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 NameInstitutionGrant TitleGrant TypeAward Amount
Dr. Steve M. FinkbeinerGladstone Institutes, J. DavidLewy body dementia, 𝛼-synuclein, and cell-specific mechanisms of neurodegenerationFoundation – Discovery Stage Research Projects$1,739,760
Dr. Andres Bratt-LealAspen NeuroscienceAutologous therapy for Parkinson’s disease: single cell RNAseq for in depth characterization of transplanted cellsProgression Award – Discovery Stage Research Projects$177,579
Stuart A LiptonScripps Research InstituteDrug Development of Inhibitors of Inflammation Using Human iPSC-Derived Microglia (hiMG)Quest – Discovery Stage Research Projects$1,658,123
Dr Justin Cooper-WhiteScaled Biolabs Inc.A tool for rapid development of clinical-grade protocols for dopaminergic neuronal differentiation of Parkinson’s Disease patient-derived iPSCsQuest – Discovery Stage Research Projects$657,528
Dr. Birgitt SchueleParkinson’s InstituteCRISPR/dCas9 mutant targeting SNCA promoter for downregulation of alpha-synuclein expression as a novel therapeutic approach for Parkinson’s diseaseQuest – Discovery Stage Research Projects$1,288,415
Dr. Jeanne F LoringScripps Research InstituteAutologous cell therapy for Parkinson’s disease using iPSC-derived DA neuronsQuest – Discovery Stage Research Projects$2,299,786
Vicki NienaberZenobia TherapeuticsA new phenotypic screening platform that identifies biologically-relevant targets and lead compounds for the treatment of Parkinson’s diseaseInception – Discovery Stage Research Projects$112,500
Abhay JoshiAxent Biosciences Inc.A high quality, accessible cell therapy for Parkinson’s Disease produced in a scalable bioreactor system for 3D cell expansion and differentiationTherapeutic Translational Research Projects$3,999,241
Dr. Emmanuel E BaetgeBrainXell TherapeuticsDevelopment of Autologous Cell Replacement Therapy for Parkinson’s Disease: Path to Personalized TreatmentTherapeutic Translational Research Projects$3,841,110
Dr. Edward Wirth IIIAspen NeurosciencePhase 1/2a Dose Escalation Study of ANPD001 in Sporadic Parkinson DiseaseClinical Trial Stage Projects$8,000,000
Krystof S. BankiewiczBrain Neurotherapy BioA Phase 1b Safety Study for MRI guided delivery of AAV2-GDNF for the treatment of Parkinson’s diseaseClinical Trial Stage Projects$5,510,462
Howard J FederoffKenai TherapeuticsAllogeneic iPSC derived Dopaminergic Drug Product for Parkinson’s diseaseLate Stage Preclinical Projects$4,000,000
Dr. Krystof BankiewiczUniversity of California, San FranciscoMRI Guided Delivery of Neural Progenitor Cells Secreting GDNF for the Treatment of Parkinson’s diseaseLate Stage Preclinical Projects$5,757,078
Dr. David V. SchafferUniversity of California, BerkeleyEngineered Biomaterials for Scalable Manufacturing and High Viability Implantation of hPSC-Derived Cells to Treat Neurodegenerative DiseaseTools and Technologies III$1,239,276
Xinnan WangStanford UniversityMisregulated Mitophagy in Parkinsonian NeurodegenerationBasic Biology V$1,174,943
Dr. Steve M. FinkbeinerGladstone Institutes, J. DavidCommon molecular mechanisms in neurodegenerative diseases using patient based iPSC neuronsBasic Biology IV$1,395,184
Dr. Marcel Daadi Dr.Sanford Burnham Prebys Medical Discovery InstituteNeural Stem Cell-Based Therapy For Parkinson’s DiseaseDisease Team Therapy Planning I$63,952
Dr. Stuart A LiptonSanford Burnham Prebys Medical Discovery InstitutehESC-derived NPCs Programmed with MEF2C for Cell Transplantation in Parkinson’s DiseaseDisease Team Therapy Planning I$96,448
R. Jeremy NicholsParkinson’s InstituteUnderstanding the role of LRRK2 in iPSC cell models of Parkinson’s DiseaseBasic Biology III$1,482,822
Dr. David V. SchafferUniversity of California, BerkeleyEngineering Defined and Scaleable Systems for Dopaminergic Neuron Differentiation of hPSCsTools and Technologies II$1,340,816
Dr. Birgitt SchueleParkinson’s InstituteEditing of Parkinson’s disease mutation in patient-derived iPSCs by zinc-finger nucleasesTools and Technologies II$1,327,983
Dr Daniel A. Lim MD, PHDUniversity of California, San FranciscoDevelopment and preclinical testing of new devices for cell transplantation to the brain.Tools and Technologies II$1,795,891
Dr. Michele CalosStanford UniversitySite-specific integration of Lmx1a, FoxA2, & Otx2 to optimize dopaminergic differentiationTools and Technologies II$1,592,897
Dr. Fred H GageSalk Institute for Biological StudiesCrosstalk: Inflammation in Parkinson’s disease (PD) in a humanized in vitro modelEarly Translational II$2,472,839
Dr. Xianmin ZengBuck Institute for Age ResearchBanking transplant ready dopaminergic neurons using a scalable processEarly Translational II$4,983,013
Dennis SteindlerParkinson’s InstituteStem Cell Pathologies in Parkinson’s disease as a key to Regenerative StrategiesResearch Leadership$0
Dr. Lei WangSalk Institute for Biological StudiesGenetic Encoding Novel Amino Acids in Embryonic Stem Cells for Molecular Understanding of Differentiation to Dopamine NeuronsNew Faculty I$2,587,742
Dr Evan Y SnyderSanford Burnham Prebys Medical Discovery InstituteDevelopmental Candidates for Cell-Based Therapies for Parkinson’s Disease (PD)Early Translational I$5,190,752
Mr. James “Bill” William Langston Mr.Parkinson’s InstituteUsing patient-specific iPSC derived dopaminergic neurons to overcome a major bottleneck in Parkinson’s disease research and drug discoveryEarly Translational I$3,698,646
Dr. David V. SchafferUniversity of California, BerkeleyDirected Evolution of Novel AAV Variants for Enhanced Gene Targeting in Pluripotent Human Stem Cells and Investigation of Dopaminergic Neuron DifferentiationTools and Technologies I$918,000
Dr. Zhuohua ZhangSanford Burnham Prebys Medical Discovery InstituteDerivation of Parkinson’s Disease Coded-Stem Cells (PD-SCs)New Cell Lines$1,556,448
Dr. Stuart A LiptonSanford Burnham Prebys Medical Discovery InstituteMEF2C-Directed Neurogenesis From Human Embryonic Stem CellsComprehensive Grant$2,832,000
Dr. Fred H GageSalk Institute for Biological StudiesMolecular and Cellular Transitions from ES Cells to Mature Functioning Human NeuronsComprehensive Grant$2,749,293
Dr. Zhuohua ZhangSanford Burnham Prebys Medical Discovery InstituteModeling Parkinson’s Disease Using Human Embryonic Stem CellsSEED Grant$701,060
Dr. Susan K McConnellStanford UniversityOptimization of guidance response in human embryonic stem cell derived midbrain dopaminergic neurons in development and diseaseSEED Grant$607,363
Dr. Su GuoUniversity of California, San FranciscoIdentifying small molecules that stimulate the differentiation of hESCs into dopamine-producing neuronsSEED Grant$542,619
Dr. Susan K McConnellStanford UniversityIdentification and characterization of human ES-derived DA neuronal subtypesBasic Biology I$1,404,853

CIRM Parkinson’s Disease Videos

Lorenz Studer, Winner of the 2017 Ogawa-Yamanaka Stem Cell Prize

Lorenz Studer, Winner of the 2017 Ogawa-Yamanaka Stem Cell Prize

Suzanne Peterson, Scripps – CIRM Stem Cell #SciencePitch

Jessica Westfall, The Parkinson’s Institute – CIRM Stem Cell #SciencePitch

Jeanne Loring, Scripps – CIRM Stem Cell #SciencePitch: Parkinson’s Disease

Parkinson’s: Ask the Stem Cell Expert | Xianmin Zeng, Buck Institute

Greg Wasson, Parkinson’s Action Network: Patient Advocate Presentation

Parkinson’s Disease: Advancing Stem Cell Therapies – 2011 CIRM Grantee Meeting

Stem Cells and Parkinson’s Disease

Progress and Promise in Parkinson’s

Spotlight on Parkinson’s Disease: Seminar by Jeff Bronstein, M.D., Ph.D.

Spotlight on Parkinson’s Disease: Seminar by Arnold Kriegstein, M.D., Ph.D.

Spotlight on Parkinson’s Disease: Seminar by Bruce Wisnicki

News and Information


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