Disease Fact Sheet: Parkinson’s Disease (PD)
Clinical Status: Over the last 15 years, technologies and protocols have evolved to the point that midbrain dopamine neurons can now be made from both embryonic stem (ES) and induced pluripotent stem (iPS) cell sources, which show good survival in animal models of PD and demonstrate functional benefits. This work has progressed to the point where the first in-human clinical trials have started. The first one was initiated at Kyoto University in Japan 2018. Similar clinical trials using ES cells have also been initiated in both the USA and Europe. The USA trial conducted by BlueRock Therapeutics has been completed and showed that the approach was well tolerated with no major safety issues and with some early signs of graft survival and effect. A phase 2 trial is planned. In Europe, the STEM-PD trial, an academic trial involving Lund University/University Hospital in Sweden and Cambridge University/University Hospital in UK was initiated in 2023 and is still ongoing. In addition, a single case report of a patient receiving autologous iPSC-derived dopamine cells has been published.