Publication on ANPD001 Autologous Neuronal Cell Replacement in Journal of Neurosurgery

The Journal of Neurosurgery recently published a study conducted by the Wisconsin National Primate Research Center at the University of Wisconsin, Madison. This study focuses on a novel cell transplantation method for delivering ANPD001, an autologous dopaminergic neuronal cell replacement. Aspen Neuroscience is investigating ANPD001 as a potential treatment for Parkinson's Disease in their ongoing Phase 1/2a clinical trial, ASPIRO.

The research demonstrated that the cell transplantation approach for ANPD001 is both safe and feasible in non-human primates. This crucial finding has allowed Aspen Neuroscience to advance with their clinical trial in humans suffering from moderate to severe Parkinson's disease. The study's incorporation into Aspen's Investigational New Drug (IND) application received approval from the U.S. Food & Drug Administration (FDA), permitting the company to commence human trials.

Leading the study was Marina Emborg, M.D., Ph.D., a professor in the Department of Medical Physics at the University of Wisconsin. Dr. Emborg and her team previously published research in 2021 showing that autologous neuronal replacement therapy could reverse Parkinson’s-like symptoms in primates. "The results established that this approach can safely target neuronal replacement therapy with precision to the brain, supporting its clinical investigation for neurodegenerative diseases," Dr. Emborg stated.

Andrés Bratt-Leal, Ph.D., a co-author of the study and senior vice president of research and development at Aspen Neuroscience, emphasized the importance of this research in progressing towards a viable cell-replacement therapy for individuals with Parkinson’s disease. "The results were instrumental in opening our first-in-human trial and informing how we deliver patients' own cells," he noted.

ANPD001 treatment begins with a small sample of the patient's skin cells, which are reprogrammed into induced pluripotent stem cells (iPSCs). These iPSCs, capable of becoming any cell type, are then differentiated into dopaminergic neuron precursor cells (DANPCs). These precursor cells are transplanted into the putamen region of the brain using real-time MRI guidance, aiming to replace cells lost or damaged due to Parkinson's disease.

Edward Wirth III, M.D., Ph.D., Aspen’s chief medical officer and co-author of the study, explained the necessity of targeting specific brain areas with high surgical precision. "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. Utilizing the latest intraoperative MRI-guided techniques, the patient's new cells are transplanted, a few microliters at a time, to the exact area where they are most needed," he elaborated.

Parkinson's Disease (PD) affects more than one million Americans, causing walking and motor problems, as well as impaired balance and coordination. Current therapies alleviate symptoms but do not address the underlying disease, highlighting an urgent need for more effective treatments.

The ASPIRO trial is designed to assess the safety, tolerability, and potential efficacy of ANPD001 in patients with moderate to severe Parkinson's disease. The primary endpoint of the study is to evaluate the safety and tolerability of ANPD001. Secondary endpoints include improvements in "on" time—periods when patients experience symptom control—and enhancements in motor symptoms and quality of life based on standard Parkinson’s disease rating scales.

Aspen Neuroscience, headquartered in San Diego, focuses on autologous regenerative medicine. Their patient-derived iPSC platform aims to create personalized therapies, beginning with neuron replacement for Parkinson’s disease. The company merges cell biology with cutting-edge machine learning and genomic techniques to develop patient-specific restorative cell treatments. Aspen Neuroscience is committed to quality, as seen in their advanced platform for creating and optimizing pluripotent-derived cell therapies, which includes in-house bioinformatics, manufacturing, and quality control.