ALS Therapy Development Institute

Augie's Quest Translational Research Center

ALS is not a single disease, and meeting the needs of everyone with ALS will require multiple treatments.

The Augie's Quest Translational Research Center (AQTRC) is central to the ALS Therapy Development Institute's mission of developing effective treatments for all people with ALS.

By studying ALS in people with ALS, translational research bridges the gap between understanding the biology of the disease and translating this knowledge into meaningful advancements.

The AQTRC empowers ALS TDI scientists to conduct pioneering translational research, providing the state-of-the-art facilities and resources essential for progress.

AQTRC: Key Focuses of Research

Understanding ALS Mechanisms: Researchers investigate the biological processes that drive ALS onset and progression by working with cells collected from people with ALS.

Accelerating Drug Discovery: By studying how ALS affects cells, like motor neurons, from people with ALS, our team gains insights that facilitate the discovery of high-quality drug candidates.

Bridging Research and Treatment: ALS TDI increases the likelihood that findings will lead to effective treatments by identifying drug targets and developing tailored therapies for patient subsets.

AQTRC: Current Work

Research carried out through the AQTRC enables each of these impactful initiatives.

Rapid Drug Screening

Utilizing induced pluripotent stem cell (iPSC) models, the center has developed high-throughput screening methods to test and optimize drug candidates swiftly and effectively.

Identifying Drug Targets

Ongoing studies, using high-content techniques, aim to identify and validate specific drug targets and pathways in ALS, paving the way for more targeted therapies and the discovery of drug candidates.

Leveraging mRNA Technology

Researchers are pioneering the use of mRNA technology as a method for treating ALS.

Genome Sequencing

Researchers are pioneering the use of mRNA technology as a method for treating ALS.

Identifying ALS Biomarkers

The center uses data shared by participants in the ARC Study to search for blood-based ALS biomarkers. These biomarkers could allow us to better predict disease progression and assess the effects of treatments.

Advanced Drug Programs Funded by Augie's Quest

In addition to supporting the AQTRC, Augie's Quest provides critical funding to advance our most promising therapies toward clinical development, including:

Tegoprubart

Tegoprubart is the first ALS treatment developed by a nonprofit biotech (ALS TDI) to reach human clinical trials, completing a Phase 2a trial in May 2022.

Copper Complexes

ALS TDI is investigating copper complexes as potential treatment for ALS. A lead drug candidate has been selected for IND-enabling studies, the final step before human trials.

Type 1 PRMT Inhibitors

ALS TDI researchers are also advancing type 1 PRMT inhibitors as potential ALS treatments. ALS TDI is now advancing them into animal testing to evaluate their potential for human trials.

Naming of Augie's Quest Translational Research Center

Research at the Augie's Quest Translational Research Center is made possible by the unwavering support of the Augie's Quest to Cure ALS organization and its generous, dedicated supporters.

The center is named in loving memory of Augie Nieto, founder of Augie's Quest to Cure ALS and ALS TDI's board chair from 2007 until his passing from ALS in 2023. Augie's visionary leadership and tireless efforts raised more funds for ALS research than almost any individual in history, serving as the driving force behind many of ALS TDI's advancements in drug discovery.

Today, his legacy lives on through his wife, Lynne Nieto, who continues his work as ALS TDI's board chair and the passionate leader of Augie's Quest to Cure ALS.

About Augie's Quest to Cure ALS

Founded by Augie Nieto, Augie's Quest to Cure ALS is the unwavering force driving breakthroughs in ALS research, defying limitations, and empowering hope for a future without this devastating disease. Augie's Quest has been working with the ALS Therapy Development Institute since 2006 to bring new treatments and ultimately a cure to those living with ALS. Augie's Quest is committed to the greater goal of ending the disease for good.

www.augiesquest.org