Non-motor Effects of Bemdaneprocel for Parkinson’s Disease: 18-month Results from a Phase 1 Study
Harini Sarva1, Claire Henchcliffe2, Andres Lozano3, Suneil Kalia4, Kenny Kwok Hei Yu5, Cameron Brennan5, Michele York6, Whitney Stemple7, Nauman Abid7, Marcus Yountz7, Ahmed Enayetallah7, Antoine Lampron7, Viviane Tabar5
1Weill Cornell Medical Center, 2University of California, Irvine, 3Toronto Western Hosp, 4University of Toronto, 5Memorial Sloan Kettering Cancer Center, 6Baylor College of Medicine, 7BlueRock Therapeutics
Objective:
Explore the impact of bemdaneprocel on non-motor symptoms in participants with Parkinson’s disease.
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 all 12 participants in a phase 1 study.
Design/Methods:
In this open-label, 24-month, non-controlled study, 12 participants received 1 of 2 different doses of bemdaneprocel bilaterally to the post-commissural putamen and a 1-year immunosuppression regimen. Exploratory outcomes assessed at 12 months included the impact of bemdaneprocel on non-motor symptoms. Assessments will be repeated at 18 months, 6 months post discontinuation of immunosuppression.
Results:
At 12 months, participants who received the higher dose of bemdaneprocel showed an improvement in non-motor symptoms, including a median (Q1, Q3) change from baseline of −28.7% (−58.5%, −1.2%) on the Non-Motor Symptom Scale total score and a median (Q1, Q3) change from baseline of −83.3% (−100%, −50.0%) on the Neuropsychiatric Inventory Questionnaire. Measures of activities of daily living and quality of life remained stable during this time, with a median (Q1, Q3) change from baseline of −9.1% (−42.9%, 11.1%) in part II of the Movement Disorders Society-Unified Parkinson’s Disease Rating Scale and a median (Q1, Q3) change from baseline of −9.9% (−55.1%, 47.4%) on the 39-item Parkinson’s Disease Questionnaire-39 (PDQ-39) summary index. Participants in the low-dose cohort had a median (Q1, Q3) change from baseline of −21.9% (−46.7%, 12.5%) on the PDQ-39 summary index.
Conclusions:
Overall, participants who received the higher dose of bemdaneprocel had improvement or no worsening from baseline in non-motor outcomes. Data from additional cognitive assessments and 18-month follow-up (6 months post discontinuation of immunosuppression) will be presented.