NIH awards Mass General $8.3 million to expand access to experimental ALS drug

Overview

Updated Sep 17

The National Institutes of Health (NIH) awarded $8.3 million to Massachusetts General Hospital to run a program giving ALS patients access to an experimental drug they can't get through clinical trials. The program, called an intermediate-size expanded access protocol (EAP), begins September 1.

The drug, NUZ-001, targets TDP-43, a protein that clumps inside motor neurons in about 97% of ALS cases. If the therapy works, the protocol could speed its path toward Food and Drug Administration (FDA) approval. If it doesn't, the program still produces safety data that tells researchers what to try next.

Why it matters

ALS patients outside clinical trials gain funded access to a promising drug, and the data will shape its FDA fate.

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Key Indicators

$8.3M

NIH award to Mass General

Obligated federal funding for the NUZ-001 expanded access protocol.

97%

ALS cases with TDP-43 pathology

Share of ALS patients whose motor neurons show TDP-43 protein clumps, the defect NUZ-001 targets.

Sept 1, 2026

Program start date

When the expanded access protocol begins enrolling patients at Mass General.

Voices

Curated perspectives — historical figures and your fellow readers.

Organizations Involved

Massachusetts General Hospital

Academic medical center

Award recipient and EAP operator

The largest hospital-based research enterprise in the U.S. and a teaching hospital of Harvard Medical School.

National Institutes of Health

Federal research agency

Funder of the award

The U.S. government's principal biomedical research agency, funding roughly 50,000 competitive grants each year.

Timeline

August 2026 September 2026

2 events Latest: September 1st, 2026 · 2 months ago

September 2026
Expanded access protocol begins at Mass General
Latest Program Start

The NUZ-001 EAP begins enrolling ALS patients at Mass General, per the award record's work start date.
September 1st, 2026
August 2026
NIH records $8.3M award to Mass General for NUZ-001 expanded access in ALS
Funding

Federal award record shows the National Institutes of Health obligated $8.3 million to The General Hospital Corporation for an intermediate-size expanded access protocol for NUZ-001, an experimental ALS therapy.
August 28th, 2026

Scenarios

NUZ-001 Wins Breakthrough or Accelerated Approval

Possible Resolves by Q3 2028

Discussed by: ALS patient advocacy groups and clinical trial observers

The EAP enrolls dozens of patients. Safety data look clean and efficacy signals align with earlier trial results. The drug sponsor uses EAP data to support an FDA breakthrough therapy designation or accelerated approval, shortening the path to market.

EAP Terminated Early After Safety Signal or Trial Failure

Unlikely Resolves by Q3 2028

Discussed by: Clinical research analysts

Slow enrollment, a safety signal, or a failed parallel Phase III trial leads the sponsor to halt the protocol. The program closes with limited data and patients lose access.

Positive Signals, But Confirmatory Trial Required

Possible Resolves by Q3 2028

Discussed by: Regulatory policy analysts

EAP data are encouraging but insufficient for approval on their own. The FDA requires a Phase III confirmatory trial before deciding. The drug remains available through the EAP while the trial runs, and approval is pushed years out.

Historical Context

2 moments from history that rhyme with this story — and how they unfolded.

April 2023

Tofersen for SOD1-ALS approval (2023)

The FDA granted accelerated approval to tofersen (Qalsody), the first therapy targeting a genetic cause of ALS. It was studied in patients with SOD1 mutations, a rare ALS subtype, using a biomarker — not clinical outcomes — as the approval endpoint.

Then

SOD1-ALS patients gained immediate access to a targeted therapy while confirmatory trials continued.

Now

Established a template for biomarker-driven, target-specific ALS drug approval.

Why this matters now

NUZ-001 follows the same logic, targeting a molecular defect — TDP-43 aggregation — that appears in nearly all ALS cases, not just a rare genetic subset.

2019-2023

NurOwn expanded access controversy (2019-2023)

BrainStorm Cell Therapeutics' stem-cell therapy NurOwn faced repeated FDA rebuffs over expanded access requests. Patient advocates sued the agency, demanding access to an unproven treatment. The therapy ultimately failed its Phase III trial in 2023.

Then

Patients gained access to an unproven therapy, and the failed trial undercut the approach.

Now

Showed both the intense patient demand for ALS access and the risk of expanded access outpacing evidence.

Why this matters now

NUZ-001's EAP runs in the same regulatory landscape, where patient demand for access collides with the FDA's evidentiary standards.