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First Test to Objectively Diagnose Fibromyalgia Now Available

A recent peer-reviewed study, published in BMC Clinical Pathology,[1] reveals a medical breakthrough discovering multiple biomarkers based upon highly sensitive and reproducible medical investigations. Conducted by the University of Illinois College of Medicine at Chicago (UIC) and EpicGenetics, a privately-held biomedical company, the research has led to the development of The FM/a® Test (www.thefmtest.com), the first test to objectively diagnose fibromyalgia.

Researchers at the UIC Department of Pathology conducted a statistically significant study involving more than 200 patients, comparing those clinically diagnosed with fibromyalgia to healthy patients. The study revealed that patients with fibromyalgia have a dysregulation disorder affecting protein molecules called chemokines and cytokines, produced by white blood cells. While fibromyalgia patients have been classified to be hyperactive (or overactive) responders, the study showed that people with fibromyalgia have immune production patterns which may make them more vulnerable to stress, thereby leading to chronic pain, severe fatigue, diffuse muscle tenderness, insomnia, and other unbearable symptoms long associated with fibromyalgia. Evaluation of The FM Test patient results will provide a continuing database that could lead to further insight into what may cause and exacerbate fibromyalgia and permit the development of standards to determine effective treatments.

EpicGenetics worked with fibromyalgia support groups to recruit participants in the clinical research study and is collaborating with groups such as the National Fibromyalgia & Chronic Pain Association to educate the fibromyalgia community and others about the availability of The FM Test.

“The results of our research have allowed us to ‘pull back the curtain’ and identify specific diagnostic biomarkers in fibromyalgia,” said Bruce S. Gillis, MD, MPH, member of the clinical faculty at the UIC College of Medicine and founder of EpicGenetics. “For decades, the medical community has viewed fibromyalgia with much skepticism. Patients have been stigmatized, and many are spending thousands of dollars and years of frustration in search of a diagnosis. Our breakthrough provides patients with hope and validation that their symptoms are real, and we hope physicians who commonly see patients with fibromyalgia will embrace and value this test for its role in the advancement of medical care.”

According to the American College of Rheumatology, fibromyalgia affects more than 12.3 million people in the United States, comparable to the number of people affected by cancer. Patients who experience symptoms, such as chronic pain and fatigue, depression and insomnia, spend an average of three to five painful years seeking a diagnosis and $4,800-$9,300 annually on associated medical costs. Until now, there has been no conclusive test to confirm the diagnosis of fibromyalgia.

The FM Test, a simple blood test with more than 93 percent sensitivity, now makes it possible for anyone to find out if they have fibromyalgia. The FM Test costs $744 with conclusive results available to most patients usually in a week or less – a fraction of the time and money the average patient currently spends seeking a diagnosis.

By completing a simple symptoms questionnaire at www.thefmtest.com, patients and their doctors can find out if they qualify for the test. If a patient meets the requirements, they can arrange for the test at their doctor’s office or at an independent blood draw facility.

“The elegantly designed study by Dr. Gillis and his co-investigators represents a milestone on the path our group charted 25 years ago when we first hypothesized that cytokines play a role in fibromyalgia2. It is hoped that this and future work sponsored by EpicGenetics will lead to a greater understanding of how the immune system, fatigue, sleep disorders, chronic stress and pain interact in patients with fibromyalgia and related disorders,” said Daniel J. Wallace MD, FACP, FACR, a clinical professor of medicine at the David Geffen School of Medicine at UCLA based at Cedars-Sinai Medical Center, and a member of the Scientific Advisory Board of EpicGenetics.

Source: Business Wire

The Medicines Company and Alnylam Form Strategic Alliance to Develop and Commercialize RNAi Therapeutics Targeting PCSK9 for the Treatment of Hypercholesterolemia

The Medicines Company (Nasdaq: MDCO) and Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, recently announced that they have formed an exclusive global alliance for the development and commercialization of Alnylam’s ALN-PCS RNAi therapeutic program for the treatment of hypercholesterolemia.

“This new alliance unites two organizations with a shared culture and commitment to innovation. In my view and past experience, there could be no stronger partner for our ALN-PCS program than The Medicines Company, which has demonstrated industry-wide leadership in the advancement of cardiovascular medicines to patients and remarkable success in its strategy of in-licensing, developing, and commercializing breakthrough products,” said John Maraganore, Ph.D., Chief Executive Officer of Alnylam. “For Alnylam, this new partnership enables the advancement of ALN-PCS, an important program within our ‘Alnylam 5×15’ product development and commercialization strategy focused on RNAi therapeutics directed toward genetically validated targets. We believe that the ALN-PCS program holds great promise for the development of a significant therapeutic option for patients with hypercholesterolemia, and that the unique mechanism of action for ALN-PCS could provide a differentiated and potentially best-in-class strategy for PCSK9 antagonism.”

“Our focus on acute and intensive care medicine has led us to a leadership position with Angiomax® (bivalirudin) and potentially with cangrelor in the management of patients in extreme risk as a consequence of the rupture of their vulnerable coronary artery plaque at and around the time of acute coronary syndromes. Meantime, we have made progress with MDCO-216 (ApoA-1 Milano), a turbocharged form of HDL-C (‘good cholesterol’) which has the potential to modify disease through reverse cholesterol transport,” said Clive Meanwell, M.D., Ph.D., Chairman and Chief Executive Officer of The Medicines Company. “Now, this exciting collaboration with Alnylam – leaders in their field of RNAi – adds a second potentially disease modifying approach and more cutting edge technology to our portfolio. We have seen that PCSK9 gene silencing can substantially reduce LDL-cholesterol in patients and has epidemiological and disease mechanisms studies suggest this can further reduce the risks of the world’s number one killer, coronary artery disease. Clearly we see the complementarity of approaches which increase ‘good cholesterol’ (HDL-C) and decrease ‘bad cholesterol’ (LDL-C). We look forward to working with our colleagues at Alnylam for whom we have the greatest respect and admiration based upon earlier collaborations particularly around Angiomax, which was invented by John Maraganore.”

PCSK9 (proprotein convertase subtilisin/kexin type 9) is a protein that regulates low-density lipoprotein (LDL) receptor levels on hepatocytes; gain-of-function human mutations in PCSK9 are associated with hypercholesterolemia while loss-of-function mutations are associated with lower levels of LDL cholesterol and a reduced risk of cardiovascular disease. ALN-PCS is a PCSK9 synthesis inhibitor that reduces intracellular and extracellular levels of PCSK9 resulting in lowered plasma levels of LDL-C. MDCO-216 is a naturally occurring variant of a protein found in high-density lipoprotein, or HDL. It is a reverse cholesterol transport agent designed to reduce atherosclerotic plaque burden development and thereby reduce the risk of adverse thrombotic events.

Under this alliance, The Medicines Company and Alnylam intend to collaborate on the advancement of the ALN-PCS program. Alnylam’s ALN-PCS program includes ALN-PCS02 – an intravenously administered RNAi therapeutic which has completed a Phase I trial, and ALN-PCSsc – a subcutaneously administered RNAi therapeutic currently in pre-clinical development. Alnylam will continue the program for an estimated one to two years to complete certain pre-clinical and Phase I clinical studies. The Medicines Company is responsible for leading and funding development from Phase II forward and commercializing the ALN-PCS program if successful. Under the terms of the agreement, The Medicines Company will make an upfront cash payment of $25 million to Alnylam. Alnylam may also receive potential development and commercial milestone payments of up to $180 million. Alnylam will be eligible to receive scaled double-digit royalties on global products sales of ALN-PCS products.

Alnylam has completed a Phase I trial of ALN-PCS02 in healthy volunteer subjects with elevated baseline LDL-C. Results showed that administration of a single intravenous dose of drug, in the absence of concomitant lipid-lowering agents such as statins, resulted in statistically significant and durable reductions of PCSK9 plasma levels of up to 84% and lowering of LDL-C of up to 50%. ALN-PCS02 was shown to be generally safe and well tolerated in this study and there were no serious adverse events related to study drug administration. Alnylam has also presented pre-clinical data from its ALN-PCSsc program demonstrating potent knockdown of the PCSK9 target gene with an ED50 of less than 0.3 mg/kg after a single subcutaneous dose.

“Cardiovascular disease remains the leading cause of mortality worldwide, with elevated LDL-C a major modifiable risk factor. New strategies are needed to dramatically and rapidly reduce LDL-C and prevent acute cardiovascular events that result from the rupture of cholesterol rich plaque when patients are at their most vulnerable,” said Daniel J. Rader, M.D., professor of Medicine and chief, Division of Translational Medicine and Human Genetics, at the Perelman School of Medicine at the University of Pennsylvania. “As a key regulator of the LDL receptor, liver-expressed PCSK9 is one of the most important and best validated new targets in molecular medicine for the treatment of hypercholesterolemia. The ALN-PCS data generated to date are very encouraging and I look forward to continued clinical studies that highlight the unique mechanistic approach of PCSK9 synthesis inhibitors.”

Dr. Rader serves as a member of Alnylam’s Scientific Advisory Board and as a consultant on Alnylam’s ALN-PCS program, and Alnylam and Dr. Rader collaborate on research for which Alnylam provides materials.

Source: The Medicines Company

Verastem Enters Biomarker Agreement with LabCorp for Cancer Stem Cell Agent Companion Diagnostic

Verastem, Inc., (NASDAQ: VSTM) a clinical-stage biopharmaceutical company focused on discovering and developing drugs to treat cancer by the targeted killing of cancer stem cells, entered an agreement with Laboratory Corporation of America® Holdings (LabCorp®) (NYSE: LH) to validate biomarkers for its lead focal adhesion kinase (FAK) inhibitor VS-6063 in the development of an applicable companion diagnostic.

Parkinson’s Brain Chemistry Changes Now Trackable in Man

KineMed, Inc. announced today the publication of a novel discovery in biomarkers for neurodegenerative diseases from a study funded by The Michael J. Fox Foundation. The absence of meaningful biomarkers has remained a roadblock in the development and clinical application of treatments for neurological disorders. The publication describes in detail a unique class of biomarkers that measure the transport efficiency of key cargo molecules through neurons in the living human brain. Biomarkers of this pathogenically causal process may be used for the development of drugs to treat Parkinson’s disease.

SCan-­Mark Explorer V1 Launched to Accelerate Cancer Biomarker Discovery and Support Personalized Medicine

After 2 years of software product development funded by the National Cancer Institute (NCI) SBIR Program, Sophic recently announced the launch of the Sophic Cancer Biomarker Knowledge Environment, SCan‐MarK Explorer V1. The SCan-­MarK Explorer software is intended to accelerate research, drive drug discovery, support translational medicine, improve the accuracy of disease diagnosis, increase the effectiveness of treatments and support finding cures for cancer. SCan­‐MarK Explorer combines a growing database of manually curated breast, ovarian, colon, non-­Hodgkin’s lymphoma and melanoma biomarker targets with the power of BioXM, an integrated knowledge management system. Throughout the development project, Sophic collaborated with and had the benefit of a strong and active Scientific Advisory Board that included scientists from cancer research centers and hospitals, NCI labs and executives from the pharmaceutical industry who all provided invaluable feedback, advice and direction on the development of SCan­‐MarK Explorer.

During the two­‐year Phase II SBIR project, a team of Sophic scientists curated select full-text papers that were rich in clinical data and experimental evidence from human biospecimens. Sophic scientists identified and extracted details for up to 33 often hard-to-find Critical Data Elements (CDEs) from each paper. A CDE represents detailed evidence intended to improve a researcher’s confidence that potential biomarkers identified in the literature will deliver value to the broad cancer community of basic researchers, biotech scientists, drug developers and clinicians. CDEs are integrated into highly computational Sophic Cancer Biomarker Objects (SCBOs) that allow scientists to quickly and easily find correlations between valid semantic and scientific relationships between CDEs, SCBOs, enrichment databases and clinical information. BioXM uses precise scientific data models and high-quality ontologies, thesauri and vocabularies that allow scientists to find and map complex biomarker knowledge networks. An intuitive, easy to use WIKI interface allows researchers to visualize networks of valid semantic and scientific relationships connecting SCBOs, pathways, near-neighbors, analyzed sequence data, mutations and drug compound information.

K. Stephen Suh, PhD., Director of the Tissue Bank and Genomics Program at the Juris Research Center, Hackensack University Medical Center, said:

In addition to participating on the Sophic SBIR Scientific Advisory Board, my lab has collaborated with Sophic for over 5 years on a range of projects and we have published biomarker papers together in various cancer journals. We participated in the Beta Test for SCan-MarK and found the WIKI interface very easy to use which allowed bench scientists to access valuable, hard-to-find biomarker information. This saved my lab not only time and money, but also allowed us to accelerate our biomarker discovery projects. This ultimately lead to promising lymphoma, ovarian cancer, breast cancer biomarker targets. Many have been validated against cancer patient tissues and are still under investigation.

Michael Liang, PhD., the Sophic Principal investigator responsible for the SCan-MarK project, said:

We considered the full range of biomarker papers with a range of data and various confidence levels but decided to curate only papers rich with clinically relevant information supported by strong evidence derived from human experiments. The Sophic team mined and selected papers for manual curation based on the quantity and quality of evidence in each paper and the value of the information to researchers. Sophic scientists studied each paper to identify and extract up to 33 CDEs that are the building blocks for SCBOs. To date, we have curated over 1,400 papers in order to extract these highly structured “building blocks”. The CDEs also function as information hooks that relate to other SCBOs and, more importantly, as bridges to valuable information stored in our bank of enrichment databases.

Patrick Blake, Sophic’s President, said:

Phase II of the SBIR Program gave us 2 years to develop SCan-MarK so we focused on quality information vs. quantity in our mining and curation efforts. Our strategy was to concentrate the time-consuming manual curation efforts to focus on five of the most deadly diseases: breast, ovarian, colon, Non-Hodgkin’s lymphoma and melanoma. This allowed Sophic scientists to dig very deep into the full text papers to extract valuable details while in parallel, integrating information from over 30 enrichment databases that help scientists “connect the dots” between biomarker targets with quality information in widely used data sources. Scientists can now save time and avoid dead-end research projects by accessing rich biomarker information related to pathways, sequence, mutations, etc., in a single system. Our strategy from the beginning was to collaborate with the cancer community, so we are grateful for the support we received over the past 2 years from each member of our Scientific Advisory Board. It would have been impossible to develop a comprehensive, multi-purpose system like SCan-MarK Explorer without their help.

SCan­‐MarK Explorer is a software­‐as­‐a­‐service offering that will provide scientists with quarterly updates and enhancements to the BioXM software configuration. Sophic provides a 2-­week free trial for scientists to evaluate a sample of the curated information and the power of BioXM on the company’s website. Government and academic researchers can license SCan-­MarK Explorer as a hosted, annual term service. SCan­‐MarK will be licensed for installation at commercial biotechs, pharmaceuticals and government/academic institutions. Sophic also provided a wide range of contract research services.

Source: Sophic Systems Alliance