University of Cambridge: Clinical trial for new stem cell-based treatment for Parkinson’s disease given go ahead
Cambridge researchers 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.
This could transform the way we treat Parkinson’s disease
Roger Barker
The Swedish Medical Products Agency has granted approval for the trial to proceed; ethical approval has already been obtained from the Swedish Ethics Review Authority. The team, led from Lund University in Sweden, is poised to begin recruitment.
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 has ‘programmed’ the cells to develop into dopamine nerve cell, 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.
The trial is a collaboration with colleagues at Skåne University Hospital, the University of Cambridge, Cambridge University Hospitals NHS Foundation Trust (CUH), and Imperial College London.
Professor Roger Barker from the Wellcome-MRC Stem Cell Institute at the University of Cambridge and CUH is clinical lead on the project. “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”
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’ at the Royal Free Hospital in London and have undergone rigorous testing in the lab.
Professor Malin Parmar who leads the STEM-PD team from Lund University said: “We are looking forward to this clinical study of STEM-PD, hoping that it could potentially help address 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.”
The STEM-PD trial will assess safety and tolerability of the transplanted product one year after transplantation, measuring the effects on Parkinson’s symptoms. The trial will enrol eight patients for transplantation, starting with patients from Sweden, and with subsequent plans for enrolment of patients also from Cambridge University Hospitals. All transplantation surgery will be performed at Skåne University Hospital in Lund.
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.