Parkinson's Disease Fact Sheet

CIRM funds many projects seeking to better understand Parkinson's disease and to translate those discoveries into new therapies.


Parkinson's disease is a neurodegenerative disease that affects approximately a million people in the United States and seven million people around the world. Symptoms include tremors, slow movement, muscle rigidity, balance issues and lack of facial expressions. Parkinson's disease occurs when the neurons or nerve cells in the portion of the brain that controls movement die off. These neurons send signals by releasing a chemical called dopamine, and are referred to as dopaminergic neurons. No cure exists for the disease and current medications become less effective over time.

Stem cell scientists are taking two general approaches to target Parkinson's disease. The first approach involves understanding the disease and looking for new drugs to treat it. CIRM grantees have taken skin cells from people with Parkinson’s disease, reprogrammed them back to an embryonic-like state, turning them into the kind of stem cell that can be transformed into any other cell in the body, then coaxing those cells to become dopaminergic neurons that are lost to the disease. Those cells showed signs of the disease in the lab dish, and were distinctly different from the same cells created from healthy people.


Brain Neurotherapy Bio

Dr. Krystof Bankiewiecz and his team are using a gene therapy approach to promote the production of a protein called GDNF, which is best known for its ability to protect dopaminergic neurons, the kind of cell damaged by Parkinson’s Disease. The approach seeks to increase dopamine production in the brain, alleviating PD symptoms and potentially slowing down the disease progress.

Video: Progress and Promise in Developing a Cure for Parkinson's Disease

Being able to study human Parkinson’s disease cells in a lab dish is a major milestone. Now, scientists can expose those cells to different drugs to find the ones that eliminate signs of the disease. If scientists find drugs that treat the disease in a lab dish, they will then test those same drugs in animal models and develop the most promising into a therapy for people with the disease. Several teams of CIRM-funded researchers are using stem cell techniques to create Parkinson’s disease cells in the lab dish and then screening them for new drugs.

Other groups are creating dopamine-producing cells in the lab dish with the hope that they could replace the neurons that are damaged in people with the disease. See below for a list of a CIRM-funded projects related to Parkinson's disease. 

CIRM Grants Targeting Parkinson's Disease

Researcher name Institution Grant Title Grant Type Award Amount
David Schaffer University of California, Berkeley Engineering Defined and Scaleable Systems for Dopaminergic Neuron Differentiation of hPSCs Tools and Technologies II $1,340,816
Susan McConnell Stanford University Identification and characterization of human ES-derived DA neuronal subtypes Basic Biology I $1,404,853
R. Jeremy Nichols Parkinson's Institute Understanding the role of LRRK2 in iPSC cell models of Parkinson's Disease Basic Biology III $1,482,822
Xianmin Zeng Buck Institute for Age Research Banking transplant ready dopaminergic neurons using a scalable process Early Translational II $4,983,013
Stuart Lipton Sanford Burnham Prebys Medical Discovery Institute hESC-derived NPCs Programmed with MEF2C for Cell Transplantation in Parkinson’s Disease Disease Team Therapy Planning I $96,448
Fred Gage Salk Institute for Biological Studies Crosstalk: Inflammation in Parkinson’s disease (PD) in a humanized in vitro model Early Translational II $2,472,839
Marcel Daadi Sanford Burnham Prebys Medical Discovery Institute Neural Stem Cell-Based Therapy For Parkinson’s Disease Disease Team Therapy Planning I $63,952
Zhuohua Zhang Sanford Burnham Prebys Medical Discovery Institute Derivation of Parkinson's Disease Coded-Stem Cells (PD-SCs) New Cell Lines $1,556,448
Steven Finkbeiner Gladstone Institutes, J. David Common molecular mechanisms in neurodegenerative diseases using patient based iPSC neurons Basic Biology IV $1,395,184
Su Guo University of California, San Francisco Identifying small molecules that stimulate the differentiation of hESCs into dopamine-producing neurons SEED Grant $542,619
Xinnan Wang Stanford University Misregulated Mitophagy in Parkinsonian Neurodegeneration Basic Biology V $1,174,943
Susan McConnell Stanford University Optimization of guidance response in human embryonic stem cell derived midbrain dopaminergic neurons in development and disease SEED Grant $607,363
Zhuohua Zhang Sanford Burnham Prebys Medical Discovery Institute Modeling Parkinson's Disease Using Human Embryonic Stem Cells SEED Grant $701,060
David Schaffer University of California, Berkeley Engineered Biomaterials for Scalable Manufacturing and High Viability Implantation of hPSC-Derived Cells to Treat Neurodegenerative Disease Tools and Technologies III $1,239,276
Stuart Lipton Sanford Burnham Prebys Medical Discovery Institute MEF2C-Directed Neurogenesis From Human Embryonic Stem Cells Comprehensive Grant $2,832,000
Jeanne Loring Scripps Research Institute Autologous cell therapy for Parkinson’s disease using iPSC-derived DA neurons Quest - Discovery Stage Research Projects $2,299,786
Fred Gage Salk Institute for Biological Studies Molecular and Cellular Transitions from ES Cells to Mature Functioning Human Neurons Comprehensive Grant $2,749,293
Birgitt Schuele Parkinson's Institute CRISPR/dCas9 mutant targeting SNCA promoter for downregulation of alpha-synuclein expression as a novel therapeutic approach for Parkinson’s disease Quest - Discovery Stage Research Projects $1,288,415
J. William Langston Parkinson's Institute Using patient-specific iPSC derived dopaminergic neurons to overcome a major bottleneck in Parkinson's disease research and drug discovery Early Translational I $3,698,646
Justin Cooper-White Scaled Biolabs Inc. A tool for rapid development of clinical-grade protocols for dopaminergic neuronal differentiation of Parkinson’s Disease patient-derived iPSCs Quest - Discovery Stage Research Projects $657,528
Evan Snyder Sanford Burnham Prebys Medical Discovery Institute Developmental Candidates for Cell-Based Therapies for Parkinson's Disease (PD) Early Translational I $5,190,752
Vicki Nienaber Zenobia Therapeutics A new phenotypic screening platform that identifies biologically-relevant targets and lead compounds for the treatment of Parkinson’s disease Inception - Discovery Stage Research Projects $112,500
David Schaffer University of California, Berkeley Directed Evolution of Novel AAV Variants for Enhanced Gene Targeting in Pluripotent Human Stem Cells and Investigation of Dopaminergic Neuron Differentiation Tools and Technologies I $918,000
Krystof Bankiewicz University of California, San Francisco MRI Guided Delivery of Neural Progenitor Cells Secreting GDNF for the Treatment of Parkinson's disease Late Stage Preclinical Projects $5,811,340
Lei Wang Salk Institute for Biological Studies Genetic Encoding Novel Amino Acids in Embryonic Stem Cells for Molecular Understanding of Differentiation to Dopamine Neurons New Faculty I $2,587,742
Andres Bratt-Leal Aspen Neuroscience Autologous therapy for Parkinson's disease: single cell RNAseq for in depth characterization of transplanted cells Progression Award - Discovery Stage Research Projects $177,579
Michele Calos Stanford University Site-specific integration of Lmx1a, FoxA2, & Otx2 to optimize dopaminergic differentiation Tools and Technologies II $1,592,897
Krystof Bankiewicz Brain Neurotherapy Bio A Phase 1b Safety Study for MRI guided delivery of AAV2-GDNF for the treatment of Parkinson’s disease Clinical Trial Stage Projects $5,510,462
Birgitt Schuele Parkinson's Institute Editing of Parkinson’s disease mutation in patient-derived iPSCs by zinc-finger nucleases Tools and Technologies II $1,327,983
Stuart Lipton Scripps Research Institute Drug Development of Inhibitors of Inflammation Using Human iPSC-Derived Microglia (hiMG) Quest - Discovery Stage Research Projects $1,658,123
Daniel Lim University of California, San Francisco Development and preclinical testing of new devices for cell transplantation to the brain. Tools and Technologies II $1,795,891

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