Pridopidine Therapy for ALS With Axonal Transport Restoration

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Solution Overview

Problem

Current treatments for amyotrophic lateral sclerosis (ALS) provide only modest effects on disease progression and survival, and there is a need for more effective therapies to manage symptoms and slow disease progression.

Innovation Solution

Administering pridopidine or its pharmaceutically acceptable salts to ALS patients to improve axonal transport, enhance ERK activation, restore neuromuscular junction activity, and reduce mutant SOD1 aggregates, thereby maintaining or improving muscle function and respiratory function.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Reliability

If current approved drugs (riluzole, edaravone, AMX0035) are used for treating ALS, then disease progression and survival are modestly improved, but treatment effectiveness remains insufficient

Engineering Contradiction:

Improvetreatment effectivenessVSAvoiddisease progression rate

Core Design Contradiction:

ReliabilityVSProductivity

Solution Approach 1:

The patent changes the therapeutic parameter by introducing pridopidine, a novel compound with different mechanism of action (S1R activation) compared to existing drugs. This parameter change enables improved axonal transport and reduced mutant SOD1 aggregates, directly addressing the insufficiency of current treatments while maintaining acceptable safety profiles

Inventive Principle:

Principle #35Parameter changes

2Reliability

If pridopidine is administered to ALS patients, then axonal transport is improved and neuromuscular junction activity is restored, but the complexity of the treatment mechanism increases

Engineering Contradiction:

Improveaxonal transport functionVSAvoidtreatment mechanism complexity

Core Design Contradiction:

ReliabilityVSDevice complexity

Solution Approach 1:

Pridopidine acts as an intermediary compound that mediates its therapeutic effects through activation of the S1R receptor. This single intermediary molecule addresses multiple pathological features including axonal transport deficits, mutant SOD1 aggregate formation, and neuromuscular junction dysfunction, simplifying the overall treatment approach despite the multifaceted mechanism

Inventive Principle:

Principle #24Intermediary (Mediator)

3Reliability

If pridopidine treatment is applied to early-stage or fast-progressing ALS patients, then muscle strength and respiratory function are better maintained, but the risk of inadequate treatment coverage for other patient subgroups increases

Engineering Contradiction:

Improvemuscle function maintenanceVSAvoidtreatment adaptability to different patient subgroups

Core Design Contradiction:

ReliabilityVSAdaptability or versatility

Solution Approach 1:

The patent applies pridopidine treatment with emphasis on early-stage and fast-progressing patients who would benefit most from the neuroprotective effects. This partial action strategy focuses therapeutic resources on the subgroup with greatest potential benefit, accepting that other subgroups may receive less optimal coverage while still providing modest benefit through the same mechanism

Inventive Principle:

Principle #16Partial or excessive action

Data Source

PatentUS20260034109A1Method of treating amyotrophic lateral sclerosis with pridopidine

Publication Date: 2026.02.05 PRILENIA NEUROTHERAPEUTICS LTD

AI summary

Provided herein is a method for treating a human subject afflicted with ALS by administering to the subject a therapeutically effective amount of pridopidine or pharmaceutically acceptable salt thereof.