Aspen Neuroscience’s autologous, personalised stem cell therapy is set for a pivotal trial after it showed improved “ON” and “OFF” time in Parkinson’s disease patients.
Data from the first eight patients in the Phase Ia/II ASPIRO trial (NCT06344026), sasineprocel, an autologous induced-pluripotent stem cell (iPSC)-derived dopaminergic neuron precursor cell therapy (DANPC), was presented at the AD/PD 2026 International Conference on Alzheimer’s and Parkinson’s diseases, taking place between 17 and 21 March in Copenhagen, Denmark.
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Of the eight patients dosed, half received a low dose and half received a higher dose of the therapy. After 12 months, both dosing cohorts showed numerical improvements in function, physician- and patient-reported outcomes, and quality-of-life (QoL).
There was a mean Good “ON” time of 2.1 hours in low dose and 2.4 hours in high dose. The mean MDS-UPDRS III OFF scores reduced by 15.5 points in the low dose cohort, and 13.5 points in the high dose. The “OFF” time refers to a period when the effects of medication wear off, and symptoms return or worsen.
The mean MDS-UPDRS II scores reduced by 5.3 points at low dose, and 2.3 points at high dose, and the mean improvement in PDQ-39 scores was 51.6% and 28.5% in the low and high dose cohorts, respectively.
There were no serious surgical adverse events (AEs), and no cases of severe graft-induced dyskinesia were observed. No symptomatic haemorrhages or infarctions were observed.
Aspen’s therapy involves converting a patient’s own skin cells into stem cells, which are then converted into implantable dopamine-producing cells to replace neurons.
Aspen CMO Dr Revati Shreeniwas said: “We’re very encouraged by these data showing improvements in function, physician- and patient-reported outcomes, and quality-of-life, and look forward to advancing sasineprocel to a Phase III study later this year.
“These findings support our approach of reprogramming a patient’s own cells to wind back the epigenetic clock to a pre‑disease state. Combined with precisely delivering our multi-cell composition to the putamen, we’re starting to see the positive clinical impact of rebuilding the neural networks damaged by this debilitating disease.”
Aspen is not the only company looking to advance a cell therapy in Parkinson’s disease.
BlueRock Therapeutics, which is owned by Bayer, is also investigating a cell therapy, bemdaneprocel, in Parkinson’s. However, its therapy is an off-the-shelf product using donor cells. While this means it is easier to manufacture en masse, it requires patients to go through immunosuppressive treatments. The therapy is now in a Phase III trial.
Hope Biosciences Research Foundation (HBRF) is also set to advance its Parkinson’s disease cell therapy into Phase III trials after it showed benefit in a Phase II study.
Meanwhile, Kenai recently dosed its first patient in a Phase I trial evaluating its cellular neuron replacement therapy, RNDP-001, in moderate-to-severe idiopathic Parkinson’s disease. Initial data related to the tolerability, safety and brain imaging impacts are expected this year.
In a previous interview with Clinical Trials Arena, David Dexter, head of research at nonprofit Parkinson’s UK, said that although researchers and drugmakers are finally starting to make headway in better characterising therapies for Parkinson’s, there are still multiple factors to be addressed to facilitate continued progress in the space.
GlobalData, parent company of Clinical Trials Arena, currently forecasts that the Parkinson’s disease market will be worth $7.9bn across the seven major markets in 2033.
Cell & Gene therapy coverage on Clinical Trials Arena is supported by Cytiva.
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