Publications by authors named "H Widner"
Immunology of cell and gene therapy approaches for neurologic diseases.
Handb Clin Neurol
September 2024
Repair and replacement strategies using cell replacement or viral gene transfer for neurologic diseases are becoming increasingly efficacious with clinically meaningful benefits in several conditions. An increased understanding of disease processes opens up opportunities for genetic therapies and precision medicine methods aiming at disease modification or repair of lesioned neurologic structures. However, such therapeutic effects may be limited or rendered ineffective by immune responses against gene products or cells used for the intended treatments.
View Article and Find Full Text PDFBackground: New disease-modifying ways to treat Parkinson's disease (PD) may soon become a reality with intracerebral transplantation of cell products produced from human embryonic stem cells (hESCs). The aim of this study was to assess what factors influence preferences of patients with PD regarding stem-cell based therapies to treat PD in the future.
Methods: Patients with PD were invited to complete a web-based discrete choice experiment to assess the importance of the following attributes: (i) type of treatment, (ii) aim of treatment, (iii) available knowledge of the different types of treatments, (iv) effect on symptoms, and (v) risk for severe side effects.
Cell replacement therapies for Parkinson's disease (PD) based on transplantation of pluripotent stem cell-derived dopaminergic neurons are now entering clinical trials. Here, we present quality, safety, and efficacy data supporting the first-in-human STEM-PD phase I/IIa clinical trial along with the trial design. The STEM-PD product was manufactured under GMP and quality tested in vitro and in vivo to meet regulatory requirements.
View Article and Find Full Text PDFArticle Synopsis
- - Multiple system atrophy (MSA) is a severe disease with varying motor and autonomic symptoms, and previous studies have linked certain clinical factors to reduced survival rates.
- - Researchers analyzed 210 MSA patients over 17 years to create a survival risk model using clinical factors like age at symptom onset and early autonomic failure.
- - They developed a nomogram to predict individual survival probabilities over 7 years, which showed good accuracy and could enhance patient counseling and treatment strategies.
Background: Cerebral dopamine neurotrophic factor (CDNF) is an unconventional neurotrophic factor that protects dopamine neurons and improves motor function in animal models of Parkinson's disease (PD).
Objective: The primary objectives of this study were to assess the safety and tolerability of both CDNF and the drug delivery system (DDS) in patients with PD of moderate severity.
Methods: We assessed the safety and tolerability of monthly intraputamenal CDNF infusions in patients with PD using an investigational DDS, a bone-anchored transcutaneous port connected to four catheters.