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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

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

Molecular Response Launches TargetXTM Platform for Rapid Discovery & Validation of New Oncology Targets

Molecular Response recently announced the launch of its TargetX platform for rapid discovery and validation of new oncology targets. The program provides partners with access to the world’s largest bank of living tumor specimens, matched genomic database, and in vivo/ex vivo patient derived tumor models for validation. The integrated platform enables investigators to do in days what used to take months.

Target discovery and validation in oncology has largely relied on molecular and functional studies performed in cell lines. Recent advances in genomics have now created large databases based on well-characterized tumor tissue, which has enabled direct investigation of patient tumors for novel targets. Following these discoveries, it is routine to perform functional studies in cell line-based systems; however, it is often challenging to find a relevant cell line model and if found, there are often numerous factors which confound biology when using historical cell lines for functional studies. The result can be a process which takes considerable time and does not readily translate to clinical relevance.

“TargetX is the largest scale genomic database matched to living patient-derived tumor models,” said Dr. Mohit Trikha, CSO of Triphase Accelerator and founder of Drug Design Corp. “We plan to access it for our drug pipeline development and biomarker identification; having everything in one place allows us to do in days what used to take months. Additionally, we can now work with living patient derived tumor samples rather than cultured cell lines.”

The platform relies on Molecular Response’s proprietary bank of more than 144,000 patient derived tumor cells, of which nearly 400 tumors have been genomically characterized and databased for target discovery studies. The database is growing, but currently features the following cancer indications: colon carcinoma, NSCLC, melanoma, ovarian carcinoma, prostate cancer and Non-Hodgkins Lymphoma. Upon discovery of a novel target, tumors of interest are immediately implanted into mice to perform functional studies in direct patient derived models–either in vivo or ex vivo. Molecular Response currently has more than 60 such patient derived xenograft models established for in vivo studies.

“We continue to focus on the use of patient derived models, both in vivo and ex vivo, for advancing oncology drug development,” said Thomas Broudy, CSO of Molecular Response. “Everybody would like to perform studies in the patient derived tumor setting starting as early as possible, but without the resource to do so, it’s nearly impossible. TargetX now enables you to do that.”

Molecular Response presented results from the TargetX platform at the AACR meeting; the company has identified a novel kinase target for potential therapeutic development. They investigated prevalence of target overexpression across 7 cancer indications, and identified melanoma as a clinical indication of high interest. Growth characteristics from patient tumors featuring high kinase gene expression vs. low expression were examined to help characterize the role of this target in oncology disease progression. Functional studies in these patient derived models to further validate the novel kinase are ongoing, as is a small molecule and antibody-based therapeutic development program.

Source: Business Wire

KineMed, Isis and CHDI Collaborate to Advance Therapeutic Development for Huntington’s Disease and Develop Novel Biomarkers for Pre-clinical and Clinical Use in Drug Development and Therapeutic Monitoring

KineMed, Inc., Isis Pharmaceuticals Inc. (NASDAQ: ISIS), and CHDI Foundation, Inc., announced today that they are collaborating to utilize KineMed’s translational biomarker platform with Isis’ antisense therapeutic program for Huntington’s disease (HD). This collaboration, which builds on an earlier alliance between CHDI and KineMed to develop companion biomarkers of therapeutic response in HD, will provide Isis access to novel biomarkers for use in the development of an antisense drug to treat HD.

“A pharmacodynamic biomarker could be an important contribution to our clinical development efforts, and we look forward to working with KineMed and CHDI to evaluate KineMed’s biomarker platform in our Huntington’s disease program,” said Frank Bennett, Ph.D., Senior Vice President of Research at Isis.

“This companion biomarker partnership represents personalized medicine in action,” said Patrizia Fanara, Ph.D., Vice President of Neuroscience KineMed. “Our neurodegeneration-specific biomarkers, which provide dynamic measures of axonal transport deficits in degenerating brain cells, predict therapeutic response in neurological and neuromuscular disorders. This is a translational biomarker that could prove crucial to the advancement of disease-modifying treatments. With the Huntington’s disease domain knowledge that CHDI brings and the therapeutic approach that Isis is pioneering, this partnership has the potential to develop biomarkers for specific therapeutics for Huntington’s disease and lead to personalized medicines for Huntington’s patients.”

“There is a critical need to identify appropriate biomarkers to determine target engagement and predict early clinical efficacy for future Huntington’s disease clinical trials,” said Jonathan Bard, Ph.D., Director of Molecular Pharmacology for CHDI Foundation. “Combining Isis’ knowledge of antisense therapies with KineMed’s expertise in developing unique pharmacodynamic biomarkers creates a collaboration with great potential for discovering such tools for Huntington’s disease.”

KineMed’s neurodegeneration biomarker of axonal transport deficit has been recently published in the Journal of Clinical Investigation1 and validated in patients with other neurological disorders.

Source: Business Wire

Ballenger Trust Selects the Myelin Repair Foundation to Advance Myelin Repair Therapeutic Development for Multiple Sclerosis

The Myelin Repair Foundation (MRF) recently announced the support of the Ballenger Trust to accelerate the development of myelin repair therapeutics for multiple sclerosis (MS). The $700,000 gift will provide support for the MRF biomarker project, myelin repair research in human brain tissue and a repurposing opportunity. With the Ballenger Trust’s support, these critical projects will accelerate the Myelin Repair Foundation’s research to develop a myelin repair therapeutic for multiple sclerosis.