ALS TDI maintains an unparalleled and unique research approach

The Institute focuses their research in four main areas:

  1. Operating “one of the largest preclinical drug validation programs in ALS” to produce a strong, high-quality drug pipeline.

  2. Validating more cell and animal models of ALS to identify and understand the complexities of the disease, which allows for rigorous testing to ensure drug efficacy.

  3. Using new technologies to improve the clinical trials process in an effort to increase efficiency, which will “reveal the most promising treatments faster.”

  4. Learning more about ALS from people with ALS in the Precision Medicine Program. This is done by combining “medical histories, family histories, genetics, biomarkers, and patient cell biology to better understand the processes that drives and influences” the disease.

Because ALS TDI is a nonprofit, they are not limited to using specific technologies or developing certain treatments. As a result, they are able to explore multiple drug pipelines based on different biological pathways at the same time.

The Institute’s research practices build off of the work of others. Researchers often seek to reproduce or expand existing studies in an effort to duplicate results and further establish findings. ALS TDI does this particularly with research conducted by graduate students during their education, as many of the studies are not typically follow up upon.

As part of their commitment to “open-door science,” as well as embodying the ethos of a community-funded lab, anyone can request to tour ALS TDI’s lab or seek more information from their scientists.

precision medicine program

According to TDI, “the Precision Medicine Program (PMP) is the most comprehensive and longest running translational research study in ALS. Through PMP, researchers at ALS TDI partner with people with ALS around the world to share and gather data on voice, movement, lifestyle, medical and family histories, genetics, biomarkers, and patient cell biology to better understand the disease.” PMP allows the Institute to:

  • Discover new targeted treatments for ALS based on differing biomarkers

  • Make clinical trials faster and more efficient

  • Empower people with ALS by giving them access to their own data to track their disease progression, unlike other studies

The more than 550 participants of the Program wear accelerometers approximately six times per month to track their fine motor movements, in addition to providing speech recordings, lifestyle surveys, blood samples, genome samples, and ALSFRS-R scores. Each participant is provided with a secure log-in to an online portal, where they are able to see the reporting of their provided data.