A stem cell-based therapy for Parkinson's disease entered higher dose clinical testing after a positive initial safety evaluation.

STEM-PD uses human pluripotent stem cells that have been programmed to develop into dopamine nerve cells, which produce a chemical called dopamine that helps to control body movement. The stem cells are then transplanted into the brains of Parkinson's disease patients to replace cells that are lost during the course of the disease and to repair the damage caused. Current drugs, such as levodopa, only temporarily replace dopamine, but do not target the underlying disease.

'The vision is that it could be given as a one-time treatment and the hope is that the patients can reduce their medication, avoid side effects of the drug treatment and get a long-term good motor effect from the cells for life," Dr Gesine Paul-Visse, principal investigator from Lund University and Skne University Hospital, both in Sweden, said.

The method of growing transplantable dopamine cells from stem cells was initially developed by scientists at Lund University. The trial is now a collaboration between Lund University, Skne University Hospital, the University of Cambridge, Cambridge University Hospitals NHS Foundation Trust, and Imperial College London.

The human pluripotent stem cells used for generating the STEM-PD product are obtained from human embryonic stem cells, grown in the laboratory from a surplus embryo from IVF. The cells are then transplanted into a specific area of the patient's brain that is involved in motor control. After a few months, they start sending out nerve fibres and producing dopamine.

STEM-PD has already been shown to be safe and effective at reverting motor deficits in animal models of Parkinson's disease, and entered a first-in-human clinical trial in February 2023 at Skne University Hospital (see BioNews 1164).

An initial four patients were injected with a lower dose of seven million cells, with the team reporting no concerning side effects from the therapy. Furthermore, imaging of the patient brains 6-12 months' post transplantation showed signs of dopamine cell survival. Yet, the team cautions that it is still too early to evaluate the clinical effects of the transplanted stem cells.

The first patient to receive the stem cell therapy a year ago, Thomas Matsson, was diagnosed with Parkinson's disease when he was 42. He can now move freely again and has regained his sense of smell: 'I've reduced my medication for Parkinson's. Before, everything was slow and everything was difficult I do long-distance skating, slalom, cross-country skiing, padel tennis, and, above all, golf,' he said.

Now, a further patient has been injected with a higher dose of 14 million cells, with a further three patients to be treated in 2024. The primary objective of this trial is to assess the safety and tolerability of the therapy after one year, however, the patients will be monitored for three years with a secondary objective to evaluate the clinical efficacy of the therapy.

'There is absolutely hope. Absolutely there is!', added Dr Paul-Visse.

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Stem cell therapy success in early trial to treat Parkinson's disease | PET - BioNews