Clinical Trial for Parkinson's Uses MRI to Guide Implantation of Autologous Neurons

SAN DIEGO, June 20, 2024 – Aspen Neuroscience has announced that it is leveraging the MRI-guided ClearPoint® Navigation System for patients enrolled in the newly initiated ASPIRO Phase 1/2a clinical trial. This trial focuses on transplanting dopaminergic neuron precursor cells (DANPCs) in individuals suffering from Parkinson's Disease (PD). The ASPIRO trial is designed as an open-label study to evaluate the safety and tolerability of ANPD001, an autologous dopaminergic neuron cell therapy, in patients experiencing moderate to severe Parkinson's Disease.

Edward Wirth III, MD, PhD, Chief Medical Officer of Aspen Neuroscience, highlighted the critical need for precision in targeting the brain area affected by the loss of dopaminergic neurons in PD patients. "By the time PD is diagnosed, many patients have lost the majority of their dopaminergic neurons, resulting in a decline in motor and neurological functions," he explained. "To restore these lost cells, precise targeting in the brain is essential. Using the latest intraoperative MRI-guided techniques offered by the ClearPoint system, the new cells are transplanted very accurately, one microliter at a time, to the specific area where they are most needed."

In this procedure, DANPCs are transplanted into a small region of the mid-brain called the putamen, with the help of ClearPoint MRI guidance. This is done using the SmartFlow® Cannula and the Aspen Metered Delivery Syringe (AMDS). The surgical approach for this trial was developed by Paul Larson, MD, FAANS, a leading neurosurgeon in MRI-guided stereotactic neurosurgery, who is also a professor at the University of Arizona College of Medicine – Tucson, and a neurosurgeon at Banner University Medical Center, Tucson.

Jeremy Stigall, Chief Business Officer at ClearPoint Neuro, expressed pride in contributing to this significant study. "We are honored to participate in a groundbreaking study that aims to show the potential of personalized medical approaches in treating neurodegenerative diseases," he said. "The ClearPoint Neuro Navigation System is used in over 80 centers globally for various applications, including investigational gene and cell therapy trials. We are delighted to collaborate with Aspen in supporting the first multi-center trial for autologous neuron replacement therapy for Parkinson's Disease."

The ClearPoint system employs real-time MR images during procedures to provide precise navigational guidance for neurosurgeons, ensuring the targeted anatomical site is reached with submillimetric accuracy. Paired with the SmartFlow® Cannula, which is less than 2 millimeters in diameter, this allows for minimally invasive delivery of therapeutic agents to a patient's brain.

The ASPIRO trial, named the Autologous-derived Study of a Parkinson's Investigational Regenerative Therapy in an Open-label trial, is a Phase 1/2a clinical trial aiming to evaluate the safety, tolerability, and potential efficacy of ANPD001 in patients with moderate to severe Parkinson's Disease. The study includes patients aged 50–70 and excludes those with cognitive impairments or other conditions that could obstruct treatment. Participants are under the care of a movement disorder specialist. The primary endpoint of the study is safety and tolerability, while secondary endpoints include improvements in symptom control periods and motor function, as well as quality of life enhancements based on standard Parkinson's Disease rating scales.

ANPD001 is an investigational therapy focusing on autologous neuronal replacement for PD. Aspen's approach eliminates the need for immunosuppressive drugs, as the therapy uses the patient's own cells. The process starts with a small sample of the patient's skin cells, which are reprogrammed into induced pluripotent stem cells (iPSCs) and then differentiated into DANPCs. These cells are transplanted into the putamen to replace the lost or damaged neurons. Aspen employs proprietary machine learning-based genomics tests to ensure the quality of each patient's cells at every stage of manufacturing.

ClearPoint Neuro, Inc., the company behind the ClearPoint Navigation System, specializes in enabling precise brain and spine navigation for various medical applications, including drug and device delivery. Their system is FDA-cleared and CE-marked, with extensive use in healthcare and research centers worldwide. The company collaborates with leading pharmaceutical and biotech firms, academic centers, and contract research organizations to provide solutions for direct CNS delivery of therapeutics in both preclinical and clinical settings.

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