Gene Therapy for Parkinson's Disease

Revolutionary gene therapy treatment targeting the root cause of Parkinson's. This innovative approach delivers therapeutic genes directly to brain cells to restore dopamine production and improve motor function.

Trial Overview

This Phase 2 clinical trial evaluates a novel gene therapy approach for Parkinson's disease. The treatment uses a viral vector to deliver genes that restore dopamine production in the brain.

Unlike traditional treatments that only manage symptoms, this gene therapy aims to address the underlying cause by restoring the brain's ability to produce dopamine naturally. This could potentially halt disease progression and improve quality of life.

Key Benefits

  • • Targets root cause of Parkinson's
  • • One-time treatment
  • • Potential to halt progression
  • • Restores natural dopamine production

Trial Details

Phase:Phase 2
Status:Recruiting
Duration:36 months
Compensation:Available

Eligibility

  • • Adults 40-75 with Parkinson's
  • • Diagnosed within last 5 years
  • • Responsive to levodopa
  • • No significant cognitive decline

How Gene Therapy Works

1

Gene Delivery

Therapeutic genes are packaged in a safe viral vector for delivery

2

Brain Targeting

Vector delivers genes to specific brain regions affected by Parkinson's

3

Gene Expression

Delivered genes begin producing therapeutic proteins in brain cells

4

Dopamine Restoration

Brain cells restore dopamine production, improving motor function

Treatment Process

Surgical Procedure

  • • Minimally invasive brain surgery
  • • Stereotactic guidance for precision
  • • Local anesthesia with sedation
  • • 2-3 hour procedure duration

Recovery

  • • 1-2 night hospital stay
  • • Gradual return to normal activities
  • • Follow-up appointments
  • • Long-term monitoring

Pre-Treatment

  • • Comprehensive medical evaluation
  • • Brain imaging (MRI, PET)
  • • Neurological assessments
  • • Blood work and testing

Post-Treatment

  • • Regular neurological exams
  • • Motor function assessments
  • • Quality of life measures
  • • Safety monitoring

Expected Outcomes

Motor Function

  • • Improved walking ability
  • • Reduced tremors
  • • Better balance and coordination
  • • Increased movement speed

Daily Activities

  • • Easier dressing and grooming
  • • Improved eating and drinking
  • • Better handwriting
  • • Enhanced mobility

Quality of Life

  • • Reduced medication needs
  • • Better sleep quality
  • • Improved mood
  • • Increased independence

Safety & Risk Assessment

Potential Risks

  • • Surgical complications
  • • Brain bleeding or infection
  • • Immune system reactions
  • • Unintended gene effects
  • • Neurological side effects

Safety Measures

  • • Extensive preclinical testing
  • • Precise surgical techniques
  • • Comprehensive monitoring
  • • Emergency response protocols
  • • Long-term safety tracking

Long-term Benefits

Disease Modification

Unlike symptomatic treatments, gene therapy has the potential to:

  • • Halt disease progression
  • • Restore lost brain function
  • • Reduce future medication needs
  • • Improve long-term prognosis

Sustained Effects

Gene therapy effects are designed to be long-lasting, potentially providing benefits for years after a single treatment.

Future Potential

Success in this trial could lead to expanded use and combination with other emerging Parkinson's treatments.

Ready to Transform Your Parkinson's Treatment?

This revolutionary gene therapy could be the breakthrough you've been waiting for. Join the future of Parkinson's treatment and potentially halt disease progression.