NIH Awards $2.5M Grant to Amydis to Develop Eye Test for ALS Biomarker

Early detection remains one of the biggest challenges in neurodegenerative diseases. Conditions like Amyotrophic Lateral Sclerosis, Frontotemporal Dementia, and Limbic‑predominant Age‑related TDP‑43 Encephalopathy often go undiagnosed for months or even years.

A new research effort aims to change that — using the human eye as a diagnostic window.

Amydis Inc., a clinical-stage biotechnology company developing ocular tracers, has received a $2.5 million Phase 2 grant from the National Institute on Aging, part of the National Institutes of Health.

The funding will support the development of a non-invasive eye test designed to detect TDP-43, a key biomarker linked to several neurodegenerative diseases.

Why Detecting TDP-43 Matters?

A protein called TAR DNA‑binding protein 43 (TDP-43) plays a central role in multiple neurological conditions.

In fact:

• Around 97% of ALS patients show TDP-43 pathology
• The protein is also linked to FTD and LATE
• Currently, no blood test or brain imaging method can detect TDP-43 in living patients

Because of this limitation, ALS is still largely diagnosed by ruling out other conditions. The process can take months or even years.

Earlier detection could help:

• Start treatments sooner
• Improve clinical trial enrollment
• Enable faster drug development

A New Approach: Diagnosing Brain Disease Through the Eye

Amydis is developing a fluorescent ocular tracer designed to detect TDP-43 deposits in the retina.

The concept is simple but powerful.

Instead of relying on invasive procedures or complex imaging, the tracer could allow doctors to identify disease biomarkers through routine eye imaging technologies already used in ophthalmology clinics.

This approach could make early neurological screening far more accessible. According to Stella Sarraf, Founder and CEO of Amydis:

“For patients and families facing ALS and related dementias, time is everything. The eye represents a new frontier in neurodegenerative disease detection.”

If successful, the technology could integrate molecular biomarker detection into everyday eye exams.

What Phase 1 Research Already Demonstrated?

The new funding builds on results from an earlier NIH Phase 1 grant.

In that study, Amydis demonstrated that its fluorescent tracer could detect TDP-43 deposits in retinal tissue samples from patients with ALS, FTD, and LATE.

The work involved collaborations with several major research institutions, including:

• Georgetown University
• Washington University in St. Louis
• Mass General Brigham
• Cedars‑Sinai
• Banner Health

The findings were presented at two major scientific meetings:

• Northeast ALS Consortium Conference
• International Symposium on ALS/MND

A peer-reviewed manuscript is currently being prepared.

What the New Phase 2 Grant Will Support

The Phase 2 funding will expand the research in several ways.

Scientists will:

• Conduct further analysis of retinal cadaver tissue samples
• Map and characterize TDP-43 deposits in the retina
• Apply artificial intelligence to identify disease-specific biomarker patterns

The goal is to determine whether retinal imaging could eventually distinguish between ALS, FTD, and LATE at the molecular level.

That capability would be a major step toward precision medicine for neurodegenerative diseases.

Why Researchers Are Paying Attention?

Experts in the ALS research community see strong potential in the approach.

Merit Cudkowicz, Executive Director of the Mass General Brigham Neuroscience Institute, noted that detecting TDP-43 before symptoms fully develop could significantly advance the field.

A reliable biomarker test could help:

• Improve clinical trial design
• Enable more targeted drug development
• Allow earlier patient enrollment in trials

This could accelerate the path toward new treatments.

Looking Ahead: The Eye as a Diagnostic Window

Amydis is building a broader platform around ocular molecular diagnostics.

The company’s technology focuses on developing proprietary tracers capable of identifying biomarkers linked to diseases affecting the:

• Brain
• Heart
• Eye

Alongside this effort, the company is also developing a multi-omics data platform combining ocular biomarkers with AI-driven analytics.

The long-term goal is to transform routine eye exams into powerful tools for early disease detection.

If the approach proves successful, the eye may soon become one of the most accessible ways to detect complex neurological disorders — long before symptoms appear.

Optimize Your trial insights with Clival Database.

Are you exhausted from the uncertainty of trial insights pricing? Clival Database ensures the clarity in the midst of the global scenario for clinical trials to you.

Clival Database is one of the best databases that offers an outstanding number of clinical trial data in terms of 50,000+ molecules and from primary regulatory markets as well as new entrants like Indian and Chinese markets.

With Clival, you get accurate positioning of historical sales data, patent database, company profiling, safety & efficacy, and prediction of launch of new innovative molecules helping you to align your research and driving down the cost.

To add value, we further break down our analytics for you so that improving your operational effectiveness; optimizing your clinical trials; and offering you accurate and high-quality data at lowest possible prices becomes possible.

Elevate your trial success rate with the cutting-edge insights from Clival database.

Check it out today and make more informed sourcing decisions! Learn More!