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Swedish Medical Products Agency grants approval for clinical study of new stem cell based Parkinson’s Disease treatment

Dopamine-producing neurons that researchers from Lund University have grown in the laboratory from human embryonic stem cells. Photo: Agnete Kirkeby.
Dopamine-producing neurons that researchers from Lund University have grown in the laboratory from human embryonic stem cells. Photo: Agnete Kirkeby.

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 Swedish Ethics Review Authority, and the STEM-PD team, led from Lund University in Sweden, is thereby ready to proceed with the trial.

“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 Malin Parmar who leads the STEM-PD team from Lund University in close collaboration with her colleagues at Skåne University Hospital, the University of Cambridge, Cambridge University Hospitals NHS Foundation Trust (CUH), and Imperial College London.

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 Europe and the preclinical and clinical studies of STEM-PD have been funded by national and EU funding agencies. In addition, the STEM-PD team has obtained funding and valuable support for the current study from Novo Nordisk; a collaboration which will continue for future product development.

The cells to be used in the trial have been manufactured under good manufacturing practice (GMP) at the Royal Free Hospital in London and have undergone rigorous testing in the lab.

“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 Agnete Kirkeby from Lund University, who has led the preclinical development of the product.

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 Sweden, and with subsequent plans for enrolment of patients also from Cambridge University Hospital in the UK. Trial initiation is pending clinical site inspection from the authorities. It is not possible to volunteer to participate in the trial.

Skåne University Hospital in Lund (Sweden) is the clinical sponsor of the trial and the site where all transplantation surgery will be performed. STEM-PD builds on a long history of similar work at this site: “Our teams have previously performed cell transplantation trials in Parkinson’s disease, but this is the first trial using a stem cell derived medicinal product for replacement of dopamine neurons.“ says Håkan Widner, sponsor representative from Skåne University Hospital. Professor Roger Barker from the University of Cambridge and CUH, the clinical lead on the project also comments that “The use of stem cells will in theory enable us to make unlimited amounts of dopamine neurons and thus opens the prospect of producing this therapy to a wide patient population. This could transform the way we treat Parkinson’s disease”.

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 UK. The STEM-PD product has been manufactured at a GMP facility at the Centrefor Cell, Gene and Tissue Therapeutics at Royal Free Hospital in London, and is imported into the European Union by HiTech Health in Dublin.

The cell suspension is administered to the brain by stereotactic surgery using a dedicated implantation device. The device is in-house manufactured at Region Skåne andhas previously been used for administration of tissue-derived dopamine neurons at Skåne University Hospital. Region Skåne –Skåne University Hospital is the surgical site in the trial, and will thus be the first-dosing site in this first-in-human trial.

Principal investigators of the STEM-PD trial

  • Malin Parmar, Project lead - Professor, Lund University
  • 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
  • Gesine Paul-Visse, Principal Investigator – Adjunct Professor, Lund University and Senior Consultant Neurologist, Skåne University Hospital in Lund
  • Håkan Widner, Sponsor representative/co-investigator – Docent, Lund University and Senior Consultant Neurologist, Skåne University Hospital in Lund
  • Hjálmar Bjartmarz, Co-Investigator – Senior Consultant Neurosurgeon, Skåne University Hospital in Lund
  • Paola Piccini, Brain imaging expert – Professor and Honorary Consultant, Imperial College London

Institutions involved in the project

Lund University:

Project lead, owner of STEM-PD product and site of product development.

Skåne University Hospital

Sponsor of the STEM-PD trial and site of surgical implantation for all patients.

Cambridge University Hospitals NHS Foundation Trust (CUH):

Trial coordination and planned secondary site of trial for patient recruitment.

University of Cambridge:

STEM-PD development and trial planning.

Imperial College London:

PET imaging coordination.

Novo Nordisk A/S:

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 Royal Free Hospital, London:

GMP manufacturer of STEM-PD product.

Covance Inc.:

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.

Hitech Health:

Partner for EU QP release of STEM-PD.