After a positive initial safety evaluation, the pioneering STEM-PD clinical trial has advanced to higher dose testing.
STEM-PD is a first-in-human clinical trial testing a new investigational therapy for Parkinson's disease aimed at replacing the dopamine cells lost to the disease with healthy ones derived from stem cells.
The first patient in the the trial was transplanted at Skåne University Hopsital, Lund, Sweden, in February 2023, and the STEM-PD team now announces to have completed dosing of the first four patients with the initial lower dose of cells (3.5 million cells per putamen).
Based on the safety data, the study team has now proceeded as planned and initiated dosing of the next four patients with a higher dose of cells (7.1 million cells per putamen). The patients in the higher dose cohort will be grafted during 2024 and followed for 36 months within this trial.
The primary objective of the trial is to assess the safety and tolerability of the stem cell product STEM-PD over one year post grafting. No concerning side effects from the intervention have been reported and the patients are all doing well.
Secondary objectives of the trial include assessment of transplanted cell survival through dopamine PET imaging as well as evaluation of clinical efficacy three years post transplantation. It is still too early to evaluate clinical effects of the transplanted cells, but PET imaging of patient brains at 6-12 months post transplantation shows signs of dopamine cell survival.
The trial was approved by the Swedish Medical Products Agency in October 2022 and by the UK Medicines & Healthcare products Regulatory Agency in October 2023, enabling inclusion of UK patients in cohort number two.
More information about STEM-PD clinical trial: www.stem-pd.org
Key facts: research leaders and STEM-PD organisation
Malin Parmar, Project lead – Professor, Lund University
Gesine Paul-Visse, Principal Investigator – Adjunct Professor, Lund University and Senior Consultant Neurologist, Skåne University Hospital in Lund
Håkan Widner, Sponsor representative/co-investigator – Associate Professor, Lund University and Senior Consultant Neurologist, Skåne University Hospital in Lund
Hjálmar Bjartmarz, Co-Investigator – Senior Consultant Neurosurgeon, Skåne University Hospital in Lund
Agnete Kirkeby, Pre-clinical development lead – Associate Professor, Lund University
Roger Barker, Clinical Lead & Principal Investigator – Professor, University of Cambridge and Honorary Consultant Neurologist, Cambridge University Hospitals NHS Foundation Trust
Paola Piccini, Brain imaging expert – Professor and Honorary Consultant, Imperial College London
Oskar Hansson, PET imaging – Professor at Lund University and Senior Consultant at Skåne University Hospital
Ruben Smith, PET imaging – Associate Professor and researcher at Lund University and Physician at Skåne University Hospital
The trial is managed by Cambridge Clinical trials unit Neuroscience theme and Skåne University Hospital neurology research unit.
The development of the product and the trial is funded by national and EU funding agencies and Novo Nordisk A/S.
