How We're Making an Impact in 2023
With your support, ALS Therapy Development Institute (ALS TDI) scientists will work relentlessly to fuel the clinical pipeline by discovering and advancing potential ALS treatments.
Here is how ALS TDI aims to advance ALS drug discovery in 2023.
Launch a First-in-Kind Global ALS Data Sharing Platform
In 2023 ALS TDI will launch the ALS Research Collaborative (ARC) Data Commons – a data sharing and visualization platform that will invite researchers, academics, and industry partners to analyze the comprehensive ALS natural history and omics data that has been collected through the Precision Medicine Program (PMP) since 2014.
- This is the first time that ALS natural history data of this scale will be made available for analysis by researchers around the world.
- Through ARC Data Commons we will provide innovative tools for researchers to analyze these deidentified data so that they can define and characterize ALS patient subsets and discover potential targets to treat these subsets.
- We will build on these data by inviting more people with ALS to join our study – because more data has the potential to make a greater impact on research.
Accelerate ALS Blood-Based Biomarkers Discoveries
In 2023 we will utilize a $281,000 grant from the Department of Defense to reveal potential blood-based biomarkers for ALS. These biomarkers could allow us to better diagnose the disease, more accurately track disease progression, and find more targeted drugs.
- We will do this by analyzing over 7,000 proteins in blood samples shared by people with ALS in the PMP to reveal potential protein-based biomarkers and potential therapeutic targets.
- Using these PMP blood samples we will also analyze over 20,000 mRNA transcripts to reveal RNA-based biomarkers and potential therapeutic targets.
Advance Two Promising Drug Programs Toward Clinical Trial
In 2022 ALS TDI continued to advance two of the most promising discoveries that have emerged from our research - Type I PRMT inhibitors and Copper Complexes.
In 2023 ALS TDI will advance these discoveries to the next phases of testing in our lab
- We will advance Copper Complexes – one of ALS TDI’s most promising series of potential drugs for ALS – closer to human trials by identifying a lead molecule and initiating larger animal studies required to commence human studies.
- We will simultaneously work to advance ALS TDI’s discovery of PRMT inhibitors. These drugs have proven to effectively target a faulty pathway in motor neurons of people with C9orf72-related ALS, but we believe that they may be effective in many forms of ALS.
Continue to Fuel the Clinical Pipeline by Identifying New Drug Targets for ALS
In 2023 our scientists will work to perform large-scale screens to identify new potential drug targets in our labs
- We will do this by completing a robust genetic screen in stem cell-derived motor neurons to discover new drug targets.
- We will also accelerate our research efforts to discover drug targets related to C9orf72, a gene associated with the most common form of genetic ALS, and TDP43, a protein found to be dysfunctional in almost all forms of genetic and sporadic ALS.
What to do next?
- Have questions? Talk to someone at ALS TDI to learn more about our current research. Contact us
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- View our 2022 ALS TDI Research Milestones. View Milestones
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