First-in-Human Trial for Parkinson's Autologous Cell Therapy Begins

In a significant advancement for the treatment of Parkinson's disease, a biotech firm has initiated a pioneering clinical trial. Aspen Neuroscience has commenced the ASPIRO trial, marking the first U.S. multi-center, multi-patient Phase 1/2a study of an autologous neurological therapy. The trial aims to evaluate the safety and tolerability of ANPD001, a novel autologous dopaminergic neuron cell replacement therapy designed for individuals with moderate to severe Parkinson's disease.

The ASPIRO study's inaugural transplantation procedure was conducted by Dr. Paul Larson, a neurosurgeon at Banner – University Medical Center Tucson and a professor at the University of Arizona College of Medicine – Tucson. Dr. Larson, a leader in interventional MRI-guided stereotaxy with over a thousand iMRI procedures under his belt, is spearheading the ASPIRO study at his institution, which is one of five clinical sites participating in the trial.

Parkinson's disease, characterized by the depletion of dopamine neurons in the midbrain, often leads to a progressive loss of motor and neurological functions. The ASPIRO trial is a critical step towards developing personalized, regenerative neurological therapies to address unmet medical needs, with the goal of halting or reversing the loss of dopamine neurons and slowing Parkinson's progression.

The trial is open-label and includes patients aged 50 to 70, excluding those with cognitive impairments or other comorbidities that could interfere with treatment. The primary endpoint focuses on the safety and tolerability of two escalating doses of ANPD001. Secondary endpoints aim to measure improvements in symptom control, motor symptoms, and quality of life using established Parkinson's disease rating scales.

ANPD001 represents an innovative autologous neuronal replacement therapy. Unlike traditional treatments, it does not require patients to take immunosuppressive drugs, as it utilizes the patient's own cells. The therapy's manufacturing process involves reprogramming a small skin cell sample into induced Pluripotent Stem Cells (iPSCs), which are then differentiated into dopaminergic neuron precursors. These precursors are transplanted into the brain to replace lost or damaged cells, with each stage of manufacturing quality-checked using Aspen's proprietary machine learning-based genomics tests.

The University of Arizona College of Medicine – Tucson, established in 1967, is renowned for its innovative medical education, research, and patient care. In collaboration with Banner Health, a leading nonprofit health care system, the college is at the forefront of academic medicine.

Aspen Neuroscience, based in San Diego, is a clinical-stage private company dedicated to autologous regenerative medicine. The company's patient-derived iPSC platform is utilized to develop personalized therapies for diseases with significant unmet needs, starting with Parkinson's disease. Aspen employs a combination of cell biology, machine learning, and genomics to explore patient-specific restorative cell treatments, supported by in-house bioinformatics, manufacturing, and quality control capabilities.