Abstract Details

Title Imaging Outcomes of a Dopaminergic Neuronal Cell Therapy for Parkinson’s Disease: 18-month Results from a Phase 1 Study of Bemdaneprocel

Topic Movement Disorders

Presentation(s) S30 - Movement Disorders: Clinical Trials in Movement Disorders (1:48 PM-2:00 PM)

Poster/Presentation
Number 005

Background Bemdaneprocel is an investigational cellular therapy composed of pluripotent stem cell–derived dopaminergic neuron precursor cells under development for the treatment of Parkinson’s disease. At 1 year post transplantation, bemdaneprocel was generally safe and well tolerated in 12 participants. Relative to baseline, increases in putamen ¹⁸F-DOPA PET signal indicated successful transplantation and graft survival in the target region.

Objective Evaluate [¹⁸F]-fluorodopa (¹⁸F-DOPA) positron emission tomography (PET) signal 18 months post transplantation (6 months after cessation of immunosuppression) in 12 participants who received intraputamenal transplants of bemdaneprocel.

Design/Methods In this open-label, 24-month, non-controlled study, 12 participants received 1 of 2 doses of bemdaneprocel to the post-commissural putamen bilaterally and a 1-year immunosuppression regimen. Changes from baseline in ¹⁸F-DOPA uptake were measured by striatal-to-occipital ratio for each participant within regions of interest defined by voxel-based brain mapping analysis at a group level. Participants were also assessed for morphologic changes by magnetic resonance imaging (MRI) before and after surgery and at multiple time points through year 1.

Results Compared with baseline, group-level analysis (N=12) of ¹⁸F-DOPA uptake at 1 year post transplantation revealed clusters of increased signal within the striatal hypothesis testing space (p<0.05). Whereas increases in ¹⁸F-DOPA uptake were discerned along surgical tracts through the putamen (p<0.005), caudate signal declined over the same time interval (p<0.01). MRI demonstrated no changes in target volume over the 1 year post transplantation.

Conclusions Increased post-transplantation ¹⁸F-DOPA PET signal along posterior putamen surgical trajectories is consistent with survival of bemdaneprocel grafts at 1 year. Concurrent MRI assessments of target volume did not reveal evidence of cell overgrowth during the same time period. Additional results will be presented, including imaging data acquired 18 months after transplantation (6 months after the cessation of immunosuppression).

Authors/Disclosures
Claire Henchcliffe, MD, PhD, FAAN (University of California, Irvine) PRESENTER	Dr. Henchcliffe has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Bayer. Dr. Henchcliffe has received personal compensation in the range of $500-$4,999 for serving as a Consultant for AskBio. Dr. Henchcliffe has received personal compensation in the range of $0-$499 for serving as a Consultant for Certara. Dr. Henchcliffe has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Meira GTx. Dr. Henchcliffe has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for ProJenX. Dr. Henchcliffe has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Canary Global Inc . Dr. Henchcliffe has stock in Axent Biosciences Inc. The institution of Dr. Henchcliffe has received research support from Weston Brain Institute. The institution of Dr. Henchcliffe has received research support from Blue Rock Therapeutics. Dr. Henchcliffe has received personal compensation in the range of $500-$4,999 for serving as a Speaker with Parkinson Study Group. Dr. Henchcliffe has received personal compensation in the range of $500-$4,999 for serving as a Speaker with UCLA. Dr. Henchcliffe has received personal compensation in the range of $500-$4,999 for serving as a Speaker travel with World Congress of Neurology. Dr. Henchcliffe has received personal compensation in the range of $0-$499 for serving as a Speaker travel with IAPRD. Dr. Henchcliffe has received personal compensation in the range of $0-$499 for serving as a Speaker travel with ASGCT. Dr. Henchcliffe has received personal compensation in the range of $500-$4,999 for serving as a Speaker/Course director with AAN.
Yilong Ma, PhD (Feinstein Institute for Medical Research)	No disclosure on file
Harini Sarva, MD, FAAN (Weill Cornell Medical Center)	Dr. Sarva has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Novo Nordisk. Dr. Sarva has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Novo Nordisk. The institution of Dr. Sarva has received research support from Insightec. The institution of Dr. Sarva has received research support from NeuroNext. The institution of Dr. Sarva has received research support from Neuroderm. The institution of Dr. Sarva has received research support from Covance. The institution of Dr. Sarva has received research support from Prevail. The institution of Dr. Sarva has received research support from Bluerock Therapeutics. The institution of Dr. Sarva has received research support from Biogen. The institution of Dr. Sarva has received research support from Roche. The institution of Dr. Sarva has received research support from National Institute of Aging. The institution of Dr. Sarva has received research support from Michael J Fox Foundation. The institution of Dr. Sarva has received research support from Novo Nordisk. The institution of Dr. Sarva has received research support from Bukwang. The institution of Dr. Sarva has received research support from Bial. The institution of Dr. Sarva has received research support from Cerevance. The institution of Dr. Sarva has received research support from UCB.
Andres M. Lozano, MD (Toronto Western Hosp)	Dr. Lozano has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Abbott. Dr. Lozano has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Insightec. Dr. Lozano has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Boston Scientific. Dr. Lozano has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Medtronic. Dr. Lozano has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Functional Neuromodulation.
Alfonso Fasano, MD, PhD, FAAN (Toronto Western Hospital - U of Toronto)	Dr. Fasano has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Abbott. Dr. Fasano has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Abbvie. Dr. Fasano has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Ipsen. Dr. Fasano has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Medtronic. Dr. Fasano has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Boston Scientific. Dr. Fasano has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Abbott. Dr. Fasano has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Medtronic. Dr. Fasano has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Ceregate. Dr. Fasano has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Inbrain Neuroelectronics. Dr. Fasano has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Syneos Health. Dr. Fasano has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Annovis. Dr. Fasano has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for CADTH. The institution of Dr. Fasano has received research support from Boston Scientific. The institution of Dr. Fasano has received research support from Medtronic. The institution of Dr. Fasano has received research support from Abbvie. Dr. Fasano has received publishing royalties from a publication relating to health care.
Suneil Kalia	No disclosure on file
Kenny Yu (Memorial Sloan Kettering Cancer Center)	No disclosure on file
Cameron Brennan	No disclosure on file
	No disclosure on file
Whitney Stemple (BlueRock Therapeutics)	No disclosure on file
Nauman Abid, MD (BlueRock Therapeutics)	Dr. Abid has received personal compensation for serving as an employee of BlueRock Therapeutics.
Marcus Yountz, MD, FAAN (BlueRock Therapeutics)	Dr. Yountz has received personal compensation for serving as an employee of Alexion, AstraZeneca Rare Disease. Dr. Yountz has stock in AstraZeneca.
Ahmed Enayetallah, PhD, MBBCh (BlueRock Therapeutics)	Dr. Enayetallah has received personal compensation for serving as an employee of Alexion. Dr. Enayetallah has received stock or an ownership interest from Alexion. Dr. Enayetallah has received stock or an ownership interest from AstraZeneca.
Antoine Lampron, PhD (Bluerock Therapeutics)	Mr. Lampron has received personal compensation for serving as an employee of Bluerock Therapeutics. Mr. Lampron has received personal compensation for serving as an employee of Alexion Inc..
Viviane Tabar	No disclosure on file
David Eidelberg, MD, FAAN (Feinstein Institute for Medical Research)	Dr. Eidelberg has received personal compensation in the range of $100,000-$499,999 for serving as a Consultant for MeiraGTx. Dr. Eidelberg has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Aspen Neuroscience. Dr. Eidelberg has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Oxford University Press. The institution of Dr. Eidelberg has received research support from The Michael J. Fox Foundation for Parkinson's Research. The institution of Dr. Eidelberg has received research support from Lupus Research Alliance. The institution of Dr. Eidelberg has received research support from National Institutes of Health. The institution of Dr. Eidelberg has received research support from Blue Rock Therapeutics. The institution of Dr. Eidelberg has received research support from National Institute of Neurological Disorders and Stroke. The institution of Dr. Eidelberg has received research support from The Michael J. Fox Foundation for Parkinson's Research. The institution of Dr. Eidelberg has received research support from National Institute of Allergy and Infectious Diseases. Dr. Eidelberg has received intellectual property interests from a discovery or technology relating to health care. Dr. Eidelberg has received intellectual property interests from a discovery or technology relating to health care.