With your support in 2023, the ALS Therapy Development Institute (ALS TDI) fostered collaboration and innovation in our lab and beyond, propelling us closer to accomplishing our mission of ending ALS. Here’s how we made an impact in 2023: 


Launch of ARC:  The World’s Most Comprehensive Natural History Study in ALS

In March, ALS TDI introduced the ALS Research Collaborative (ARC), an ambitious initiative to better understand the underlying biology of ALS and expedite treatment discovery. By gathering and analyzing comprehensive data from people with ALS worldwide, the ARC study is facilitating breakthroughs in ALS research.


ARC Expansion: Doubling Efforts for Deeper Insights

In June we saw the approval of several additional enhancements of the ARC Study, including: 

  • Doubling the number of participants over the coming years, exponentially increasing the depth and impact of our data. 
  • Integrating electronic health records into the study, providing a previously untapped wealth of data about participants’ medical history. 
  • Inclusion of asymptomatic carriers of ALS-related mutations – presenting an unprecedented opportunity to gain insights into the influence of genetics on ALS risk and the early stages of the disease. This initiative aligns with our vision for advancing ALS detection, treatment, and prevention.


Global Data Access: ARC Data Commons Revolutionizes Research

 In March 2023, we officially launched the ARC Data Commons. This pioneering ALS data-sharing platform allows researchers around the world to access the large, ever-expanding ARC Study dataset and provides powerful tools for filtering and visualizing these data. By allowing researchers across the ALS space access to the most comprehensive natural history study in ALS, the ARC Data Commons will accelerate the pace of research globally.


Innovative Trial Guidance: Introducing the ALS Trial Navigator 

In 2023, we partnered with the ALS community to tackle the complexities of searching for clinical trials and to help educate people with ALS about their options. In early 2024 we will unveil the ALS Trial Navigator—a user-friendly platform that guides people with ALS and their caregivers through the process of determining their preferences and likely eligibility for clinical trials tailored toward their specific condition.


Trailblazing Research: Unveiling ALS Lifestyle Risk Factors

In August 2023, ALS TDI secured a substantial grant from the Centers for Disease Control and Prevention (CDC) to fund a groundbreaking  study aimed at uncovering lifestyle risk factors associated with ALS. This study’s findings have the potential to inform preventative measures or early interventions in ALS by increasing our understanding of potential environmental, occupational, and other risk factors for the disease.


Drug Discovery Collaborations: Pioneering Partnerships in 2023

In 2023, our commitment to fueling the clinical pipeline led to several pioneering collaborations, including a strategic partnership with Corsalex to discover treatments for ALS caused by the C9orf72 mutation.

 

Lab Advances: Propelling Promising ALS Treatments Forward 

In 2023, our researchers tirelessly advanced ALS drug programs. Notably, copper complexes and type I PRMT inhibitors moved closer to clinical testing, with copper complexes entering IND enabling studies and type I PRMT Inhibitors progressing to animal testing.


Groundbreaking Discoveries: Peer-Reviewed Papers Unveiled

In 2023, ALS TDI scientists published two peer-reviewed papers in prestigious journals: 

  • April 2023: Frontiers in Cellular Neuroscience published our insights into how C9orf72 gene mutations lead to abnormal protein toxicity in motor neurons. 
  • August 2023: A collaborative paper in Nature showcased the reliability of accelerometers, providing a more sensitive measure of ALS progression than traditional assessments, using data from the ARC study.


What to Do Next: