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AD/PD 2024: cell therapies show promise for non-motor symptoms seen with Parkinson’s

Within 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) announced positive results at the 18-month mark of their Phase I clinical trial of the cell therapy bemdaneprocel. Bemdaneprocel not only reduced PD motor symptoms, but also showed trends towards addressing the loss of dopaminergic neurons and PD non-motor symptoms. In doing so, it joins other neuronal stem cell therapies that also leads to improvements in non-motor symptoms and core motor PD symptoms.

In BlueRock Therapeutics’ Phase I bemdaneprocel trial (NCT04802733), dopaminergic neuronal progenitor stem cells were stereotactically transplanted directly into the posterior putamen of 12 participants with idiopathic PD. The participants were divided into a low and high dose cohort, receiving 0.9 million cells/putamen and 2.7 million cells/putamen, respectively. Results show that these cells survived 18 months post transplantation and sixmonths post cessation of immunosuppression, strengthening the hope that Bbemdaneprocel could address the loss of dopaminergic neurons in PD by replacing lost neurons.

The treatment was well tolerated across the cohorts with only one severe adverse event (seizure) reported. This event is believed to have been due to the transplantation surgery rather than the cell therapy. In addition, there were no reported cases of graft-induced dyskinesia, an adverse event that has previously occurred in PD cell transplantation trials (Lane, 2021). Therefore, the trial met overall safety and tolerability outcomes.

While the trial was not powered to measure statistical significance and did not include a control group (with results being compared against baseline measurements), a reduction in 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 behaviors. These included measurements indicating improvements in immediate memory and delayed memory. This is particularly significant as addressing non-motor symptoms in PD is an unmet need in the current market, with key opinion leaders (KOLs) interviewed by GlobalData stating that they are “unimpressed” with current treatment availability for PD, mild cognitive impairment, and dementia. However, further clinical trials are needed before conclusions can be drawn on the efficacy of bemdaneprocel. There are plans for a Phase II trial with bemdaneprocel starting June 2024.

Globally, there are currently two other neuronal progenitor stem cell therapies that have completed trials in PD where results were disclosed; Celavie’s HSCfPD in Phase I, and Cyto Therapeutics’ ISC-hpNSC in Phase I/IIA. Similar to the bemdaneprocel trial, in Celavie’s HSCfPD trial (NCT02780895), eight participants underwent stereotactical transplantation of stem cells into the putamina. However, in this case, one million undifferentiated human neural progenitor stem cells were deposited into the anterior and posterior of both putamina. In Cyto Therapeutics’ Phase I/IIa trial (NCT02452723), cells were also transplanted via stereotactic bilateral injection. However, the ISC-hpNSC cells transplanted comprised parthenogenetic neural stem cells and were injected into the striatum, caudate nucleus, putamen, and substantia nigra at three dose levels (30, 50 and 70 million cells) in four participants.

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As with bemdaneprocel, Cyto Therapeutics reported an improvement not only in motor symptoms but also in non-motor symptoms with ISC-hpNSC. On the other hand, while there were improvements in motor symptoms, improvements in non-motor symptoms with Celavie’s HSCfPD were noted as minor. Although still early stage, the results from the bemdaneprocel and ISC-hpNSC trials are encouraging in terms of the potential of cell therapy to address not only motor symptoms but also non-motor symptoms.

There are currently 55 known cell therapies in various stages of development for Parkinson’s disease, including nine gene-modified cell therapies. The highest stage of development is Phase II, of which there are five neural progenitor stem cells therapies like bemdaneprocel. ISC-hpNSC, two of which have ongoing trials with no end date yet stated, one is thought to be completed and awaiting the publication of results (CHA Biotech Co’s CBT-NPC), and S.Biomedics’ TED-A9 for which a Phase I/IIa trial was reported last month to have completed transplantation with trial completion expected in 2026. Therefore, the next few years will be crucial in determining the full potential of neuronal stem cell therapies for the treatment of PD, including replacing lost dopaminergic neurons and the treatment of non-motor symptoms.