Curious about the groundbreaking research happening at ALS TDI? We're on the cutting edge of ALS research, driving innovation at every stage of drug discovery and therapeutic development. Here are our active ALS research initiatives.


Advancing Potential ALS Therapies

ALS TDI researchers are dedicated to discovering and developing ALS therapies that can be advanced to human trials. These are the most advanced drug programs in our preclinical pipeline:

  • Copper Complexes:  Evidence shows that copper mismanagement in ALS and other neurodegenerative diseases can result in cellular dysfunctions. ALS TDI is investigating copper complexes as potential treatments for ALS – focusing on their ability to regulate oxidative stress and copper balance. We have selected a lead drug candidate for IND enabling studies. ALS TDI is also assessing patient stratification strategies for future clinical development of this lead molecule.
  • Type 1 PRMT Inhibitors: In 2020, ALS TDI researchers discovered that type 1 PRMT inhibitors can alter ALS-linked proteins associated with the C9orf72 mutation, paving the way for targeted treatments. Recent studies indicate that type 1 PRMT inhibitors may also treat other types of ALS. ALS TDI is advancing these inhibitors into animal testing to assess their potential for human trials. Learn more about this drug program

Innovation in ALS Drug Testing

ALS TDI rigorously tests thousands of treatments to identify those with the most potential for advancement. Our drug testing programs include:

  • Corsalex Collaboration: ALS TDI is partnering with Corsalex, a biotech firm specializing in therapies for C9orf72 spectrum disorders, to evaluate several therapeutic leads. This partnership aims to prioritize the most promising therapies for development and rapid advancement toward clinical trials. Learn more about this collaboration.
  • iPSC Drug Screening: ALS TDI uses induced pluripotent stem cell (iPSC) models of ALS to efficiently screen for potential therapies. IPSCs are created through a process that allows researchers to “differentiate,” or change, cells taken from anywhere in the body back to the state of a stem cell. Then, these stem cells can be further differentiated into any other type of cell, including motor neurons – the cells affected by ALS. By using patient-derived cells from the ALS Research Collaborative (ARC) study, we replicate ALS pathology and study the disease in a human cell context, increasing the likelihood of finding relevant therapeutic targets. Explore iPSC drug screening in ALS.
  • Expanded Animal Models of ALS: Research suggests that ALS is not a single disease. Using diverse animal models to represent different types of ALS is crucial for research and treatment development. ALS TDI is expanding its use of a variety of animal models to improve the translatability of preclinical findings to human studies. Learn about animal models in ALS.


Pioneering ALS Research Studies

ALS research is vital for understanding the disease, identifying risk factors, and developing effective treatments. ALS TDI’s current research studies include:

  • ALS Research Collaborative: The ALS Research Collaborative (ARC) is an initiative to collect and share comprehensive lifestyle and genetic data from people with ALS, to learn more about the disease and accelerate the discovery of treatments. This ongoing study has already led to the discovery of new biomarkers in ALS and is vital to much of the research carried out at ALS TDI. Join the ARC study.
  • Study of ALS Reversals: In collaboration with Dr. Richard Bedlack of the Duke University ALS Clinic, ALS TDI is studying rare cases of ALS reversals to uncover potential mechanisms that could inform new therapeutic strategies. Initiated in 2021, this program involves detailed analysis of clinical data and biological samples from people with ALS who have experienced unexpected symptom improvements. Learn more about the ALS reversals study.
  • CDC Funded Study on ALS Risk Factors: In August 2023, ALS TDI secured a substantial grant from the CDC to study lifestyle risk factors associated with ALS. This study aims to inform preventative measures or early interventions by increasing our understanding of environmental, occupational, and other risk factors. Currently in the data optimization stage, the study utilizes data from the ARC study. Learn more about our study on lifestyle risk factors.

Expanding ALS Data Sharing:

Data sharing accelerates the discovery of new treatments by enabling researchers to build on each other's work and validate findings across larger datasets. ALS TDI is involved in the following data-sharing efforts:

  • ARC Data Commons: The ARC Data Commons is a platform developed by ALS TDI to share and analyze the comprehensive data collected through the ARC Study. It allows researchers worldwide to access and analyze this deidentified data, enhancing collaborative research and accelerating the discovery of new treatments and biomarkers. Explore the ARC Data Commons.
  • Neuromine Collaboration: In March of 2024 ALS TDI, Answer ALS, and Microsoft announced a collaboration that will bring together the two largest existing clinical and omics datasets relating to ALS. Through this partnership, comprehensive data collected through ALS TDI’s ALS Research Collaborative will be added to Neuromine, a data portal launched in 2021 by Answer ALS and powerred by Microsoft Azure. Read the collaboration announcement.
  • Unite Genomics and EHRs: In August 2023, ALS TDI partnered with Unite Genomics to integrate electronic health record (EHR) data into the ARC study. This collaboration aims to advance ALS research by leveraging the power of ARC study participants' complete medical histories. Learn about this data-sharing effort.


New ALS Biomarker Discovery

Reliable biomarkers for ALS can improve diagnosis, better track disease progression, and provide more precise endpoints for clinical trials. Here are ALS TDI's current efforts in biomarker discovery:

  • Google Voice Scoring: In collaboration with Google researchers, ALS TDI has developed voice scoring algorithms to monitor ALS progression through speech analysis. This innovative program, which uses data collected through the ARC study, was initiated to improve patient monitoring and outcome assessment. Discover our digital biomarkers.
  • Google and MGH Movement Monitoring: ALS TDI is partnering with Google and Massachusetts General Hospital (MGH) to use accelerometer data from wearable devices to detect subtle changes in movement in people with ALS. This initiative provides another digital biomarker that can be used accurately and objectively to measure ALS progression and outcomes. Learn more about these research findings.
  • LifeArc Collaboration: ALS TDI is collaborating with LifeArc to identify and validate blood-based biomarkers for ALS. This program, using blood sample data from the ARC study, aims to enhance early diagnosis and monitor therapeutic responses. Learn more.
  • DoD Funded Biomarker Study: In 2022, ALS TDI received a $281,000 grant from the U.S. Department of Defense to identify new protein-based biomarkers for ALS. ALS TDI researchers are now working on comprehensive molecular and genetic analyses to discover novel biomarkers and therapeutic targets. Read the study announcement.

Leveraging New Technologies

Leveraging new technologies in ALS research offers innovative approaches to discovering new treatments.  ALS TDI's researchers are currently focused on the following technologies:

  • mRNA: mRNA represents a relatively new therapeutic modality, or type of drug, in all of biomedicine, particularly in central nervous system diseases like ALS. ALS TDI is now exploring the use of mRNA in our ALS research. This promising program, currently in the preclinical stage, aims to harness mRNA technology to develop potential treatments for ALS.


Current Research Collaborations

Collaboration in ALS research is essential because it allows for the pooling of resources, expertise, and data. Click the links below to learn more about some of our current research collaborations.

To learn more you can watch recordings the 2024 ALS TDI Summit, featuring presentations about the latest research at ALS TDI and beyond!

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