Aspen Neuroscience has released six-month interim results from its ongoing Phase 1/2a ASPIRO clinical trial evaluating ANPD001 stem cell therapy in Parkinson’s disease.
This is a stem cell therapy solution for treating patients with moderate to advanced Parkinson’s disease. The interim findings mark a significant advancement in the development of regenerative treatments aimed at replacing lost dopamine-producing neurons, which is a central factor and hallmark in the progression of Parkinson’s disease.
Feel welcome to share your own thoughts on this development in clinical research in the comment section below as well. I will be happy to discuss and learn more about how you see the potential of this solution.
You can view the official news release from Aspen Biosciences here.
Overview of ANPD001 and the ASPIRO Trial
ANPD001 is an investigational stem cell therapy utilizing autologous induced pluripotent stem cells (iPSCs) to potentially treat Parkinson’s disease.
In this approach, a patient’s own skin cells are reprogrammed into iPSCs, which are then differentiated into dopaminergic neuronal precursor cells (DANPCs). These cells are designed to mature into dopamine-producing neurons once implanted into the brain, specifically targeting areas affected by Parkinson’s disease.
A key advantage of this autologous method is the elimination of the need for immunosuppressive drugs, as the cells are derived from the patient’s own tissue, reducing the risk of immune rejection.
The ASPIRO trial (NCT06344026) is an open-label, multicenter study initiated in 2024, aiming to assess the safety, tolerability, and preliminary efficacy of ANPD001 in up to nine participants aged 50–70 years with moderate to severe Parkinson’s disease. Eligible participants are those who respond to levodopa but continue to experience motor fluctuations and “off” periods, where symptoms are not adequately controlled by medication. Patients with cognitive impairments or other conditions that increase surgical risk are excluded from the study. The trial’s primary endpoints focus on safety and tolerability, while secondary endpoints evaluate improvements in motor function, quality of life, and “on” time periods when symptoms are well-managed without troublesome side effects.
Six-Month Interim Results
The interim analysis shows data from the first three patients who received a low-dose implantation of five million DANPCs per putamen. These cells were delivered using an MRI-guided surgical procedure, which includes the use of ClearPoint® Navigation System to ensure precise placement in the posterior putamen, a brain region critically involved in motor control.
- Safety outcomes
- No serious adverse events, such as hemorrhages or significant surgical complications, were reported.
- Minor procedure-related adverse events included tongue swelling, back pain, and incision site discomfort, all of which were transient and resolved without long-term effects.
- Efficacy outcomes
- Patients exhibited early improvements in both clinician-assessed and self-reported measures of motor function and daily living activities.
- Notably, there was an average 45% improvement in the MDS-Unified Parkinson’s Disease Rating Scale Part III “off” scores, indicating enhanced motor function during periods when medication effects are minimal.
These preliminary findings suggest that ANPD001 is safe and may provide functional benefits, warranting further investigation in larger cohorts and over extended follow-up periods.
Broader Context and Future Directions
Aspen Neuroscience are on track to become the first company with a stem cell therapy solution for Parkinson’s disease, which may avoid the necessity for immunosuppression. This is significant because other stem cell therapies in development for various diseases requires immunosuppression. The prospect of this treatment is to eliminate Parkinson’s disease by addressing the loss of neurons caused by the disease.
The ASPIRO trial is notable for being the first multi-patient, multi-center clinical study of a stem cell therapy for Parkinson’s disease in the United States. The use of intraoperative MRI guidance enhances the precision of cell delivery, and may potentially improve therapeutic outcomes.
While these early results are promising, it is important to recognize that they are based on a small sample size and a relatively short follow-up period. Long-term data will be critical to fully understand the safety, durability, and efficacy of ANPD001. The trial is scheduled to conclude by 2030, which includes an extended follow-up period of five years, and with plans for an additional safety monitoring extending to 15 years post-treatment.
If subsequent results continue to demonstrate safety and therapeutic benefit ANPD001 may represent a significant advancement in the treatment of Parkinson’s disease and potentially cure the disease altogether.
