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The Michael J. Fox Foundation Launches New Arm Of Parkinson’s Progression Markers Initiative Studying At-Risk Populations In Parkinson’s Disease

The Parkinson’s Progression Markers Initiative (PPMI), a landmark biomarker clinical study, has completed enrollment of its initial 600-member cohort of Parkinson’s patients and controls, and will launch additional study cohorts to leverage the existing PPMI infrastructure and evaluate multiple potential biomarkers for Parkinson’s disease (PD). The first of these new cohorts launches today and will investigate risk factors for PD that may enable diagnosis before the onset of motor symptoms.

The pre-motor arm of PPMI will enroll participants who do not have Parkinson’s disease but do have 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). Research to date indicates that each of these factors can be linked to an increased risk of developing Parkinson’s disease, though many people with these conditions do not go on to develop PD. Validating these risk factors and better characterizing their connection to Parkinson’s could enable detection of the disease prior to the onset of motor symptoms and open new avenues toward identifying biomarkers — critical tools in the quest for therapies that can slow or stop disease progression.

“If scientists can learn more about the biological processes taking place in people with any of these three risk factors, we may be able to define biomarkers even before typical symptoms begin,” said Ken Marek , MD, principal investigator of PPMI and president and senior scientist at the Institute for Neurodegenerative Disorders in New Haven, Connecticut. “Finding a biomarker for PD could mean earlier diagnosis of the disease and lead to new drugs that may delay or even prevent the onset of motor symptoms.”

PPMI seeks 10,000 individuals to complete a brief online survey to determine eligibility for the loss-of-smell cohort. Participants in the other groups will largely be enrolled via research centers. This new arm will take place at 23 sites across the world where participants will undergo the same clinical assessments, imaging and collection of biospecimens as the original study.

PPMI’s open-source design and infrastructure has opened the door to evaluating multiple potential biomarkers under one umbrella, building on a precedent created by the Alzheimer’s Disease Neuroimaging Initiative (ADNI). All PPMI clinical data and characterized biosamples are available in real time, providing researchers around the world with an unprecedented resource to help speed and unify disparate biomarker validation studies. To date, 460 scientists from academia and industry have downloaded PPMI data more than 50,000 times in over 30 countries worldwide, and 21 applications have been made for use of PPMI biospecimens in biomarker research. Initial baseline data from PPMI’s original newly diagnosed and control cohorts will be presented this June at the Movement Disorders Society and is expected to be published later this year.

“Lessons learned from Alzheimer’s have taught us the importance of pursuing biomarker research concurrent with therapeutic development,” said Todd Sherer , Ph.D., CEO of The Michael J. Fox Foundation for Parkinson’s Research. “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’s pursuit of a cure.”

Source: PR Newswire

Nodality, Inc. Announces Expansion of Company’s Patent Estate Covering Programs to Improve the Treatment of Patients with Severe Diseases in Immunology, Oncology, and Autoimmune Diseases

Nodality, Inc., a next-generation life sciences company partnering with leading firms to advance precision medicine, announced today the issuance by the United States Patent and Trademark Office (USPTO) of Patent Number 8,394,599, which broadly covers methods for applying the Company’s Single Cell Network Profiling (SCNP) technology. SCNP provides the core foundation for Nodality’s programs dedicated to making medicines better by increasing the productivity of therapeutic R&D programs, enhancing life cycle management for commercialized drugs, and introducing new predictive diagnostics. With the issuance of this patent, Nodality now owns or has exclusive rights to more than 25 issued patents and 80 patent applications.

Laura Brege, President and Chief Executive Officer, commented, “The new patent extends and complements Nodality’s other foundational patents covering SCNP, a uniquely powerful tool for correlating clinical data with functional biology. The patent provides further protection for unique biomarkers of protein activation and cell function in single cells and, importantly, applications of this information in clinical management and therapeutic product development. Through our growing patent estate and partnered programs, Nodality is in a key position to improve the treatment of patients with severe diseases, including in immunology, oncology, and autoimmune diseases.”

Source: Nodality

The Michael J. Fox Foundation Launches New Arm of Parkinson’s Progression Markers Initiative Studying At-Risk Populations in Parkinson’s Disease

The Parkinson’s Progression Markers Initiative (PPMI), a landmark biomarker clinical study, has completed enrollment of its initial 600-member cohort of Parkinson’s patients and controls, and will launch additional study cohorts to leverage the existing PPMI infrastructure and evaluate multiple potential biomarkers for Parkinson’s disease (PD). The first of these new cohorts launches today and will investigate risk factors for PD that may enable diagnosis before the onset of motor symptoms.

The pre-motor arm of PPMI will enroll participants who do not have Parkinson’s disease but do have 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). Research to date indicates that each of these factors can be linked to an increased risk of developing Parkinson’s disease, though many people with these conditions do not go on to develop PD. Validating these risk factors and better characterizing their connection to Parkinson’s could enable detection of the disease prior to the onset of motor symptoms and open new avenues toward identifying biomarkers — critical tools in the quest for therapies that can slow or stop disease progression.

“If scientists can learn more about the biological processes taking place in people with any of these three risk factors, we may be able to define biomarkers even before typical symptoms begin,” said Ken Marek, MD, principal investigator of PPMI and president and senior scientist at the Institute for Neurodegenerative Disorders in New Haven, Connecticut. “Finding a biomarker for PD could mean earlier diagnosis of the disease and lead to new drugs that may delay or even prevent the onset of motor symptoms.”

PPMI seeks 10,000 individuals to complete a brief online survey to determine eligibility for the loss-of-smell cohort. Participants in the other groups will largely be enrolled via research centers. This new arm will take place at 23 sites across the world where participants will undergo the same clinical assessments, imaging and collection of biospecimens as the original study.

PPMI’s open-source design and infrastructure has opened the door to evaluating multiple potential biomarkers under one umbrella, building on a precedent created by the Alzheimer’s Disease Neuroimaging Initiative (ADNI). All PPMI clinical data and characterized biosamples are available in real time, providing researchers around the world with an unprecedented resource to help speed and unify disparate biomarker validation studies. To date, 460 scientists from academia and industry have downloaded PPMI data more than 50,000 times in over 30 countries worldwide, and 21 applications have been made for use of PPMI biospecimens in biomarker research. Initial baseline data from PPMI’s original newly diagnosed and control cohorts will be presented this June at the Movement Disorders Society and is expected to be published later this year.

“Lessons learned from Alzheimer’s have taught us the importance of pursuing biomarker research concurrent with therapeutic development,” said Todd Sherer, PhD, CEO of The Michael J. Fox Foundation for Parkinson’s Research. “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’s pursuit of a cure.”

Source: Michael J. Fox Foundation

Precision Therapeutics Announces Topline Prospective Clinical Data Demonstrating Significant Improvement in Overall Survival and Progression Free Survival in Recurrent Ovarian Cancer using Personalized Chemotherapy Diagnostic ChemoFx®

Precision Therapeutics, Inc., a life science company that develops and markets personalized medicine products for individualizing cancer care, recently announced results from a nine-year, multi-center, blinded prospective clinical study which demonstrated statistically significant improvement in overall survival and progression free survival in 262 patients with recurrent ovarian cancer whose tumors were tested with ChemoFx®, a personalized chemotherapy diagnostic.

Recurrent ovarian cancer patients in the clinical study who were treated with a chemotherapy drug identified to be responsive by the ChemoFx assay experienced a statistically significant improvement of 50% in overall survival, versus those patients treated by drugs classified as resistant by ChemoFx. The study also showed statistically significant improvement in progression free survival. In the clinical study, ChemoFx was able to identify at least one sensitive chemotherapy drug for more than half of the recurrent ovarian cancer patients studied, approximately doubling the statistics that show only 25 to 30 percent of cancer patients with recurrent ovarian cancer benefit from a chemotherapy treatment chosen empirically.1 These topline results were presented at a medical symposium and in a poster session held during the Society of Gynecologic Oncology (SGO) Annual Meeting on March 9-12, 2013 in Los Angeles, California.

“This clinical study is a landmark for the treatment of ovarian cancer because it is the first prospective data that definitively shows that a personalized diagnostic test can make a significant clinical impact by improving overall survival by 50% in women with this devastating cancer,” said Thomas J. Rutherford MD, PhD, Professor of ObGyn and Reproductive Sciences and Section Chief, Gynecologic Oncology at the Yale School of Medicine, and lead investigator in the ChemoFx clinical study.

“We see ChemoFx as an example of the power of personalized cancer diagnostics that put innovative technology in the hands of physicians to help inform treatment decisions, with the ultimate goal of improving patient outcomes,” said Sean McDonald, President and CEO of Precision Therapeutics. “We are excited to bring our late-breaking data to share with the treatment community at the SGO meeting, and look forward bringing ChemoFx to all gynecologic patients through the 1,000 board certified treating gynecologic oncologists in the US. We believe this test will have a profound impact on the treatment of this dreadful disease.”

Patients in the study’s treatment group had their cancer cells tested with ChemoFx to assess tumor response among 12 to 15 clinically equivalent chemotherapy options that National Comprehensive Cancer Network (NCCN) cancer guidelines recommend for persistent or recurrent ovarian cancer. ChemoFx was confirmed as an independent predictive factor for progression free survival and overall survival per multivariate Cox proportional hazards analysis. The company expects to publish comprehensive data from the clinical study in the near future.

“The results of this prospective study suggest that ChemoFx may serve as an important tool in selecting more effective drugs to improve the outcome of recurrent ovarian cancer patients,” said John Chan, MD, Associate Professor, Division of Gynecologic Oncology, University of California at San Francisco, Helen Diller Family Comprehensive Cancer Center. “This late breaking data on the utility of assay-directed treatment with ChemoFx will move us forward toward more personalized treatment for ovarian cancer.”

“With a devastating cancer, like ovarian cancer, which has an extremely high recurrence rate in 70% to 90% of patients, it is a groundbreaking moment when there is new clinical data, like these data with ChemoFx, showing that we have a chance to dramatically improve treatment for many patients,” said Thomas Krivak, MD, Associate Professor, University of Pittsburgh Physicians Magee Gynecologic Cancer Program, and an investigator in the ChemoFx clinical study.

“We have seen modest improvements in long-term survival for women with ovarian cancer in the past two decades, despite the availability of several new drugs and treatment strategies.

The data from this multi-institution, blinded observational clinical trial with ChemoFx is a highly significant advancement in understanding how the biology of each patient’s tumor may hold answers for treatments that could result in improvements in survival beyond what we are seeing currently,” said Robert Holloway, MD, Medical Director of Florida Hospital Gynecologic Oncology. “Furthermore, the significance of this study from a clinical standpoint is that this test may have predictive abilities, enabling a physician to choose the most effective pharmaceutical treatment from among the available options for ovarian cancer.”

Source: Business Wire

New Effort to Identify Parkinson’s Biomarkers

Last month, the National Institutes of Health announced a new collaborative initiative that aims to accelerate the search for biomarkers — changes in the body that can be used to predict, diagnose or monitor a disease — in Parkinson’s disease, in part by improving collaboration among researchers and helping patients get involved in clinical studies. As part of this program, launched by the National Institute of Neurological Disorders and Stroke (NINDS), part of the NIH, Clemens Scherzer, MD, a neurologist and researcher at Brigham and Women’s Hospital (BWH), was awarded $2.6 million over five years to work on the development of biomarkers and facilitate NINDS-wide access to one of the largest data and biospecimens bank in the world for Parkinson’s available at BWH. This NINIDS initiative is highlighted in an editorial in the March issue of Lancet Neurology.

“There is a critical gap in the research that leads to lack of treatment for diseases like Parkinson’s,” said Scherzer. “Biomarkers are desperately needed to make clinical trials more efficient, less expensive and to monitor disease and treatment response. We are hopeful that this initiative will fast track new discoveries in this area.”

According to Scherzer, most of our knowledge of the human brain is based on the analysis of just 1.5 percent of the human genome that encodes proteins. The first part of Scherzer’s project will examine the function of the remaining 98.5 percent of the genome that, so far, has been unexplored in the human brain. While this remainder had been previously dismissed as “junk”, it is now becoming clearer that parts of it actively regulate cell biology. Scherzer and colleagues believe that “dark matter” RNA transcribed from stretches of so called “junk” DNA is active in brain cells and contributes to the complexity of normal dopamine neurons and, when corrupted, Parkinson’s disease.

“This offers a potentially ground breaking opportunity for biomarker development. Initially, the team will search for these RNAs associated in brain tissue of individuals at earliest stages of the disease. Then, this team will look for related biomarkers in the bloodstream and cerebrospinal fluid in both healthy brains and those with Parkinson’s,” Scherzer said.

Scherzer’s lab has been spearheading biomarker research in this field since 2004 and the team already has 2,000 patients enrolled and being followed in a longitudinal study with rich clinical data and one of the largest biobanks in the world for Parkinson’s tissue with support from the Harvard NeuroDiscovery Center. The biobank was designed as an incubator for Parkinson’s research and until now was chiefly available for research collaborations within the Harvard-affiliated community. As part of this new project, this vast resource will be open to all NIH-funded investigators.

“Our ultimate goal is to personalize treatment for our patients with Parkinson’s.” said Scherzer. “By opening up this vast collection of specimens, we are exploding the resources that are available to NIH-funded investigators looking at this disease. We hope to harness the power of collaboration to speed up biomarkers discovery.”

Study: NINDS boosts research for biomarkers in Parkinson’s disease

Source: EurekAlert!