Juvenile Parkinson's Disease (JPD)
Advancing Breakthrough Gene Therapies for Rare Neurological Disorders
At Pathways Neuro Pharma, we are dedicated to transforming the treatment landscape for Juvenile Parkinson’s Disease (JPD) and other rare pediatric neurological disorders.
JPD is a severe neurodegenerative disease that affects children and adolescents, leading to progressive motor and cognitive impairment. Current treatments rely on dopamine agonists, which offer only symptomatic relief and carry long-term complications such as dyskinesia, neurotoxicity, and treatment resistance.
There is an urgent need for disease-modifying therapies that address the root biological dysfunction.
A Paradigm Shift in Dopamine Regulation & Neuroprotection
Pathways Neuro Pharma is pioneering an AAV-based gene therapy approach that restores dopamine receptor function and enhances neuroprotection—a fundamental departure from conventional agonist therapy.
Unlike agonists, which force receptors to remain overstimulated and eventually desensitized, our therapy modulates receptor density and signaling at a physiological level, reducing the need for chronic dopamine replacement.
Our investigational approach provides:
- A safer alternative to dopamine agonists, avoiding receptor overstimulation and long-term motor complications.
- Reduced treatment burden for pediatric patients, eliminating the need for daily medication regimens.
- Potential disease-modifying benefits, targeting the biological drivers of functional deterioration rather than masking symptoms.
By increasing dopamine receptor DRD1A expression—while modulating key neuromodulatory pathways such as GPR139 and serotonin receptor 5-HT4—our approach aims to enhance synaptic communication and neuroprotection, potentially stabilizing or reversing disease progression.
Understanding Our Approach: Dopamine Receptor Upregulation
The Challenge in JP:
Impaired dopamine signaling leads to motor dysfunction (rigidity, tremors) and non-motor symptoms (sleep disruption, anxiety, behavioral disorders).
Existing treatments increase dopamine artificially, which over time leads to severe outcomes including dyskinesia, hallucinations, and impulse control disorders—while failing to correct the underlying deficit.
Why Our Method Is Different:
Think of dopamine as the voice and dopamine receptors as the ears that listen.
- In JPD, dopamine is a whisper.
- Agonist therapy turns the volume up until the receptors burn out.
- Our therapy turns up the sensitivity of the receptors—allowing the brain to “hear” better using physiological signaling.
The Science Behind the Solution:
Our investigational gene therapy uses an adeno-associated viral (AAV) delivery system to introduce genetic instructions that upregulate DRD1 dopamine receptors in targeted populations of neurons.
This strategy:
- Avoids agonist-induced overstimulation
- Provides sustained benefit from a single administration
- Addresses both motor and non-motor domains of the disease