from this project?  

AT: AMP ALS has the potential to play a key role in future drug development in ALS research. By analysing the data set AMP ALS is building, researchers might uncover entirely new pathways or molecules involved in ALS. These could be the starting points for drugs that could slow or even stop the disease. 

Biomarkers identified by AMP ALS could be the key to developing targeted therapies. These drugs would specifically address the underlying mechanisms of ALS in each patient, leading to far more effective and personalised treatments. 

RA: Are personalised medicine approaches key to treating ALS? 

AT: ALS is not a single disease and ALS treatment has the potential to become personalised. ALS has different genetic and biological factors at play, causing variations in how the disease manifests and progresses.  

The AMP ALS program will focus on comprehensive data collection and analysis that can help identify subgroups within the ALS population. These subgroups might have distinct underlying causes that will allow researchers to develop targeted therapies specific to the subgroups. 

RA: What are your anticipated outcomes from this programme?  

AT: The AMP ALS program has the potential to revolutionise the fight against ALS on multiple fronts. Here are some of the anticipated outcomes: 

  • Development of the ALS Knowledge Platform: The vast amount of data collected by AMP ALS will be a powerful tool for researchers. By analysing this data, scientists may gain a deeper understanding of the underlying mechanisms of ALS, which could pave the way for entirely new treatment strategies. 
  • Development of reliable biomarkers, to diagnose ALS earlier, as well as track disease progression and response to treatment.  
  • Development of novel and optimised clinical outcome assessments to streamline clinical trials, potentially leading to faster development and approval of new drugs for ALS. 
  • Deeper understanding of the disease, earlier diagnosis, and more effective therapies will all contribute to better outcomes for patients with ALS. 

Biography 

Alessio Travaglia, PhD, PMP, Director, Translational Science Neuroscience 

Dr Alessio Travaglia is a neuroscientist with more than 15 years of experience in basic and translational neuroscience in settings including academia, non-profit, management consulting, and venture philanthropy. He currently works as Director of Translational Science, Neuroscience, at the Foundation for the National Institutes of Health (FNIH). In this role, he leads the FNIH’s neuroscience programs, facilitates the advancement and execution of innovative public-private partnerships, and collaborates with government, industry, academia, patient advocacy groups, and private sector organisations. 

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