Personalized iPSC-Derived Dopamine Progenitor Cells for Parkinson's Disease.

We report the implantation of patient-derived midbrain dopaminergic progenitor cells, differentiated in vitro from autologous induced pluripotent stem cells (iPSCs), in a patient with idiopathic Parkinson's disease. The patient-specific progenitor cells were produced under Good Manufacturing Practice conditions and characterized as having the phenotypic properties of substantia nigra pars compacta neurons; testing in a humanized mouse model (involving peripheral-blood mononuclear cells) indicated an absence of immunogenicity to these cells. The cells were implanted into the putamen (left hemisphere followed by right hemisphere, 6 months apart) of a patient with Parkinson's disease, without the need for immunosuppression. Positron-emission tomography with the use of fluorine-18-L-dihydroxyphenylalanine suggested graft survival. Clinical measures of symptoms of Parkinson's disease after surgery stabilized or improved at 18 to 24 months after implantation. (Funded by the National Institutes of Health and others.).

Introduction of Network-Based Healthcare at Kaiser Permanente.

BACKGROUND: Early 2014, Kaiser Permanente decided to adopt an innovative model for network-based allied healthcare for persons with Parkinson's disease (PD), based on the principles of the Dutch ParkinsonNet. OBJECTIVE: We present the interventions that were performed to implement this method at Kaiser Permanente and we show the first outcomes based on these interventions. METHODS: In this study, 57 physical therapists, 18 speech therapists and 20 occupational therapists, as well as 13 medical centers across the state of California were included. Nine interventions were performed more or less simultaneously, including training and education of healthcare providers and patients, a train the trainer curriculum, organizing IT, streamlining referral processes and building networks. RESULTS: At the start, less than 30% of the patients within the Southern California Region received specialized allied health treatment (consisting of, i.e., gait training, voice training or guidance in activities of daily life). After one year, almost 55% of patients received specialized allied health treatment. In the second year, this number increased to just under 67%, suggesting a sustained concentration of care (the second core component of networked care). This can be seen as a first indicator for successful implementation of the ParkinsonNet network at Kaiser Permanente. CONCLUSIONS: The importance of these findings lies in the fact that a healthcare innovation that proved effective in one country can be transferred successfully to another country and to another healthcare system.

Using the PDQ-39 in routine care for Parkinson's disease.

INTRODUCTION: Using the 39-item Parkinson's Disease Questionnaire (PDQ-39) in routine care could efficiently identify symptoms that are most important to patients, but little evidence documents its use for this purpose. METHODS: A quality improvement pilot project using interviews with patients, caregivers, and providers. RESULTS: PDQ administration and scoring were successfully integrated into clinic workflows, and results were available for discussions during outpatient visits. Patients reported that completing the instrument helped them remember what to discuss with their provider, formulate specific questions, and identify factors affecting their quality of life. Caregivers found it useful for gaining insight into patients' daily needs. The neurologist reported it made provider-patient discussions more efficient by focusing on domains that were particularly concerning for patients. Ancillary care providers reported that PDQ-39 domain subscores efficiently provided a holistic view of patients' needs and supported setting short- and long-term goals. CONCLUSION: The PDQ-39 can be integrated into a busy clinical practice and its routine use is valuable for patients, caregivers, and providers.

Pluripotent stem cell-based therapy for Parkinson's disease: Current status and future prospects.

Parkinson's disease (PD) is one of the most common neurodegenerative disorders, which affects about 0.3% of the general population. As the population in the developed world ages, this creates an escalating burden on society both in economic terms and in quality of life for these patients and for the families that support them. Although currently available pharmacological or surgical treatments may significantly improve the quality of life of many patients with PD, these are symptomatic treatments that do not slow or stop the progressive course of the disease. Because motor impairments in PD largely result from loss of midbrain dopamine neurons in the substantia nigra pars compacta, PD has long been considered to be one of the most promising target diseases for cell-based therapy. Indeed, numerous clinical and preclinical studies using fetal cell transplantation have provided proof of concept that cell replacement therapy may be a viable therapeutic approach for PD. However, the use of human fetal cells as a standardized therapeutic regimen has been fraught with fundamental ethical, practical, and clinical issues, prompting scientists to explore alternative cell sources. Based on groundbreaking establishments of human embryonic stem cells and induced pluripotent stem cells, these human pluripotent stem cells have been the subject of extensive research, leading to tremendous advancement in our understanding of these novel classes of stem cells and promising great potential for regenerative medicine. In this review, we discuss the prospects and challenges of human pluripotent stem cell-based cell therapy for PD.