Parkinson’s disease affects over 10 million people worldwide, leading to the progressive loss of dopaminergic neurons in the brain. Current treatments help manage symptoms but do not stop the disease’s progression. Stem cell therapy has long been considered a potential solution, as transplanted neurons can restore lost function. However, producing these neurons at high purity levels has remained a challenge—until now.
Researchers at Aarhus University have developed a technique called lineage restriction, which enhances the precision of stem cell differentiation. Associate Professor Mark Denham and his team found that by modifying pluripotent stem cells and removing specific genes, they could prevent unwanted differentiation, ensuring that the majority of stem cells mature into dopaminergic neurons. This method more than doubles the proportion of usable neurons, addressing a key limitation in current transplantation methods.
“As with any drug, an unknown component in the treatment poses a safety risk, and if there are cells in the transplant that aren’t contributing to efficacy then we want to get rid of them,” says Denham. The technique increases not only the purity of the transplanted cells but also their effectiveness—preclinical trials in rat models indicate that fewer cells are needed to achieve significant recovery.
This research is part of the UNIPOTENT project, which focuses on translating laboratory findings into viable clinical treatments. With support from the Lundbeck Foundation, the team is applying their method to stem cells produced under Good Manufacturing Practice (GMP) conditions. This step is critical for moving toward human trials and securing investment for further development.
This advancement represents an important step toward making stem cell therapy a practical treatment for Parkinson’s.