# An Expanded Access Protocol of  Intravenous Trehalose Injection 90 mg/mL Treatment of Patients with Amyotrophic Lateral Sclerosis

> **NIH NIH UF1** · MASSACHUSETTS GENERAL HOSPITAL · 2022 · $18,136,504

## Abstract

Amyotrophic lateral sclerosis (ALS) is a rapidly progressive neurodegenerative. There are two
FDA-approved medications to slow ALS progression, riluzole and edaravone; their effect is
modest, but additive given that they target different biological pathways. Additional
pathophysiologic pathways can be targeted to provide even more additive effect. Autophagy is
dysregulated in ALS and is a promising target for novel therapeutic development.
Trehalose (SLS-005, Seelos Therapeutics) is a disaccharide that is well known for its ability
to activate autophagy. Three in vivo studies demonstrated a protective effect in SOD1 mouse
models (G93T and G86R). In humans, trehalase breaks down trehalose in the gut, so it must be
delivered intravenously (IV) to preserve its effect.
The safety and efficacy of trehalose are currently being tested in the HEALEY ALS Platform
Trial. The trial design includes an efficacy randomized controlled trial (RCT) followed by an open
label extension (OLE). In the trial, participants undergo weekly IV infusions of trehalose, which
are done either at the center or at home by a trained infusion nurse. Unfortunately, the trehalose
OLE will end for most participants before the results of the RCT are known due to financial
constraints as Seelos is a small business. For the same reason, expanded access is not currently
available to people who are not eligible for the RCT.
The current proposal is an expanded access protocol (EAP) of trehalose that will include
both people who are not eligible for clinical trials (Cohort 1) as well as people who completed their
participation in the trehalose OLE of the HEALEY ALS Platform Trial and are no longer eligible
for participation in other trials (Cohort 2). The latter group will be exposed for an additional six
months. Outcome measures for this EAP will include safety, the biofluid biomarker neurofilament
light (NFL), clinical measures of disease progression, and survival. This study will provide real-
world data to supplement the trehalose clinical development program by evaluating the
effects of the drug in a population that is broader than the one included in the RCT and by
collecting outcomes over longer term exposure. Data will be collected in format that can be
submitted to FDA and could therefore be included in a potential NDA submission.

## Key facts

- **NIH application ID:** 10649756
- **Project number:** 1UF1NS131791-01
- **Recipient organization:** MASSACHUSETTS GENERAL HOSPITAL
- **Principal Investigator:** Suma Babu
- **Activity code:** UF1 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2022
- **Award amount:** $18,136,504
- **Award type:** 1
- **Project period:** 2022-09-28 → 2026-08-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10649756

## Citation

> US National Institutes of Health, RePORTER application 10649756, An Expanded Access Protocol of  Intravenous Trehalose Injection 90 mg/mL Treatment of Patients with Amyotrophic Lateral Sclerosis (1UF1NS131791-01). Retrieved via AI Analytics 2026-05-27 from https://api.ai-analytics.org/grant/nih/10649756. Licensed CC0.

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