Abstract
Background and Objective
- People with Parkinson’s disease experience reduced health-related quality of life that persists despite standard treatment.1
- Current standard treatments for Parkinson’s disease motor symptoms do not address the loss of dopaminergic neurons.2
- Bemdaneprocel is an investigational therapy comprising dopaminergic neuronal progenitors derived from human embryonic stem cells.3,4
- exPDite is a Phase 1 multicenter, multisite, open-label, nonrandomized study (NCT04802733).
- Objective: To evaluate the safety of bemdaneprocel and its impact on motor outcomes up to 18 months post transplantation, 6 months post discontinuation of immunosuppression, in participants with Parkinson’s disease.
Trial Design and Endpoints
- Participants with Parkinson’s disease with motor symptoms inadequately controlled by standard treatments were >=50 to <=78 years of age in Canada, or >=60 to <=78 in the US.
- A total of 12 participants enrolled sequentiallyc.
- 18F-DOPA PET was performed at baseline, 12, and 18 months post transplantation, and will be performed at 24 months post transplantation.
- MRI was performed at screening, intraoperatively, postoperatively, and 1, 3, 6, 12, and 18 months post transplantation, and will be performed at 24 months post transplantation.
- Primary: Assess safety and tolerability of bemdaneprocel at 1 year post transplantation.
- Secondary: Assess evidence of survival of transplanted cells and motor effects at 1 and 2 years post transplantation.
Safety and Tolerability
- No deaths, discontinuations, or graft-induced dyskinesias occurred.
- All participants reported >=1 TEAE, but most were mild or moderate in severity.
- 3 TESAEs occurred; none were related to bemdaneprocel.
- Feasibility of stereotactic administration was demonstrated: all participants received the intended number of cell deposits.
Conclusions
- Exploratory outcomes were stable or improved through 18 months, 6 months post discontinuation of immunosuppression.
- No serious adverse events were attributed to the transplanted cells or immunosuppression.
- No graft-induced dyskinesias were reported throughout the 18 months.
- All participants will be asked to enroll in a long-term extension study for continued evaluation of safety and efficacy.
- Participants in the high-dose cohort trended more strongly toward improvement compared with participants in the low-dose cohort.
- These findings should be interpreted carefully given the small, open-label nature of this study.
- These findings support the continued development of bemdaneprocel for the treatment of people with Parkinson’s disease.
- Bemdaneprocel was well tolerated, with no major safety issues in all 12 participants through 18 months.
View references
- Lubomski M, et al. J Mov Disord. 2021;14(1):42-52.
- Armstrong MJ, Okun MS. JAMA. 2020;323(6):548-560.
- Kim TW, et al. Cell Stem Cell. 2021;28(2):343-355.e5.
- Piao J, et al. Cell Stem Cell. 2021;28(2):217-229.e7.
- Steinbeck JA. Nat Biotechnol. 2015;33(2):204-209.
- Henchcliffe C, et al. Presented at: International Congress of Parkinson’s Disease and Movement Disorders; August 27-31, 2023; Copenhagen, Denmark.
