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How Do We Find Effective Treatments

Finding new medicines to treat diseases is a long process. In order to identify the most promising treatments, potential therapies must move through a series of steps to determine safety and effectiveness. This set of steps is often referred to as the "Drug Development Funnel".
See how ALS TDI uses this comprehensive approach to move potential ALS treatments through the funnel. Watch this video to learn about the Drug Development Funnel.
Relentless Research
We are dedicated to discovering and developing effective treatments to end amyotrophic lateral sclerosis (ALS). We will not stop until there are treatments for every single person living with ALS. Our cutting edge approach has resulted in:
  • The rigorous screening of over 400 potential treatments for ALS—more than any other ALS research lab in the world.
  • The world's first Precision Medicine Program, a partnership with people living with ALS, designed to speed up drug discovery and therapeutic development for ALS.
  • ALS TDI becoming the first nonprofit biotech in any disease to invent a potential treatment, AT-1501, and bring it from our own labs, through FDA review and into clinical trial.
  • The improvement of worldwide standards for preclinical drug screening in models of ALS.
Research Relentless

Our Approach to End ALS

At ALS TDI we understand the urgent need to discover and develop potential treatments for ALS. Our comprehensive approach to ALS research spans every aspect of drug discovery, research, and therapeutic development. Click the video to learn more about the research being done at ALS TDI.
Our Comprehensive Approach to
Discovering Effective Treatments
1 We Operate
One of the largest preclinical drug validation programs in als.
There are too few potential treatments in clinical trials for ALS. We need a robust, good quality drug pipeline to ultimately treat every person with ALS. More treatments mean more hope.
2 We Validate
More models of als to allow for rigorous testing.
ALS is extremely complex involving a myriad of biological pathways. To test drugs more rigorously, we need a variety of both cell and animal models to capture all of the complexities of ALS.
3 We Aim
To improve how als clinical trials are run.
Clinical trials for ALS currently require too many participants. That slows the studies down and makes them very costly. Using emerging technologies, we hope to make clinical trials more efficient to reveal the most promising treatments faster.
4 We Learn
About ALS from people with ALS.
We believe that best way to learn more about ALS is to partner with people with ALS. Through our Precision Medicine Program, we combine medical histories, family histories, genetics, biomarkers, and patient cell biology to better understand the processes that drive and influence ALS.
How We're Targeting ALS
ALS is a complex disease involving many biological pathways. These include oxidative stress, immune modulation, unfolded protein response, and more. This means that we have a number of targets to take aim at when testing potential treatments for ALS. At ALS TDI we work on many projects simultaneously. This ensures that there is a robust pipeline of potential treatments to treat every person with ALS.
How We're Targeting ALS
Precision Medicine Program
Our Precision Medicine Program (PMP) is the most comprehensive and longest running translational research study in ALS. Through the PMP, our researchers partner with people with ALS to share and gather data to better understand the disease. Our ultimate goals are to discover new treatments for ALS and make clinical trials faster and more efficient. But it only works if you participate.
Our Leaders in ALS Research
Meet the scientists who are leading our research to find effective treatments for ALS. Click on our leadership team members to learn more about their experience.
To learn more about our science team, click here