Target ALS

Target ALS Founded in 2013 by Dan Doctoroff, Target ALS is a medical research foundation whose mission is to acc

The ALS Global Research Initiative ( ) is home to the largest, most inclusive global study in , spanning 14 sites acros...
08/18/2025

The ALS Global Research Initiative ( ) is home to the largest, most inclusive global study in , spanning 14 sites across multiple continents and growing. This is the Target ALS Global Natural History Study ( ), and it’s unlike anything else in the field.

While most studies focus only on the U.S. or Western populations, the GNHS is designed to reflect the true diversity of the global population. Historically, just 7% of ALS study participants are of non-Caucasian descent. Our GNHS has already reached 30%, and we continue to grow. This diversity is critical to uncovering genetic, geographic, and environmental factors that influence the disease, ensuring future treatments are made for everyone.

But to make this possible, we need participants, both people living with ALS and healthy controls. Every sample and every data point strengthens the science, fuels biomarker discovery, and accelerates the path to effective treatments.

Learn more about AGRI: https://www.targetals.org/agri/

An international team of scientists led by Dr. Arti Patel (Novartis Neuroscience), Dr. Anja Schneider (DZNE), Dr. Alexan...
08/18/2025

An international team of scientists led by Dr. Arti Patel (Novartis Neuroscience), Dr. Anja Schneider (DZNE), Dr. Alexander Thompson (University of Oxford), and Dr. Ludo Van Den Bosch (KU Leuven) is working to uncover the earliest biological clues of and .

Their focus: extracellular vesicles ( ), microscopic membrane-bound particles released by cells that may carry telltale signs of pathology, one of the most prevalent, yet elusive, hallmarks of these diseases.

By refining how these vesicles are captured and analyzed, the consortium aims to make it possible to detect disease, monitor treatments, and match those living with ALS and FTD to the right therapies—potentially before symptoms begin.

By turning invisible pathology into something measurable, EV-based biomarkers could revolutionize diagnosis and treatment monitoring for ALS and FTD.

🔍 Learn how this global partnership is bringing that vision closer to reality: https://ow.ly/HOmq50WHcBk

A global team of researchers led by Dr. Johnathan Cooper-Knock, Dr. Ophir Shalem, Dr. Michael Snyder, and Dr. Eran Horns...
08/15/2025

A global team of researchers led by Dr. Johnathan Cooper-Knock, Dr. Ophir Shalem, Dr. Michael Snyder, and Dr. Eran Hornstein is using AI, advanced imaging, and genetic screening to understand what causes ALS and how to stop it.

They identified that a gene called CCDC146, a microtubule protein that plays a role in cell structure and signaling, becomes overactive in ALS. The team developed an antisense oligonucleotide (A*O) tool to “turn down” this gene. Administration of this tool in an ALS mouse model led to longer survival of motor neurons.

By mapping changes to the “organellome”, the network of tiny structures inside cells, the team is uncovering new genes involved in disease progression and studying the effects of modulation of these genes in more detail.

🔬 Read how this team is turning deep cell biology into real-world solutions: https://ow.ly/il0e50WGPnG

*O

Not all trials succeed, but every trial teaches us something.At our 2025 Annual Meeting, Denali Therapeutics shared the ...
08/15/2025

Not all trials succeed, but every trial teaches us something.

At our 2025 Annual Meeting, Denali Therapeutics shared the top-line results of their DNL343 trial in the Healey ALS Platform Trial. While the therapy, designed to target the integrated stress response (ISR), did not meet its endpoints, the study offered critical insights that will shape future ALS research.

DNL343 successfully reached the brain, engaged its target, and showed early biomarker changes, yet did not slow disease progression. Still, Denali’s willingness to share results, even when they’re disappointing, is vital for scientific progress. We applaud their leadership in their willingness to share results from a failed clinical trial to allow the broader community to benefit from the lessons learned.

💡 Why this matters: Every trial, even the ones that fall short, brings us closer to effective treatments. Transparency, rigor, and collaboration move the field forward.

🧪 Read more about what we learned from DNL343: https://ow.ly/ehC950WGlcI

What if neurons are fueling their own destruction in ?A groundbreaking study led by Drs. Mark Albers, Clotilde Lagier-T...
08/15/2025

What if neurons are fueling their own destruction in ?

A groundbreaking study led by Drs. Mark Albers, Clotilde Lagier-Tourenne, and Brian Wainger at Massachusetts General Hospital and Dr. Isaac Chiu at Harvard is shifting how we understand inflammation in ALS, not just as a symptom, but as something neurons may cause through their own immune responses.

Their research highlights Gasdermin E (GSDME), a protein that can puncture cell membranes, leading to inflammation and cell death. By silencing GSDME in mouse models, scientists were able to rescue motor neurons and reverse ALS-like symptoms, opening the door to an entirely new therapeutic strategy.

🧠 Learn how this discovery could reshape the future of ALS treatment: https://ow.ly/6qAf50WGkGy

Dr. Frank Walsh, a pioneering force in neuroscience, pharma, and biotech, has spent his career pushing boundaries, and n...
08/14/2025

Dr. Frank Walsh, a pioneering force in neuroscience, pharma, and biotech, has spent his career pushing boundaries, and now he’s channeling all his energy into ALS. As a member of the Target ALS Independent Review Committee (IRC), Frank brings decades of experience and a personal connection to the mission of accelerating progress in ALS research.

In our latest blog, Frank shares his remarkable journey: from developing monoclonal antibodies with a Nobel Laureate, to leading billion-dollar R&D pipelines, to launching a biotech transforming drug delivery to the brain. And now, he's focused solely on ALS with hope, urgency, and unwavering commitment.

💡 Read the full spotlight on a leader whose legacy is helping to shape the future of ALS research: https://ow.ly/EBlf50WGkqC

“We are not merely an interesting academic question; we are a population of people, and we are everywhere.” – Jean Swidl...
08/12/2025

“We are not merely an interesting academic question; we are a population of people, and we are everywhere.” – Jean Swidler, co-founder of End the Legacy

At Target ALS, we recognize that the future of ALS research and therapeutic development must be informed by those most directly affected.

That’s why we’re honored to partner with Genetic ALS & FTD: End the Legacy, a patient-led organization founded by individuals with deeply personal ties to familial ALS and FTD. In just over a year, they’ve created a powerful platform that redefines what community engagement in science can look like, one that brings researchers, ethicists, and clinicians into a space designed by and for the impacted community.

Together, we’re working to close critical gaps in inherited ALS research, elevate lived experiences, and shift the culture of how progress is defined, from discovery to diagnosis to clinical trials.

💡 Read more about this powerful partnership and how we’re building a future where everyone affected by ALS has a voice in the science that shapes their lives: https://ow.ly/57lW50WETrN

Now live: significant expansion of one of the most comprehensive multi-omics datasets for and research to date.The T...
08/12/2025

Now live: significant expansion of one of the most comprehensive multi-omics datasets for and research to date.

The Target ALS Data Engine has expanded with groundbreaking datasets from our ALS Global Research Initiative ( ), featuring:
🧪 MS Proteomics (TMTpro 35-plex) profiling 2,400 proteins and 28,000 peptides from matched blood and CSF in 43 healthy controls and 56 people with ALS
🧬 Whole-genome sequencing: 126 short-read and 70 long-read (PacBio HiFi) genomes from participants in the Global Natural History Study
📊 Paired clinical data, including motor and cognitive assessments, longitudinal follow-up, and genetic insights
🌎 Diverse patient representation

This integrated platform empowers researchers to identify new biomarkers, stratify patients more precisely, and validate therapeutic targets—advancing discovery across ALS and FTD.

None of this would be possible without the individuals with ALS and healthy controls, who generously donated biosamples through AGRI. Their generous contributions are fueling the future of diagnosis, treatment, and hope.

🔍 Learn more and access the data now from anywhere in the world via our no-strings-attached Data Engine: https://bit.ly/4mINrHi

🌍 A Global Community. A Shared Mission.At our 2025 Annual Meeting, more than 960 attendees from over 32 countries came t...
08/08/2025

🌍 A Global Community. A Shared Mission.

At our 2025 Annual Meeting, more than 960 attendees from over 32 countries came together. This included academic leaders, people with ALS and their loved ones, nonprofit representatives, and biotech, pharma, and VC partners, all committed to accelerating the future of ALS research.

With $25 million being invested directly into research in 2025, we continue to drive meaningful progress:

🔹 10 clinical trials launched from Target ALS-funded work
🔹 53% of funded consortia led to drug discovery programs
🔹 6 biotech companies launched from Target ALS-funded work
🔹 350+ users accessing the Target ALS Data Engine

These milestones signal momentum, not a finish line. With only three approved therapies and hundreds of thousands affected worldwide, our work is far from over. We remain focused on building a future where Everyone with ALS Lives.

Read the full Annual Meeting Impact Report: https://bit.ly/4famBFf

What if we could track ALS in real time before symptoms worsen?Abnormal aggregation of the protein TDP-43 can be detecte...
08/08/2025

What if we could track ALS in real time before symptoms worsen?

Abnormal aggregation of the protein TDP-43 can be detected in motor neurons of the postmortem brain and spinal cord of ~97% of people with ALS. How do we find a way to detect these changes in the central nervous system early in the disease process? That’s the question driving scientists Leonard Petrucelli and Nicholas Ashton as they work to develop one of the most sought-after tools in ALS research: a reliable, non-invasive biomarker for ALS.

With support from Target ALS, the duo is developing methods to measure even small amounts of “cryptic peptides” in the cerebrospinal fluid and blood from living patients. These abnormal peptides appear when TDP-43 malfunctions and can be present before significant aggregates form. Cryptic peptides may therefore serve as an early biomarker of ALS. If successful, this work could lead to a non-invasive way to detect early signs of disease in people carrying genetic mutations and monitor disease progression and treatment response in clinical settings.

By combining Petrucelli’s antibody expertise with Ashton’s experience building ultrasensitive tests, they’re bringing us closer to a future where care can be more timely, personalized, and precise.

🔍 Read the story behind this groundbreaking partnership and what it could mean for people living with ALS: https://bit.ly/45vb0wc

In , a protein called stops working properly and clumps in the wrong part of the cell, disrupting how nerve cells fun...
08/07/2025

In , a protein called stops working properly and clumps in the wrong part of the cell, disrupting how nerve cells function and survive. One major consequence is a drop in levels of STMN2, a protein essential for nerve repair, which may lead to or contribute to the damage seen in ALS.

Now, a bold collaboration between Denali Therapeutics and UC San Diego is targeting a new player in this process: OTUD4. This protein is found in RNA granules—cell structures that regulate which genes are turned on or off. The team hypothesized that by lowering OTUD4, TDP-43 would be free to regain its normal function. In addition to testing this hypothesis, the researchers are developing a novel delivery system to help the potential therapy cross the blood-brain barrier.

With early results showing promise, this work marks a potential leap forward: restoring lost gene function, improving how treatments reach the brain, and advancing a new class of targeted therapies for ALS.

🔬 Read more: https://www.targetals.org/2025/07/21/unlocking-rna-granules-a-promising-therapeutic-pathway-in-als/

With support from Target ALS, Dr. John Andersson and Dr. Caroline Ingre are uncovering striking variations in how T cell...
08/06/2025

With support from Target ALS, Dr. John Andersson and Dr. Caroline Ingre are uncovering striking variations in how T cells behave in people with . Their findings suggest that the immune system’s role in ALS is both complex and deeply personal, opening the door to more precise, immune-targeted therapies.

They’re part of a growing group of researchers we fund to pursue bold, boundary-pushing ideas—because tackling ALS from all angles means investigating every system, every signal, and every possibility that could bring us closer to effective treatments, and ultimately, a world where .

🧠 Read more about their work and the future of immune-based precision in ALS: https://bit.ly/4omH5if

Address

New York, NY

Telephone

+13323334140

Website

Alerts

Be the first to know and let us send you an email when Target ALS posts news and promotions. Your email address will not be used for any other purpose, and you can unsubscribe at any time.

Shortcuts

Featured

Share

Category