GlobalData : Cell therapies show promise for addressing Parkinson’s disease non-motor symptoms, says GlobalData

Within the current treatments of Parkinson's disease (PD) there is an unmet need for addressing the loss of dopaminergic neurons and non-motor symptoms. At the AD/PD 2024 International Conference on Alzheimer's and Parkinson's Diseases, BlueRock Therapeutics (a subsidiary of Bayer) presented positive results from their Phase I clinical trial of the gene-modified cell therapy Bemdaneprocel (NTC04802733). The trial data points to the early promise of cell therapies addressing the motor and non-motor symptoms of PD, according to GlobalData, a leading data and analytics company.

Lorraine Palmer, Pharma Analyst at GlobalData, comments: "Bemdaneprocel, BlueRock Therapeutics' pipeline cell therapy which is expected to begin Phase II enrolment this year, involves surgically transplanting dopaminergic neuronal progenitor stem cells. With the results showing that these cells survive 18-months post transplantation and 6-months post the cessation of immunosuppression, Bemdaneprocel shows early potential of addressing the loss of dopaminergic neurons by replacing them."

Bemdaneprocel was well tolerated across the cohorts with only one severe adverse event reported; a seizure event believed to have been due to the transplantation surgery rather than the cell therapy itself. In addition, there were no reported cases of graft-induced dyskinesia, an adverse event which has previously occurred in PD cell transplantation trials.

While the trial was not powered to measure statistical significance against a control group, a reduction of motor-symptoms as measured by the Movement Disorder Society-Sponsored Revision of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS) and an adjusted Hauser Diary was witnessed, particularly in the higher dose cohort. Among other positive results, the higher dose cohort also showed trends towards improvements in their neuropsychological evaluations, suggesting positive outcomes in neuropsychiatric symptoms, cognitive functioning, and frontal-lobe related behaviours.

According to GlobalData Drug Database, globally, there are currently two other neuronal progenitor stem cell therapies which have completed Phase I or above trial in PD and disclosed results; Cyto Therapeutics' ISC-hpNSC and Celavie Biosciences' HSCfPD.

Like the Bemdaneprocel trial, Cyto Therapeutics reported an improvement in non-motor symptoms in their Phase I/IIa ISC-hpNSC trial (NCT02452723), while improvements in non-motor symptoms in Phase I trial with Celavie Biosciences' HSCfPD (NCT02780895) were noted as minor.

There are five other neuronal progenitor stem cell therapies in Phase I and Phase II trial for PD, all currently have no published results and only two have disclosed estimated publication dates; Novo Nodisk AS' NN-9001 (2025) and S.Biomedics' TED-A9 (2026).

Palmer continues: "All three neuronal progenitor stem cell therapies with disclosed data in PD saw improvements in core motor PD symptoms. However, that two of them have led to improvements in neuropsychological evaluations is particularly noteworthy as addressing non-motor symptoms in PD is an unmet need in the current market."

Speaking to GlobalData, KOLs state that they are unimpressed with current treatment availability for PD mild cognitive impairment and dementia.

Palmer concludes: "It is early days for dopaminergic neuronal progenitor stem cell therapies, however, if they can address unmet needs like the loss of dopaminergic neurons through replacement, arrest disease progression, and improve not only motor symptoms but non-motor symptoms, they have the potential to become market changers. The results from the Bemdaneprocel trial strengthens these hopes and shows that further investigation is warranted."