The Medicine by Design Global Speaker Series invites established and emerging international leaders in regenerative medicine to engage with our extraordinary community of researchers and clinicians.
Medicine by Design is pleased to welcome, Lorenz Studer, MD, Director, Center for Stem Cell Biology, Memorial Sloan-Kettering Cancer Center.
Talk title: Applications of human PSCs in modeling and treating neural disease
This event will be held in-person only at the Terrence Donnelly Centre for Cellular & Biomolecular Research, Red Room.
About Lorenz Studer
Lorenz Studer investigates human stem cells as tools to understand normal and pathological development in the nervous system and to develop cell-based therapies.
His interest in medicine began when he was about 18. He had been in the hospital himself for an unusual type of bone infection and had also lost several close relatives to cancer. He decided to go to medical school, spending my first two years at the University of Fribourg and the latter four at the University of Bern. There, he met Christian Spenger, a neurosurgery fellow who thought we might be able to treat Parkinson’s disease in animals by implanting fetal tissue to yield healthy new brain cells. This idea of replacing damaged brain cells seemed revolutionary to him.
After he earned my MD degree in 1991, Christian and he set up a laboratory at the University of Bern. We were given a room that had been used to store brooms. But it was a good size, and they managed to get funding for the resources they needed.
They began tissue culture studies and developed ways to ensure quality control of the animal fetal tissue cells. He developed an assay to measure the cells’ ability to produce dopamine — the chemical whose deficiency causes the symptoms of Parkinson’s. They also studied the effects of proteins necessary for the growth of specific brain cells. Their early work culminated in 1995, when they implanted human fetal nerve cells into a Parkinson’s patient.
Talk abstract
Human pluripotent stem cells (hPSCs) present a powerful tool for studying human disease and for developing novel cell-based therapies in regenerative medicine. Our group has developed strategies to coax human PSCs into many specific neuron subtypes on demand and at scale. For some lineages, such as midbrain dopamine neurons, those efforts have recently translated into a first-in-human clinical trial using clinical grade, “off-the-shelf” dopamine neurons for treating patients with advanced Parkinson’s disease (PD). I will provide an update on the results from this trial and discuss some of the next steps and potential next generation products in PD cell therapy development.
I will further discuss recent progress in the lab on chemical and genetic strategies to drive neuronal maturation and aging in human PSC-derived neural cells type that enable improved modeling neural disorders including neurodegenerative disease. Similarly, new protocols enable the derivation of fast spiking PV+ cortical interneurons, a neuronal cell type that has been very difficult to generate in past studies, as PV marker expression and maturation occurs largely during postnatal development, while hPSC-derived neurons typically capture fetal stages of neural development. Using hPSC-derived PV neurons we applied this protocol to study a set of structural variants highly associated with increased risk for schizophrenia development. This study enabled us to investigate novel interneuron-mediated defects that may be linked to aspects of neuropsychiatric disease.