Parkinson's Disease Clinical Trial Uses Intraoperative MRI to Guide Precision Implantation of Patients' Own Autologous Replacement Neurons

Aspen Neuroscience Announces MRI-Guided Transplantation Approach for ASPIRO Clinical Trial with the ClearPoint® Navigation System

SAN DIEGO, June 20, 2024 /PRNewswire/ -- Aspen Neuroscience, Inc. announced today that it is utilizing the MRI-guided ClearPoint® Navigation System for all patients enrolled in the recently launched ASPIRO Phase 1/2a clinical trial for transplantation of dopaminergic neuron precursor cells (DANPCs) in patients with Parkinson's Disease (PD). ASPIRO is an open label trial to assess safety and tolerability of ANPD001, an autologous, dopaminergic neuron cell replacement therapy for participants with moderate to severe PD.

"By the time of diagnosis, it is common for people with Parkinson's to have lost the majority of dopaminergic neurons, leading to progressive loss of motor and neurological function," explained Edward Wirth III, MD, PhD, Chief Medical Officer of Aspen Neuroscience. "To replace these lost cells, we must target a very specific area of the brain with a high degree of surgical precision. Utilizing the latest advances in intraoperative MRI guided techniques provided by the ClearPoint system, the patient's new cells are transplanted, one microliter at a time, to the exact area where they are most needed."

These DANPCs are transplanted to the putamen, a small section located in the mid-brain, in a single transplantation procedure under ClearPoint MRI guidance, using the SmartFlow® Cannula and the Aspen Metered Delivery Syringe (AMDS). This surgical approach was developed by the trial's lead neurosurgeon and renowned MRI-guided stereotactic neurosurgery pioneer Paul Larson, MD, FAANS, professor of neurosurgery at the University of Arizona College of Medicine – Tucson and neurosurgeon at Banner University Medical Center, Tucson.

"We are honored to be a part of such an important and groundbreaking study to demonstrate the ability of personalized medical approaches for treating neurodegenerative conditions," explained Jeremy Stigall, Chief Business Officer at ClearPoint Neuro. "The ClearPoint Neuro Navigation System is being used successfully in more than 80 centers worldwide for multiple applications, and investigational gene and cell therapy trials. We are thrilled to partner with Aspen to support the first multi-center trial for an autologous neuron replacement therapy for Parkinson's disease."

The ClearPoint System utilizes intraprocedural MR images to provide real-time navigational instruction for the neurosurgeon, and confirmation that the desired anatomical target has been reached with submillimetric accuracy. Combined with The SmartFlow® Cannula, which is less than 2 millimeters in diameter, this allows minimally-invasive delivery of therapeutic agents in a patient's brain.

About the ASPIRO Trial
The Autologous-derived Study of a Parkinson's Investigational Regenerative therapy in an Open-label trial (ASPIRO) is a Phase 1/2a clinical trial to assess the safety, tolerability, and potential efficacy of ANPD001 in patients with moderate to severe Parkinson's disease. The dose escalation study includes patients 50–70 years of age and excludes patients with cognitive impairment and other comorbidities that could preclude treatment. All enrolled patients are under the care of a movement disorder specialist.

The primary study endpoint is safety and tolerability of ANPD001. Secondary endpoints include improvement in "on" time, when patients experience periods of symptom control, and improvements in motor symptoms and quality of life based on standard Parkinson's disease rating scales.

About ANPD001
ANPD001 is an investigational autologous neuronal replacement therapy being studied as a regenerative therapy for PD. Aspen's personalized approach means that patients do not require immunosuppressive drugs to counteract the body's immune response against foreign cells.

Aspen's manufacturing process starts from a small sample of the patient's own skin cells, followed by reprogramming to induced pluripotent stem cells (iPSCs) and then differentiation of the iPSCs into DANPCs. These DANPCs are transplanted into the putamen, replacing cells that were lost or damaged due to disease. The quality of each person's cells is assessed at every manufacturing stage using Aspen's proprietary machine learning-based genomics tests.  

About ClearPoint Neuro
ClearPoint Neuro, Inc. (NASDAQ: CLPT) is a device, cell, and gene therapy-enabling company offering precise navigation to the brain and spine. The Company uniquely provides both established clinical products as well as pre-clinical development services for controlled drug and device delivery. The Company's flagship product, the ClearPoint Neuro Navigation System, has FDA clearance and is CE-marked. ClearPoint Neuro is engaged with healthcare and research centers in North America, Europe, Asia, and South America. The Company is also partnered with the most innovative pharmaceutical / biotech companies, academic centers, and contract research organizations, providing solutions for direct CNS delivery of therapeutics in pre-clinical studies and clinical trials worldwide. To date, thousands of procedures have been performed and supported by the Company's field-based clinical specialist team, which offers support and services to our customers and partners worldwide. For more information, please visit www.clearpointneuro.com.

About Aspen Neuroscience 
Headquartered in San Diego, Aspen Neuroscience, Inc. is a clinical development-stage, private company focused on autologous regenerative medicine. The company's patient-derived iPSC platform is used to create personalized therapies to address diseases with high unmet medical needs, beginning with autologous neuron replacement for Parkinson's disease. 

Aspen combines cell biology with the latest machine learning and genomic approaches to investigate patient-specific, restorative cell treatments. The company has developed a best-in-class platform to create and optimize pluripotent-derived cell therapies, which includes in-house bioinformatics, manufacturing and quality control. For more information and important updates, please visit https://www.aspenneuroscience.com.

SOURCE Aspen Neuroscience, Inc.