Aspen Neuroscience has announced the successful completion of dose escalation and the first two patient cohorts in the ASPIRO study, a Phase 1/2a clinical trial evaluating ANPD001 for Parkinson's Disease (PD). This autologous cell therapy aims to replace damaged dopamine neurons in patients with moderate to severe PD. The trial, launched in 2024, is a multi-center, open-label study designed to assess the safety and tolerability of ANPD001 at escalating doses.
Safety and Tolerability
According to Aspen Neuroscience, ANPD001 and its delivery method have been well tolerated in the initial cohorts. "To date, ANPD001 and its delivery have been well tolerated, and no serious adverse events have been observed in the first two cohorts of patients in the ASPIRO study," said Edward Wirth III, MD, PhD, Chief Medical Officer of Aspen Neuroscience. All patients were discharged within 48 hours post-treatment, adhering to the study protocol.
ANPD001: A Personalized Approach
ANPD001 is an investigational autologous dopaminergic neuronal precursor cell (DANPC) therapy. Aspen's approach involves using a patient's own skin cells, which are then reprogrammed into induced pluripotent stem cells (iPSCs) and differentiated into DANPCs. These cells are then transplanted into the putamen, a region of the brain affected by Parkinson's, to replace lost or damaged cells. This personalized method eliminates the need for immunosuppressive drugs, reducing the risk of immune rejection.
Trial Design and Endpoints
The ASPIRO trial (NCT06344026) includes patients aged 50-70 years. Key exclusion criteria involve cognitive impairment and other comorbidities that could interfere with the treatment. The primary endpoint is the safety and tolerability of ANPD001. Secondary endpoints include improvements in "on" time—periods when patients experience symptom control—as well as enhancements in motor symptoms and overall quality of life, as measured by standard Parkinson's rating scales.
Implications for Parkinson's Treatment
Parkinson's disease is characterized by the progressive loss of dopaminergic neurons, leading to motor symptoms such as tremors, rigidity, and bradykinesia. Current treatments primarily focus on managing symptoms, but ANPD001 represents a potential regenerative approach by replacing the lost dopamine-producing cells. ANPD001 has received Fast Track designation by the U.S. Food & Drug Administration (FDA).
Next Steps
Aspen Neuroscience is now advancing the program to investigate a new commercial formulation of ANPD001, potentially paving the way for scalable production and broader accessibility if the therapy proves effective and safe in further trials.
