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I copy news articles pertaining to research, news and information for Parkinson's disease, Dementia, the Brain, Depression and Parkinson's with Dystonia. I also post about Fundraising for Parkinson's disease and events. I try to be up-to-date as possible. I have Parkinson's diseases as well and thought it would be nice to have a place where updated news is in one place. That is why I began this blog.
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Saturday, March 28, 2015
Friday, March 27, 2015
When Rice University chemist Matteo Pasquali set out to to create strong and conductive carbon nanotube fibers, he had aerospace applications in mind. But it turned out his microscopic fibers are also great at communicating with the brain, making them an ideal candidate for therapies that deal with neurological disorders such as Parkinson's disease. Pasquali said: "...once we [he and his team] had them in our hand, we realized that they had an unexpected property: They are really soft, much like a thread of silk. Their unique combination of strength, conductivity and softness makes them ideal for interfacing with the electrical function of the human body."
At the moment, hard metal electrodes are implanted into the brain for Parkinson's therapy (they deliver electrical signals to calm tremors), but they're not actually that compatible with the organ's soft tissues. These flexible fibers are more biocompatible -- they're also cheaper and maintain better electrical connection. Plus, the scientists' tests prove they cause little inflammation and are as stable as commercial platinum used on electrodes.
Rice U assistant professor Caleb Kemere who studies Parkinson's disease believes these fibers could lead to self-regulating treatment devices for patients. Those devices will be able to read signals from the brain, analyze the best amount of electrical stimulation needed to calm tremors on a case-by-case basis and automatically administer jolts of electricity. That's the gist of it anyway: if you want to read the team's study in greater detail, bust out your science jargon decoder and check out the paper on ACS Nano
Thursday, March 26, 2015
Tuesday, March 24, 2015
SOURCE The Michael J. Fox Foundation
PPMI now recruiting individuals with genetic mutation linked to Gaucher disease
NEW YORK, March 24, 2015 /PRNewswire-USNewswire/ -- The Parkinson's Progression Markers Initiative (PPMI), a large-scale biomarker study sponsored by The Michael J. Fox Foundation for Parkinson's Research (MJFF), is expanding to study individuals with a mutation of the GBA (glucosidase beta acid) gene. Participants will include people with or at increased risk to develop Parkinson's disease. Researchers hope that a greater understanding of the biology and clinical features of these participants will lead to therapies benefiting all Parkinson's patients and ultimately provide strategies to prevent disease onset.
"Insights gleaned from volunteers with genetic mutations will help speed research toward new and improved Parkinson's therapies, benefitting the greater Parkinson's community," said Ken Marek, MD, principal investigator of PPMI and president and senior scientist at the Institute for Neurodegenerative Disorders in New Haven, Connecticut.
Mutations in the GBA gene can cause Gaucher disease, a disorder where fatty substances accumulate in body cells. First linked to Parkinson's in 2008, these genetic mutations lower levels of an enzyme that helps break down proteins and lipids. In Parkinson's disease (PD) lower levels of this enzyme are associated with aggregation of the protein alpha-synuclein, the hallmark pathology of PD.
Genetics Provides Greater Understanding of Disease
Understanding the differences among people with and without PD who carry mutations such as in the GBA gene may help researchers understand what leads to disease onset and progression. In 2014 PPMI expanded to enroll individuals with a mutation of the LRRK2 or SNCA gene, the two targets of greatest interest in Parkinson's drug development.
One key tool missing from the development pipeline is an accurate way to measure progression of Parkinson's disease: a biomarker. Biomarkers would allow researchers to quickly and objectively measure a therapy's ability to slow, halt or reverse the Parkinson's process. PPMI is studying clinical and imaging data and biological samples of people with a genetic mutation to identify biomarkers, ultimately speeding clinical trials. In addition, when a drug targeting LRRK2, SNCA or GBA is ready for clinical testing, PPMI will have assembled a group of people with these mutations who stand ready to participate in studies.
PPMI will enroll 125 people with the GBA mutation and who have Parkinson's and 125 people with the mutation who do not have Parkinson's. These participants will be followed for five years. By collecting data and samples over time from people who have not been diagnosed with the disease but who carry an associated genetic mutation, researchers can test for characteristics that may denote greater risk of disease onset or, conversely, protection from symptoms.
Known genetic mutations currently account for only five to 10 percent of all Parkinson's cases. The vast majority of Parkinson's cases are idiopathic, meaning researchers do not know what causes the disease. However, study of individuals carrying PD-implicated genetic mutations may reveal disease traits that apply to all PD patients. The PPMI genetic cohort will expand understanding of the pathogenesis of both genetic and idiopathic Parkinson's disease.
Interested individuals can visit www.michaeljfox.org/ppmi/genetics. Similar to the LRRK2 mutation, the GBA mutation accounts for a greater number of PD cases among certain ethnic populations and families, notably those of Ashkenazi (Eastern European) Jewish descent. PPMI is particularly interested in testing individuals of this background who have Parkinson's or a close relative with the disease.
PPMI: The Search for Parkinson's Biomarkers
Biomarkers - such as cholesterol level for heart disease - are substances, processes or characteristics of the body that indicate disease risk, onset and/or progression. They aid in diagnosis and disease management and help researchers stratify for clinical trials and test new drugs faster by measuring biological changes rather than waiting for clinical improvement. There are no validated biomarkers for Parkinson's disease, a reality researchers are hoping to change with PPMI.
Launched in 2010, PPMI is a longitudinal clinical study that collects standardized clinical, imaging and biologic data. Now taking place at 33 clinical sites around the world, the study completed initial enrollment of 423 recently diagnosed Parkinson's patients and 196 controls in April 2013. Since then, the study has expanded to include cohorts of individuals at increased risk of developing Parkinson's disease. Risk factors under study include genetics, REM sleep behavior disorder and smell loss. (Enrollment of the smell loss cohort was completed earlier this year.)
"By studying multiple cohorts, we're covering all bases to find and validate these biomarkers," said MJFF CEO Todd Sherer, PhD. "Expanding PPMI to include another genetic cohort allows science to push forward toward better therapies for people living with Parkinson's today and for those who may be at risk in the future."
About The Michael J. Fox Foundation for Parkinson's Research As the world's largest nonprofit funder of Parkinson's research, The Michael J. Fox Foundation is dedicated to accelerating a cure for Parkinson's disease and improved therapies for those living with the condition today. The Foundation pursues its goals through an aggressively funded, highly targeted research program coupled with active global engagement of scientists, Parkinson's patients, business leaders, clinical trial participants, donors and volunteers. In addition to funding more than $450 million in research to date, the Foundation has fundamentally altered the trajectory of progress toward a cure. Operating at the hub of worldwide Parkinson's research, the Foundation forges groundbreaking collaborations with industry leaders, academic scientists and government research funders; increases the flow of participants into Parkinson's disease clinical trials with its online tool, Fox Trial Finder; promotes Parkinson's awareness through high-profile advocacy, events and outreach; and coordinates the grassroots involvement of thousands of Team Fox members around the world.
http://health.einnews.com/article/256452174/CuekJjsDjrrpx3GITo view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/parkinsons-biomarker-initiative-expands-genetic-study-300054418.html
Results announced from Phase 1 single ascending dose study of PRX002, a monoclonal antibody for the potential treatment of Parkinson's disease
Last updated: Tuesday 24 March 2015 at 2am PST
Prothena Corporation plc, a late-stage clinical biotechnology company focused on the discovery, development and commercialization of novel protein immunotherapy programs, has announced positive results from a Phase 1 single ascending dose study of PRX002, a monoclonal antibody for the potential treatment of Parkinson's disease and other related synucleinopathies. PRX002 is the focus of a worldwide collaboration between Prothena and Roche.
PRX002 was safe and well-tolerated, meeting the primary objective of the study. Further, results from this study showed that administration of PRX002 leads to mean reduction of free serum alpha-synuclein levels of up to 96%. These overall results were highly statistically significant (p<0.00001). Reduction of free serum alpha-synuclein, a protein potentially involved in the onset and progression of Parkinson's disease and the target of PRX002, was shown to be robust, rapid and dose-dependent after just a single dose.
"There is genetic and pathological evidence that supports a causal role of alpha-synuclein in Parkinson's disease," said Todd Sherer, PhD, CEO of the Michael J. Fox Foundation for Parkinson's Research. "We applaud Prothena and Roche for their pioneering work in developing a potentially disease-modifying therapy for this progressive neurodegenerative disease that affects millions worldwide."
The Phase 1 double-blind, placebo-controlled, single ascending dose study enrolled 40 healthy volunteers. All volunteers enrolled were randomized 3:1 into five escalating dose cohorts (0.3 mg/kg, 1 mg/kg, 3 mg/kg, 10 mg/kg or 30 mg/kg) to receive either PRX002 or placebo. No hypersensitivity reactions or drug-related serious adverse events were reported. PRX002 demonstrated favorable pharmacokinetic properties, supporting the current dosing frequency in the on-going Phase 1 multiple ascending dose study in patients with Parkinson's disease. There were no treatment emergent adverse events (TEAEs) in greater than 10% of subjects. The only TEAEs in greater than 5% of subjects were vessel puncture site pain, headache and viral infection. All PRX002-related adverse events were mild and no dose limiting toxicities were observed.
"We are extremely pleased with the results of the Phase 1 single ascending dose study as the mean reduction of free serum alpha-synuclein of up to 96% demonstrates the pharmacodynamic effects of PRX002," commented Gene Kinney, PhD, Chief Scientific Officer and Head of Research and Development at Prothena. "Importantly and for the first time in humans, we demonstrated that this robust, rapid and dose-dependent reduction of free serum alpha-synuclein was safe and well-tolerated. Thus, this approach may translate into a clinically meaningful delay or reversal of disease progression in patients with Parkinson's disease. We look forward to building upon these data with results from the on-going, multiple ascending dose study in patients with Parkinson's disease expected in the first half of 2016, where we will also be measuring levels of PRX002 in the cerebrospinal fluid and assessing additional biochemical, imaging and clinical biomarker endpoints. Separately, we are excited to co-host a symposium with Roche on March 21 at the 12th International Conference on Alzheimer's and Parkinson's Diseases and Related Neurological Disorders (AD/PD™ 2015) to continue to raise awareness of the role of alpha-synuclein as a target for Parkinson's disease."
"The results of the PRX002 study exemplify Prothena's deep domain expertise to develop novel disease-modifying protein immunotherapies with unique specificities to their targets," stated Dale Schenk, PhD, President and Chief Executive Officer of Prothena. "Prothena's consistent ability to develop targeted potential therapeutics has resulted in a strong and promising pipeline to transform patient's lives, with NEOD001 in Phase 3 clinical studies for the treatment of AL amyloidosis, PRX002 continuing in a Phase 1 multiple ascending dose study in patients with Parkinson's disease and PRX003 ready to begin clinical studies for the treatment of psoriasis and potentially other inflammatory diseases."
In December 2013, Prothena and Roche entered into a worldwide collaboration to develop and commercialize antibodies that target alpha-synuclein, including PRX002. To date, Prothena has received $45 million of the potential $600 million in total milestones through its collaboration with Roche. Prothena has an option to co-promote PRX002 in the U.S., where the companies share all profits, as well as development and commercialization costs, on a 30/70 basis (30% Prothena and 70% Roche). Outside the U.S., Roche will have sole responsibility for developing and commercializing PRX002 and will pay Prothena up to double-digit royalties on net sales.
Adapted by MNT from original media release