An investigational stem cell-based therapy for the treatment of Parkinson's Disease, STEM-PD, has been given regulatory approval for a Phase I/IIa clinical trial. Ethical approval of the trial has already been obtained from the
'We are excited and looking forward to this clinical study of STEM-PD, hoping that it could potentially help reduce the significant burden of Parkinson's disease. This has been a massive team effort for over a decade, and the regulatory approval is a major and important milestone' says Professor
STEM-PD is an investigational human embryonic stem cell based dopamine nerve cell product designed to replace the cells which are lost in Parkinson's disease. This is the first such trial in
The cells to be used in the trial have been manufactured under good manufacturing practice (GMP) at the
'Our data shows that the STEM-PD product is safe and highly efficacious in reverting motor deficits in preclinical models of Parkinson's disease' says
The STEM-PD trial is investigating the safety and tolerability of transplanting STEM-PD cells into the brain of patients with moderate Parkinson's disease (EudraCT Number:2021-001366-38). The primary outcome of the STEM-PD trial is to assess safety and tolerability of the transplanted product at 1-year post-transplantation, while secondary endpoints will assess survival and function of the transplanted cells by brain imaging, as well as measure effects on Parkinson's symptoms. The STEM-PD trial plans to enroll a total of 8 patients for transplantation, starting with patients from
Parkinson's disease and its treatment
Parkinson's disease is the second most common neurodegenerative disease worldwide, yet remains without a cure. Typical motor symptoms of Parkinson's disease are slowness of movement, tremor and stiffness and later also gait difficulties. It is not well known how the disease arises or develops, but the core pathology common to all patients is the loss of dopamine neurons in the midbrain.
The treatment of Parkinson's disease has for over 50 years relied on symptomatic pharmacological therapies that are highly effective, especially in the early years of the diagnosis. However, such therapies are ultimately limited by the development of side effects that relate to the systemic delivery and non-physiological dopamine levels. Targeted regenerative cell therapies designed to restore specifically the lost dopaminergic input to the motor system of the brain would therefore represent a major advance in treating Parkinson's disease.
The trial
STEM-PD is astem-cell based, first in human clinical trial for patients with Parkinson's disease aiming to replace the dopamine neurons lost in the disease with new healthy neurons, thereby restoring the dopaminergic innervation of the striatum.
The STEM-PD trial is designed as a single arm, first in human, dose-escalation study, investigating the intrastriatal transplantation of stem cell derived dopaminergic cells for Parkinson's disease. The STEM-PD product is based on RC17, a GMP human embryonic stem cell line derived by Roslin CT in the
The cell suspension is administered to the brain by stereotactic surgery using a dedicated implantation device. The device is in-house manufactured at
Principal investigators of the STEM-PD trial
Hjalmar Bjartmarz, Co-Investigator - Senior Consultant Neurosurgeon,
Institutions involved in the project
Project lead, owner of STEM-PD product and site of product development.
Sponsor of the STEM-PD trial and site of surgical implantation for all patients.
Trial coordination and planned secondary site of trial for patient recruitment.
STEM-PD development and trial planning.
PET imaging coordination.
Collaborator on project, partner on future development of STEM-PD product and financial support of the STEM-PD trial.
Boyds: Consultancy for regulatory filing.
Centrefor Cell, Gene and Tissue Therapeutics at
GMP manufacturer of STEM-PD product.
CRO site for GLP safety study.
Roslin CT:
Site of manufacturing for the RC17 hESC line used for STEM-PD manufacturing. Site of buffer manufacturing for STEM-PD.
Partner for EU QP release of STEM-PD.
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