Lund University: Swedish Medicines Agency Approves Clinical Trial of New Stem Cell-Based Treatment for Parkinson’s Disease | MarketScreener

An investigational stem cell-based therapy for the treatment of Parkinson’s disease, STEM-PD, has received regulatory approval for a Phase I/IIa clinical trial. Ethical approval for the study has already been obtained from the Swedish Ethics Review Boardand the STEM-PD team led by Lund University in Swedenis now ready to continue the process.

“We are excited and excited about this clinical trial of STEM-PD in the hope that it could potentially help alleviate the significant burden of Parkinson’s disease. This has been a tremendous team effort for over a decade, and regulatory approval is a major and important milestone,’ says Professor Malin Parmar who leads the MINT PD team Lund University in close cooperation with their colleagues Skane University Hospitalthe University of Cambridge, Cambridge University Hospitals NHS Foundation Trust (CUH) and Imperial College London.

STEM-PD is a dopamine neuron-based human embryonic stem cell product designed to replace the cells lost in Parkinson’s disease. This is the first such process in Europe and STEM-PD pre-clinical and clinical studies were funded by national and EU funding agencies. In addition, the MINT-PD team received funding and valuable support for the current study from Novo Nordisk; a collaboration that will continue for future product development.

The cells to be used in the study were manufactured under good manufacturing practice (GMP). Royal Free Hospital in London and have undergone rigorous laboratory testing.

“Our data show that the STEM-PD product is safe and highly effective in reversing motor deficits in preclinical models of Parkinson’s disease,” he says Agnet Kirkeby out Lund Universitywho led the pre-clinical development of the product.

The STEM-PD study is evaluating the safety and tolerability of transplantation of STEM-PD cells into the brains of patients with moderate Parkinson’s disease (EudraCT number: 2021-001366-38). The primary outcome of the STEM-PD study will be to evaluate the safety and tolerability of the transplanted product one year post-transplant, while secondary endpoints will assess the survival and function of the transplanted cells through brain imaging, as well as measure the impact on Parkinson’s symptoms. The STEM-PD study plans to enroll a total of 8 patients for transplantation, starting with patients from Swedenand with later plans for patient enrollment as well Cambridge University Hospital in which United Kingdom. The study has yet to start before the clinical trial sites are inspected by the authorities. Voluntary participation in the study is not possible.

Skane University Hospital in Lund (Sweden) is the clinical sponsor of the study and the site where all transplant surgeries will be performed. STEM-PD builds on a long history of similar work at this site: “Our teams have previously conducted cell transplant studies in Parkinson’s disease, but this is the first study using a stem cell-derived drug to replace dopamine neurons .” says Hakan Widnersponsor representatives Skane University Hospital. professor Roger Barker of the University of Cambridge and CUH, the project’s clinical leader, also comments: “The use of stem cells will theoretically allow us to produce unlimited amounts of dopamine neurons, thus opening the prospect of manufacturing this therapy for a broad patient population. This could change the way we treat Parkinson’s disease.”

Parkinson’s disease and its treatment

Parkinson’s disease is the second most common neurodegenerative disease worldwide and is still incurable. Typical motor symptoms of Parkinson’s disease are slowness of movement, tremors and stiffness, and later also gait difficulties. It is not known how the disease arises or develops, but the core pathology common to all patients is the loss of dopamine neurons in the midbrain.

Treatment of Parkinson’s disease has relied on symptomatic pharmacological therapies for over 50 years, which are particularly effective in the early years of diagnosis. However, such therapies are ultimately limited by the development of side effects related to systemic delivery and non-physiological dopamine levels. Targeted regenerative cell therapies specifically designed to restore lost dopaminergic input to the brain’s motor system would therefore represent a major advance in the treatment of Parkinson’s disease.

The process

STEM-PD is stem cell-based and is being performed for the first time in a human clinical trial for patients with Parkinson’s disease, aiming to replace dopamine neurons lost in the disease with new healthy neurons, thereby restoring dopaminergic innervation to the striatum.

The STEM-PD study is designed to be a single-arm, human-first, dose escalation study evaluating 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 developed by Roslin CT in the United Kingdom. The STEM-PD product was manufactured in a Center for Cell, Gene and Tissue Therapeutics at GMP facility Royal Free Hospital in Londonand is imported into the European Union through HiTech Health in Dublin.

The cell suspension is delivered to the brain by stereotactic surgery using a special implantation device. The device is manufactured in-house Scanian region and has previously been used to deliver tissue-derived dopamine neurons at Skane University Hospital. Scanian region –Skane University Hospital is the surgical site in the study and will therefore be the first administration site in this first human study.

Principal investigator of the STEM-PD study

Malin ParmarProject management – Professor, Lund University

Agnet KirkebyHead of Preclinical Development – Associate Professor, Lund University

Roger BarkerClinical Director & Principal Investigator – Professor, University of Cambridge and volunteer specialist in neurology, Cambridge University Hospitals NHS Foundation Trust

Gesine Paul VissePrincipal Investigator – Adjunct Professor, Lund University and Chief Physician Neurologist, Skane University Hospital in Lund

Hakan WidnerRepresentative of the sponsor/co-investigator – lecturer, Lund University and Chief Physician Neurologist, Skane University Hospital in Lund

Hjalmar Bjartmarz, Co-Investigator – Senior Physician Neurosurgeon, Skane University Hospital in Lund

Paola PicciniBrain Imaging Expert – Professor and Honorary Advisor, Imperial College London

Institutions involved in the project

Lund University:

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

Skane University Hospital

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

Cambridge University Hospitals NHS Foundation Trust (CUH):

Study coordination and planned secondary study site for patient recruitment.

University of Cambridge:

STEM-PD development and design of experiments.

Imperial College London:

PET imaging coordination.

Novo Nordisk A/S:

Project collaborator, partner in the future development of the STEM-PD product and financial support of the STEM-PD study.

Boyds: Advice on regulatory filings.

Center for Cell, Gene and Tissue Therapeutics at Royal Free Hospital, London:

GMP manufacturer of STEM-PD products.

Covance Inc.:

CRO site for GLP safety study.

Roslin CT:

Manufacturing site for the RC17 hESC line used for STEM-PD fabrication. Location of buffer manufacture for STEM-PD.

Hitech health:

Partner for EU QP release of STEM-PD.