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Intraspinal Cell Transplantation for Targeting Cervical Ventral Horn in Amyotrophic Lateral Sclerosis and Traumatic Spinal Cord Injury

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Akt Activation With IPL344 Treatment for Amyotrophic Lateral Sclerosis: First in Human, Open-Label Study.

Marc Gotkine¹, David A Schoenfeld², Ilana Cohen³

¹Neuromuscular Unit, Department of Neurology, Hadassah Medical Organization and Faculty of Medicine, Hebrew University of Jerusalem, Jerusalem, Israel.

|March 19, 2025

Summary

This study found the Akt pathway activator IPL344 to be safe and well-tolerated in patients with amyotrophic lateral sclerosis (ALS). Preliminary results suggest IPL344 may slow ALS progression and improve survival, warranting further investigation.

Keywords:

Akt IPL344 amyotrophic lateral sclerosis clinical trial

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Area of Science:

Neuroscience
Clinical Trials
Pharmacology

Background:

Dysfunction in the Akt intracellular signal transduction pathway is implicated in amyotrophic lateral sclerosis (ALS).
Targeting the Akt pathway presents a novel therapeutic strategy for ALS intervention.

Purpose of the Study:

To evaluate the safety, tolerability, and preliminary efficacy of IPL344, an Akt pathway activator, in a first-in-human study for ALS patients.

Main Methods:

Nine participants with ALS received open-label IPL344 treatment daily for up to 36 months.
Safety was monitored via adverse event reporting; plasma neurofilament light chain (NfL) levels and clinical outcomes (ALSFRS-R) were assessed.
Outcomes were compared to historical control data.

Main Results:

IPL344 was generally safe and well-tolerated, with manageable adverse events.
A 48% slower rate of decline in ALSFRS-R was observed compared to historical controls (p=0.028), with adjusted analysis showing a 64% slower progression.
Plasma NfL concentrations decreased by 27%, and median survival was notably longer in the IPL344 group (43.4 months vs. 19.1 months).

Conclusions:

Preliminary findings suggest IPL344 is safe, well-tolerated, and potentially effective in slowing ALS progression.
The observed improvements in clinical outcomes and survival warrant further investigation in larger, placebo-controlled trials.