Trial of fast-tracked new Parkinson’s cell therapy hits ‘critical’ step
2 groups get commercial 'thaw-and-inject' formulation of sasineprocel
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- A total of 15 adults have now been dosed with sasineprocel, a cell therapy for Parkinson's that replaces lost neurons using a patient's own cells.
- The clinical trial is testing a new "thaw-and-inject" formulation that's commercial-ready, according to its developer.
- Aspen Therapeutics called this new milestone a "critical step" toward scalable manufacturing of the therapy.
Aspen Neuroscience has completed dosing in the third and fourth groups of people with Parkinson’s disease receiving its new commercial-ready formulation of the cell therapy sasineprocel (ANPD001) in a small U.S. clinical trial.
This brings the total number of adults with Parkinson’s who received sasineprocel in the ASPIRO trial (NCT06344026) to 15. The Phase 1/2a study, conducted at nine sites across the country, is testing the safety and tolerability of the autologous cell therapy — one that uses a patient’s own cells — for up to five years.
The treatment’s commercial formulation uses what its developer calls cryopreserved “thaw-and-inject” cells that can be administered upon arrival at the clinical site. It was developed to facilitate therapy production and clinical delivery.
“We are proud to reach this important milestone, which reflects meaningful progress in the sasineprocel program and underscores the growing clinical experience with our personalized, autologous approach,” Damien McDevitt, PhD, Aspen’s president and CEO, said in a company press release. “Importantly, the use of our commercial formulation in these [patient groups] represents a critical step toward scalable manufacturing and commercial readiness as we prepare for Phase 3 initiation.”
Sasineprocel is an autologous cell therapy, meaning an individual’s own cells are the basis for the treatment, that aims to replace dopaminergic neurons, the nerve cells lost in Parkinson’s disease. These neurons produce dopamine, a brain signaling molecule, or neurotransmitter, involved in motor control. Because cells are derived from the patient, immunosuppressive medications are not needed, which is expected to reduce infections and other treatment-related side effects that can come with donor-based cell therapies.
The treatment’s production starts by collecting a patient’s skin cells and reprogramming them in the lab into induced pluripotent stem cells, which are then helped to develop into dopaminergic neuronal precursors. These cells are then delivered via MRI-guided injection into the putamen, a Parkinson’s-related brain region, where they can potentially develop into functional dopaminergic neurons.
Formulation being tested is manufactured and stored in advance
The ASPIRO trial is administering sasineprocel to Parkinson’s patients with moderate to severe disease who respond to levodopa therapy. That’s the gold standard treatment for easing the disease’s motor symptoms, including slowed movements, muscle stiffness, and tremors.
In the first two groups of patients, in which eight participants received a low or high treatment dose, the cell therapy was generally safe and well tolerated for up to one year. Treatment also led to improvements in daily functioning and patients’ quality of life, and was seen to ease motor symptoms.
Specifically, participants experienced an increase in good on time, when symptoms are well controlled with medications, by as much as 2.4 hours per day. Patients also experienced reductions in the MDS-Unified Parkinson’s Disease Rating Scale parts 2 and 3 scores, which evaluate experiences of daily living and motor symptoms, respectively.
Imaging studies demonstrated that the delivered cells survived and integrated into the surrounding tissue. Some patients were able to reduce their levodopa equivalent daily dose, which is a standardized way of comparing different levodopa formulations.
Compared with the formulation of sasineprocel used in the first two groups of patients, who received a freshly prepared cell product soon after preparation, the thaw-and-inject formulation is manufactured centrally and stored. According to the company, this represents an important step toward Phase 3 clinical testing and eventual commercialization.
Sasineprocel has received fast-track designation from the U.S. Food and Drug Administration, which aims to accelerate the clinical development and review of treatments with the potential to fill unmet medical needs.
Aspen also noted that testing of the treatment is supported by a 2024 grant from the California Institute for Regenerative Medicine, a state agency that funds stem cell and gene therapy research.
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