ALS TDI is the Drug Discovery Engine for ALS. Thanks to your support, we continue to advance potential treatments and learn more about ALS.
Here is a look at the progress that we have made in 2021, and how we will build upon this progress in 2022 to advance ALS drug discovery.
Advancing Redox Metabolism Modulators
- In 2021 ALS TDI scientists discovered “redox metabolism modulators” – a collection of drug-like molecules that they believe may have potential to treat many types of ALS. Tests that they performed in iPS cells and in the leading animal model of ALS have shown that these molecules can protect against ALS related cell death.
- In 2022, we will work to identify and optimize a lead small-molecule drug candidate through further in vivo experiments to advance toward clinical testing.
Advancing Type-1 PRMT Inhibitors
- Throughout 2021, our research team worked to further unlock the potential of their new drug target discovery, Type-1 PRMTs. We began testing treatments against this target, with the hope of identifying one that we can advance to animal testing. Though this target was discovered for C9orf72-related ALS, ALS TDI scientists believe that these findings may also be relevant to other forms of ALS.
- In 2022 we will work to validate up to five new animal models of ALS to test Type-I PRMT inhibitors. These include models that will allow us to further test their potential to treat C9orf72-related ALS, as well as investigate whether they may be effective in other forms of the disease.
Study of ALS Reversals with Dr. Richard Bedlack
- In 2021, ALS TDI announced a partnership with Dr. Richard Bedlack and the Duke ALS Clinic to enroll people who represent the rare instances of ALS reversals into the Precision Medicine Program (PMP). Our goal is to learn more about the underlying biological pathways behind these possible reversals and to use this understanding to inform treatment discovery and development.
- In 2022 the Precision Medicine Program will continue enrolling individuals who Dr. Bedlack has documented as having experienced ALS Reversals. We will analyze samples provided by these people in comparison with those with fast-progressing, slow-progressing, and other kinds of ALS in order to search for biological differences that may reveal potential drug targets or other avenues for treatment.
Google Collaboration on Voice Scoring
- In 2021, we continued to collaborate with Google to apply a voice scoring machine algorithm to recordings contributed by PMP participants. We began sharing these scores with participants to give them a numerical measure of their own speech wellness.
- In 2022, the algorithm scores will be shared with each participant to give them a numerical measure of their own speech wellness. Our hope is that these voice scores can be used to develop digital biomarkers of ALS disease progression.
Moving Potential Treatments to Animal Testing
- In 2021 ALS TDI scientists tested 26 potential treatments in animal models of ALS, including hits from the library of 36,000 small molecule compounds that we acquired in 2019. Those that show potential will be assessed tolerability, side effects, and efficacy – allowing us to continue to fuel our drug discovery engine.
- In 2022, we will screen possible Redox Metabolism Modulator drug candidates in animal models of ALS, while also continuing to fuel the pipeline through in vivo screens of more than 20 other promising drug candidates.
We look forward to continuing to share research news and updated throughout 2022!
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