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New York Stem Cell Foundation Announces Partnership with Parkinson’s Progression Markers Initiative

The New York Stem Cell Foundation (NYSCF) Research Institute has entered into a partnership with the Parkinson’s Progression Markers Initiative (PPMI) to build resources for studying Parkinson’s disease to accelerate new treatments. Sponsored by The Michael J. Fox Foundation for Parkinson’s Research, PPMI is a landmark effort to identify and validate biomarkers of Parkinson’s disease. The study is currently under way at 32 clinical sites worldwide with funding from 13 industry partners.

As Michael J. Fox Returns to Primetime, His Research Foundation Urgently Pursues the Cure for Parkinson’s

Last month, Michael J. Fox returned to television as the star of his own sitcom after more than two decades living with Parkinson’s disease. Fox’s decision to return to primetime has injected Parkinson’s into the national conversation — a conversation already transformed by The Michael J. Fox Foundation for Parkinson’s Research (MJFF), which the actor launched in 2000 with the exclusive goal of funding research to speed a cure for the disease.

Comprehensive Parkinson’s Biomarker Test Has Prognostic and Diagnostic Value, Penn Medicine Team Reports

Perelman School of Medicine researchers at the University of Pennsylvania report the first biomarker results reported from the Parkinson’s Progression Markers Initiative (PPMI), showing that a comprehensive test of protein biomarkers in spinal fluid have prognostic and diagnostic value in early stages of Parkinson’s disease. The study is reported in JAMA Neurology.

Compared to healthy adults, the study found that people with early Parkinson’s had lower levels of amyloid beta, tau and alpha synuclein in their spinal fluid. In addition, those with lower concentrations of tau and alpha synuclein had greater motor dysfunction. And early Parkinson’s patients with low levels of amyloid beta and tau were more likely to be classified as having the postural instability-gait disturbance- dominant (PIGD) motor type of disease, where falling, freezing, and walking difficulty are common.

“Biomarkers for Parkinson’s disease such as these could help us diagnose patients earlier, and we’ve now shown that the simultaneous measurement of a variety of neurodegenerative disease proteins is valuable,” said study senior author Leslie M. Shaw, PhD, professor of Pathology and Laboratory Medicine at Penn Medicine. Dr. Shaw and John Q. Trojanowski, MD, PhD, director of the Penn Udall Center for Parkinson’s Research, are co-leaders of the Bioanalytics Core for the Parkinson’s Progression Markers Initiative, an international observational clinical study sponsored by The Michael J. Fox Foundation for Parkinson’s Research.

The team evaluated spinal fluid collected from baseline visits of the first 102 PPMI participants – 63 with early, untreated Parkinson’s disease and 39 healthy controls. The spinal fluid was evaluated for levels of five biomarkers: amyloid beta, total tau, phosphorylated tau, alpha synuclein and the ratio of total tau to amyloid beta. Spinal fluid measures of amyloid and tau are currently used in research to distinguish Alzheimer’s disease from other neurodegenerative diseases. In contrast to Alzheimer’s, where tau levels are higher than healthy controls, the study found that early Parkinson’s patients had lower levels of tau than healthy controls. One reason, researchers suggest, could be that interactions between tau and alpha synuclein may limit the release of tau into the cerebrospinal fluid of Parkinson’s patients.

“Through PPMI, we are hoping to identify subgroups of Parkinson’s patients whose disease is likely to progress at a different rate, as early as possible,” said Dr. Trojanowski. “Early prediction is critical, for both motor and dementia symptoms.”

The Parkinson’s PIGD motor subtype has been associated with a more rapid cognitive decline as well as greater functional disability. Using the biomarker test, this initial study found that levels of all spinal fluid biomarkers were lower in the PIGD motor subtype than other types of PD as well as healthy controls. In addition, amyloid beta and phosphorylated tau were at lower levels in the PIGD motor subtype, but were no different in tremor or indeterminate subtypes compared to normal controls.

This spinal fluid testing procedure is only being used in research studies, and will be continued to be evaluated and validated in a larger study of the PPMI cohorts.

In addition to leading the Bioanalytics Core of PPMI, Penn’s Parkinson’s Disease and Movement Disorders Center is one of the two dozen trial sites where volunteers are evaluated throughout the PPMI study. The Penn PDMDC has been part of the PPMI group studying people with early Parkinson’s disease as well as healthy adults since 2010, and began enrollment for a new, pre-symptomatic arm of the study in the summer of 2013. The pre-motor arm of PPMI is enrolling participants who do not have Parkinson’s disease and are living with one of three potential risk factors for PD: a reduced sense of smell (hyposmia); rapid eye movement sleep behavior disorder (RBD; a disorder in which the individual acts out his/her dreams); or a mutation in the LRRK2 gene (the single greatest genetic contributor to PD known to date).

“In addition to biomarker tests, validating risk factors could enable earlier detection of the disease and open new avenues in the quest for therapies that could slow or stop disease progression,” said PPMI trial site study leader Matthew Stern, MD, professor of Neurology and director of Penn’s Parkinson’s Disease and Movement Disorders Center.

Study: Association of Cerebrospinal Fluid β-Amyloid 1-42, T-tau, P-tau181, and α-Synuclein Levels With Clinical Features of Drug-Naive Patients With Early Parkinson Disease [JAMA Neurology]

Source: Penn Medicine

University Of California, San Diego School Of Medicine To Investigate Risk Factors For Parkinson’s Disease Via Parkinson’s Progression Markers Initiative (PPMI)

University of California, San Diego School of Medicine will be one of 23 official clinical sites of the Parkinson’s Progression Markers Initiative’s (PPMI) new arm to study at-risk populations for Parkinson’s disease (PD). The $55 million landmark observational clinical study launched in 2010 to define one or more biomarkers of PD and now seeks to better understand potential risk factors of the disease. The University of California, San Diego School of Medicine has been a part of PPMI for three years and is currently enrolling for the new, pre-motor arm of the study.

The pre-motor arm of PPMI will enroll participants who do not have Parkinson’s disease and are living with one of three potential risk factors for PD: a reduced sense of smell (hyposmia); rapid eye movement sleep behavior disorder (RBD); or a mutation in the LRRK2 gene (the single greatest genetic contributor to PD known to date). Validating these risk factors could not only enable earlier detection of the disease, but open new avenues in the quest for therapies that could slow or stop disease progression.

“Understanding risk factors for Parkinson’s disease could help to identify therapies that may prevent the onset of motor symptoms in future generations of PD patients,” said Douglas Galasko, MD, the principal investigator for the study at the University of California, San Diego School of Medicine.

Local residents can easily get involved in this research by being one of 10,000 individuals needed to complete a brief online survey (www.michaeljfox.org/takethesmellsurvey) about their sense of smell. People over the age of 60 who do not have Parkinson’s disease are needed to take the survey. Most respondents will be sent a scratch-and-sniff smell test and brief questionnaire in the mail to be completed at home. Some individuals may also be asked to undergo more extensive testing.

“In the third year of PPMI, it is evident that a large-scale biomarker study is not only possible in Parkinson’s disease, but is already yielding scientific insights that could help transform the field of Parkinson’s research,” said Todd Sherer, PhD, CEO of The Michael J. Fox Foundation for Parkinson’s Research. “None of this progress would be possible without the willing volunteers who donate their time and energy to the pursuit of a cure.”

Source: PR Newswire

UAB Study to Examine Sense of Smell as Risk Factor for Parkinson’s

A reduced sense of smell, or hyposmia, might be a risk factor for developing Parkinson’s disease, and the University of Alabama at Birmingham (UAB) is participating in a study of hyposmia and two other potential risk factors. The study, sponsored by The Michael J. Fox Foundation for Parkinson’s Research, is a new arm of the long-running Parkinson’s Progression Markers Initiative (PPMI), which looks to attract as many as 20,000 people worldwide to participate in a brief online survey about the sense of smell.

The study is looking for people older than 60 who do not have Parkinson’s disease to take the online survey. Most respondents will then be mailed a scratch and sniff smell test and a brief questionnaire, to be completed at home. Some individuals may also be asked to undergo more extensive testing.

The study will also enroll individuals who have rapid eye movement sleep behavior disorder (RBD) or a mutation in the LRRK2 gene, which is the single greatest known genetic contributor to Parkinson’s disease.

“Understanding risk factors for Parkinson’s disease could help to identify therapies that may prevent the onset of motor symptoms in future generations of PD patients,” said David Standaert, M.D., Ph.D., professor and chair of the Department of Neurology at UAB. “UAB is proud to participate in this innovative research and respectfully ask our local community for volunteers for the study.”

UAB has been part of the PPMI study since it was launched in 2010. The now $55 million landmark observational clinical study was established to look for one biomarker of PD or more, and now it seeks to better understand potential risk factors of the disease. UAB is one of 23 participating sites worldwide and is among the top five sites in numbers of participants.

“In the third year of PPMI, it is evident that a large-scale biomarker study is not only possible in Parkinson’s disease, but is already yielding scientific insights that could help transform the field of Parkinson’s research,” said Todd Sherer, CEO of the Michael J. Fox Foundation. “None of this progress would be possible without the willing volunteers who donate their time and energy to the pursuit of a cure.”

Source: University of Alabama at Birmingham