DNP Dose Window for Endogenous BDNF Induction in Brain Tissue

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Solution Overview

Problem

Existing methods are inadequate for effectively inducing brain-derived neurotrophic factor (BDNF) expression to treat neuromuscular, neurodegenerative, autoimmune, and metabolic diseases, and there is a need for a safe and effective dose range to cross the blood-brain barrier.

Innovation Solution

Administering mitochondrial uncoupler 2,4-dinitrophenol (DNP) in a dose range of 0.01 mg/kg to 1.0 mg/kg body weight to endogenously induce BDNF expression, leveraging its non-genomic and genomic effects to increase BDNF levels in the brain and peripheral tissues.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Quantity of substance

If high doses of DNP are administered to induce BDNF expression, then BDNF levels increase, but harmful side effects occur

Engineering Contradiction:
ImproveBDNF expression levelsVSAvoidharmful side effects
Core Design Contradiction:
Quantity of substanceVSObject-affected harmful factors

Solution Approach 1:

The patent identifies and establishes a specific dose range (0.001 mg/kg to less than 10 mg/kg, preferably 0.01 mg/kg to 1.0 mg/kg) of DNP that optimizes BDNF induction while avoiding harmful effects. This parameter optimization resolves the contradiction by finding the precise dosage window where therapeutic benefit is maximized and toxicity is minimized.

Inventive Principle:
Principle #35Parameter changes

Solution Approach 2:

DNP is traditionally known as a toxic mitochondrial uncoupler, but the patent converts this harmful property into a beneficial therapeutic effect by utilizing its ability to induce BDNF expression. The harmful mitochondrial uncoupling action is harnessed at controlled doses to trigger neuroprotective BDNF production, transforming a toxin into a therapeutic agent.

Inventive Principle:
Principle #22Blessing in disguise (Convert harm into benefit)

2Reliability

If DNP is administered to cross the blood-brain barrier, then BDNF expression is induced in the brain, but safety concerns arise

Engineering Contradiction:
Improveeffectiveness in treating neurological diseasesVSAvoidsafety concerns
Core Design Contradiction:
ReliabilityVSObject-affected harmful factors

Solution Approach 1:

The patent establishes a safe and effective dose range (0.01 mg/kg to 1.0 mg/kg) that enables DNP to cross the blood-brain barrier and induce BDNF expression while maintaining safety. This parameter optimization ensures reliable therapeutic effect without compromising patient safety.

Inventive Principle:
Principle #35Parameter changes

Solution Approach 2:

BDNF acts as an intermediary molecule that mediates the therapeutic effect of DNP in the brain. DNP induces endogenous BDNF production, which then serves as the actual therapeutic agent providing neuroprotection and treating neurological diseases, thereby indirecting the therapeutic action and reducing direct exposure to DNP.

Inventive Principle:
Principle #24Intermediary (Mediator)

3Productivity

If existing methods are used to induce BDNF expression, then treatment of neurological diseases is attempted, but effectiveness is inadequate

Engineering Contradiction:
Improvetreatment effectivenessVSAvoidability to effectively induce BDNF
Core Design Contradiction:
ProductivityVSReliability

Solution Approach 1:

The patent employs DNP with optimized dosage parameters (0.01 mg/kg to 1.0 mg/kg) to effectively induce BDNF expression, achieving superior treatment effectiveness compared to existing methods. This parameter optimization enables reliable and effective BDNF induction for treating neurological diseases.

Inventive Principle:
Principle #35Parameter changes

Applied Scientific Principles

This section explains which scientific principles are used to turn an abstract innovation direction into a practical engineering solution.

Function Achieved in This Case

The specified dose range of DNP effectively increases BDNF levels, providing neuroprotection and remission of symptoms in diseases such as Multiple Sclerosis, Huntington's Disease, and other neurodegenerative disorders, while avoiding harmful side effects.

Implementation Method 1

Administering mitochondrial uncoupler 2,4-dinitrophenol (DNP) in a dose range of 0.01 mg/kg to 1.0 mg/kg body weight to endogenously induce BDNF expression

Methodology Applied
Scientific EffectMitochondrial uncoupling:

Data Source

PatentEP4306167B1Induced expression of brain derived neurotrophic factor (BDNF) for treatment of neuromuscular, neurodegenerative, autoimmune, developmental and/or metabolic diseases
Publication Date: 2025.12.24 MITOCHON PHARMACEUTICALS INC
  • EP4306167B1 patent drawingFigure 1
  • EP4306167B1 patent drawingFigure 2a~2b
  • EP4306167B1 patent drawingFigure 2c~2d

AI summary

A method of treating a host of neuromuscular, neurodegenerative, developmental, autoimmune and metabolic diseases/disorders related to aging, such as traumatic injury, stroke, Huntington's disease, Epilepsy, Multiple Sclerosis (MS), Lupus, Type-1 and Type-2 diabetes, Maturity Onset Diabetes of the Young (MODY), myasthenia gravis (MG), rheumatoid arthritis (RA), Graves' disease, Guillain-Barré syndrome (GBS), metabolic syndrome, Muscular Dystrophy or Duchenne Muscular Dystrophy (DMD), severe burns, aging, Amyotrophic Lateral Sclerosis (ALS), Friedreich's Ataxia, Batten Disease, Alzheimer's disease, optic neuritis, Leber's hereditary optic neuropathy (LHON), autism, Rett syndrome, Batten Disease, Angelman's Syndrome, Leigh disease, Fragile-X Syndrome, depression, Parkinson's disease, mitochondrial diseases, developmental disorders, metabolic disease disorders and/or autoimmune disorders by inducing endogenous BDNF expression with DNP treatment to protect from neuromuscular dysfunction/disorders and/or neurodegeneration and/or muscle wasting. DNP was administered to mice daily over a range of doses, and subsequently BDNF expression in the brain showed a dose dependent and non-linear increase in expression.