Abstract
Background and Objective
- Bemdaneprocel is an investigational therapy comprising dopaminergic neuronal progenitors derived from human embryonic stem cells.1,2
- Results from
exPDite, a Phase 1 multicenter, multisite, open-label, nonrandomized study (NCT04802733), at 12 months post transplantation demonstrated bemdaneprocel was generally safe and well tolerated, supported the feasibility of stereotactic transplantation of bemdaneprocel, and suggested stability or improvement in motor outcomes.3 - A favorable safety profile, as well as stability or improvement in motor outcomes, were maintained at 18 months post transplantation.
- At 12 months post transplantation, 18F-DOPA uptake was increased along the surgical track in the putamen, while signal declined in the caudate.3
Objective: To assess changes in 18F-DOPA uptake and safety up to 18 months post transplantation, 6 months post discontinuation of immunosuppression, using PET and MRI.
Trial Design
- 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: at baseline, 12, 18, and 24 months post transplantation.
- MRI: at screening, intraoperatively, postoperatively, and 1, 3, 6, 12, 18, and 24 months post transplantation.
- Bemdaneprocel was administered stereotactically into the posterior putamen bilaterally through a single burr hole on each side in a single session.
Conclusions
Increased 18F-DOPA PET signal in the posterior putamen is consistent with survival of bemdaneprocel at 12 and 18 months post transplantation.
- Bemdaneprocel survival continued after cessation of immunosuppression at 12 months post transplantation.
- Decreased 18F-DOPA PET signal in the caudate is indicative of continuing progression of Parkinson’s disease.
- These findings comport with findings from an independent analysis.
MRI assessments of target volume did not reveal evidence of cell overgrowth, tumor formation, or other safety concerns up to 18 months post transplantation.
These analyses demonstrate bemdaneprocel survival for 6 months post discontinuation of immunosuppression, or 18 months post transplantation, and continued dopaminergic signaling in the putamen.
View references
- Kim TW, Piao J, Koo SY, et al. Cell Stem Cell. 2021;28(2):343-55.e5.
- Piao J, Zabierowski S, Dubose BN, et al. Cell Stem Cell. 2021;28(2):217-29.e7.
- Henchcliffe C, Sarva H, Lozano A, et al. Presented at: International Congress of Parkinson’s Disease and Movement Disorders; August 27-31, 2023; Copenhagen, Denmark.
- Ma Y, Tang C, Chaly T, et al. J Nucl Med. 2010;51(1):7-15.
- Nakamura T, Dhawan V, Chaly T, et al. Ann Neurol. 2001;50:181-187.
- Ma Y, Feigin A, Dhawan V, et al. Ann Neurol. 2002;52:628-634.
