Impact

Dedicated to finding a cure for ALS.

ALS is not incurable – only underfunded.

Augie’s Quest to Cure ALS raises the funds and awareness urgently needed to advance cutting-edge research, fast-track effective treatments, and ultimately, find a cure for ALS.

In January 2020, the ALS Therapy Development Institute announced the Augie’s Quest Translational Research Center at ALS TDI. This Research Center honors the work, innovation and steadfast support of Augie’s Quest and its many generous and committed donors to finding a cure for ALS.  Housed within ALS TDI’s Cambridge, Mass., laboratory, the Augie’s Quest Translational Research Center will help to strengthen its existing and future research efforts by aiming to dissect mechanisms of clinical ALS and use the knowledge to develop new, more powerful drug discovery platforms and facilitate more effective drug development in ALS.

“We owe much of our progress and ability to expand this transformative, essential research to Augie’s Quest. And, we are honored to celebrate this impact on our science,” says Fernando Vieira, M.D., Chief Scientific Officer, ALS Therapy Development Institute.

2019 was a momentous year for Augie’s Quest, as we funded more than 48% of the science at the ALS Therapy Development Institute (ALS TDI).  Augie’s Quest funding allowed ALS TDI to enhance each of their existing research programs:

Precision Medicine Program (PMP):

ALS TDI’s translational research effort was initiated and is carried on in tight integration with its Precision Medicine Program (PMP), the most comprehensive and longest running translational research study in ALS. Through the PMP, researchers at ALS TDI partner with people with ALS around the world to share data that feeds into the Augie’s Quest Translational Research Center. This integration serves ALS TDI’s mission to discover and develop effective treatments for ALS.

The PMP uses big data to better understand ALS progression on a patient-by-patient basis, and provides people with ALS personalized information to guide their individual ALS journey.  Combing through these large datasets that trace and define a person’s unique experience with ALS helps accelerate our science to potentially streamline the clinical trial process, making it more agile and less expensive. We believe the next ALS breakthroughs will come from this robust program!

  • In 2019, thanks to funding from Augie’s Quest, ALS TDI expanded our Precision Medicine Program (PMP) by:
    • Implementing a first-in-kind “In Home” blood sample collection program.
      • We will identify biological “fingerprints” in the blood that can reveal effective strategies to treat ALS and, nearly as importantly, quickly reveal when a treatment is working for a person with ALS.
    • Collaborating with Google to utilize best-in-class machine learning technology to better understand ALS disease progression.
      • ALS TDI and Google scientists demonstrated that it is possible to detect and quantify ALS disease progression using voice recordings.
      • ALS TDI scientists helped Google to develop voice recognition tools for people with ALS related dysarthria (voice impairment) through Project Euphonia.

Drug Development

We are working day in and out to bring the next promising ALS treatments successfully through to clinical trials, and as quickly as possible.

One big reason we’re in this position today – is our innovative model. It’s driven by the business savvy of Augie Nieto and our board, combined with our collective, unlimited passion and vision to end ALS. We are certain one day we’ll go from saying “Lou Gehrig’s disease” to “Augie’s Cure.”

There will never be a single, magic silver bullet to treat and cure ALS. We know ALS is very complex, and each person’s ALS is unique, progressing and presenting differently. We will need multiple treatment options to support the ALS community and give every person with ALS a fighting chance against this disease.

  • In 2019, thanks to funding from Augie’s Quest, ALS TDI:
    • Operated the largest preclinical pharmacology program in all of ALS.
      • We rigorously tested 22 new potential ALS treatments in SOD1 mouse models of ALS, bringing the total number to over 370 – more than any ALS lab in the world.
    • Expanded our already large-scale drug discovery initiatives.
    • Purchased a library of 30,000 small molecules, each with potential to be an ALS drug, to be tested in a human induced pluripotent stem cell (iPSC) based screening system invented by our scientists.
    • Expanded the ALS model systems in use in the lab

 Fast-Tracking the Most Promising ALS Treatment Yet

We’ve turned a significant corner in ALS research, successfully completing a Stage 1 Human Clinical Trial for a drug called AT-1501.  This marks the very first time a non-profit organization has ever reached this stage of drug development.  In 2020, the drug company Anelixis Therapeutics, will coordinate a Phase 2a, multi-center, open label, multiple dose study of AT-1501.  Approximately 54 adults with ALS will be enrolled into the study in the United States at up to 12 ALS treatment sites.

It is likely our findings and treatment discoveries for ALS may also help fight other neurodegenerative diseases in the future. The development of AT-1501 may also have a significant impact in Type 1 Diabetes and Alzheimer’s disease as well as many autoimmune diseases including Lupus, Sjogren’s syndrome, psoriasis, and rheumatoid arthritis. 

“It’s really incredible. For 15 years, Augie’s Quest has been dedicated to raising the critical funds and broad awareness needed to help fast-track ALS TDI’s cutting-edge ALS research – specifically providing ALS TDI with more than $150 million to advance its efforts to end ALS,” commented Steven Perrin Ph.D., CEO of ALS TDI.

 

What is ALS?

Disease

Amyotrophic lateral sclerosis, or ALS, is a disease of the nerve cells in the brain and spinal cord that control voluntary muscle movement.

Diagnosis

There is no single diagnostic test for ALS. However, experts in the disease, usually neurologists specializing in neuromuscular diseases, are very capable of diagnosing it. There are, on average, 15 people diagnosed with ALS every day, and roughly 30,000 people in America have this condition.

Prognosis

Most people with ALS live 2-5 years after their first signs of disease. About 10% of people with ALS survive at least 10 years. This variable rate of disease progression makes prognosis difficult to predict and therapies challenging to develop.