Addenbrooke’s will play a key role in clinical trials of a new treatment that involves transplanting healthy nerve cells into the brains of patients with Parkinson’s disease.
CUH consultant Professor Roger Barker, who is also from the Wellcome-MRC Stem Cell Institute at the University of Cambridge, and clinical lead on the project, says it could “transform” the way Parkinson’s is treated.
The trial is a collaboration between Cambridge University Hospitals NHS Foundation Trust (CUH), the University of Cambridge, Imperial College London, Lund University and Skåne University Hospital in Sweden, where all transplantation surgery will be performed.
STEM-PD uses human embryonic stem cells, a type of cell that can turn into almost any type of cell in the body.
The team in Sweden has now developed ways of ‘programming’ the cells to develop into dopamine nerve cells, which will be transplanted into the brains of patients to replace cells that are lost in Parkinson’s disease. The product has already been shown to be safe and effective at reverting motor deficits in animal models of Parkinson’s disease.
Cells to be used in the trial have been manufactured under ‘Good Manufacturing Practice’ at the Royal Free Hospital in London and have undergone rigorous testing in the lab.
The trial will assess safety and tolerability of the transplanted product one year after transplantation, measuring the effects on Parkinson’s symptoms. It will enrol eight patients for transplantation, starting with patients from Sweden, and with subsequent plans to invite patients seen at the Cambridge University Hospitals.
An important milestone came when the Swedish Medical Products Agency granted approval for the trial to proceed. Ethical approval has already been obtained from the Swedish Ethics Review Authority.
STEM-PD is funded by national and EU funding agencies. In addition, the STEM-PD team has obtained funding and valuable support from Novo Nordisk, a collaboration which will continue for future product development.
Professor Barker, explained:
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.
Professor Roger Barker
Professor Malin Parmar who is the lead of the whole STEM-PD project and who works at Lund University added:
We are 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.
Professor Malin Parmar
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 feature common to all patients is the loss of dopamine neurons in the midbrain.
Suitable patients will be invited to participate in the trial; it is not possible to volunteer to participate.
Adapted from a press statement from Lund University
Listing image by Sabine van Erp on Pixabay