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A Collaborative Approach to encourage research and promote new treatments for orphan diseases
Publication date: January 2015 Source:New Horizons in Translational Medicine, Volume 2, Issue 2 Author(s): Lesley Greene The last two decades has seen a significant increase in the development of medicinal products to treat rare “orphan” diseases, largely due to the EU Orphan Medicinal Product Regulation (2000), but also because of the consistent advocacy by patient groups prior to this regulation. These groups from across Europe joined forces under the umbrella of EURORDIS, to secure the implementation of the regulation and ensure that there was patient representation on the committee at EMA (the European Medicin...
Source: New Horizons in Translational Medicine - February 8, 2015 Category: Research Source Type: research

Approaches in rare diseases and pediatrics across international boundaries
Publication date: January 2015 Source:New Horizons in Translational Medicine, Volume 2, Issue 2 Author(s): Michael N Liebman , S. Molinaro Rare diseases are designated as affecting less than 200,000 individuals (US) and of the approximately 7000 designated rare diseases, the majority of these occur in pediatric patients, and across international boundaries. An example is pediatric ARDS (Acute Respiratory Distress Syndrome) that is not diagnosed until a previously healthy child presents in the pICU with severe symptoms and in which more children die each year than from cystic fibrosis and leukemia, combined. The Natha...
Source: New Horizons in Translational Medicine - February 8, 2015 Category: Research Source Type: research

Peptide mimotopes of malondialdehyde-epitopes for clinical applications in cardiovascular disease
Conclusions Thus, we have identified specific mimotopes of MDA-LDL that serve as highly reproducible antigens to assess autoantibody titers in patients with cardiovascular disease. Future studies will reveal their usefulness for therapeutic vaccination approaches against atherosclerosis. (Source: New Horizons in Translational Medicine)
Source: New Horizons in Translational Medicine - February 8, 2015 Category: Research Source Type: research

TCR gene therapy of leukemia
Publication date: January 2015 Source:New Horizons in Translational Medicine, Volume 2, Issue 2 Author(s): Shao-An Xue Conventional cancer therapies are limited by their toxicity and lack of specificity. To achieve targeted immunotherapy of cancer, we have chosen Wilm's Tumour antigen (WT1) as a target as it is over-expressed in most leukemia and many solid cancers. Using sophisticated WT1-TCR retroviral constructs, we have performed in vivo engraftment studies with CD34+ leukemic progenitor cells. In our model, treatment with WT1-TCR engineered patiens' T cells had cleared patients own leukemic cells. As the analysis...
Source: New Horizons in Translational Medicine - February 8, 2015 Category: Research Source Type: research

Personalized medicine: Moving from correlation to causality in breast cancer
Discussion Our preliminary results begin to approach the specificity and sensitivity of the Gail model (AUC=0.957 (Gail model) and 0.745 (physiological model)) and further refinement is ongoing. By contrast, our model presents the opportunity to more directly personalize risk assessment based on an individual patient’s characteristics and present the potential to develop management plans to reduce potential risk and to identify potential opportunities for biomarker/diagnostic development (and therapeutics) based on the specifics of the disease process unique to the patient. (Source: New Horizons in Translational Medicine)
Source: New Horizons in Translational Medicine - February 8, 2015 Category: Research Source Type: research

EU early access - regulatory framework & practical considerations
The objective of this article is to provide an overview of the regulatory frameworks available in the member states, as well as practical considerations for implementation of an access program. (Source: New Horizons in Translational Medicine)
Source: New Horizons in Translational Medicine - February 8, 2015 Category: Research Source Type: research

Next-generation tissue microarrays (ngTMA) in translational research
Publication date: January 2015 Source:New Horizons in Translational Medicine, Volume 2, Issue 2 Author(s): Inti Zlobec , Guido Suter , Aurel Perren , Alessandro Lugli Over the last two decades, prognostic and predictive biomarker studies in clinical and translational research settings have become synonymous with tissue microarrays (TMAs). TMAs are essentially “tissue archives” created by repeated transfer of small tissue cores from formalin-fixed paraffin-embedded tissues (“donor” blocks) into empty paraffin blocks (“recipient” blocks). In this manner, more than 500 different tissue spots can be arrayed...
Source: New Horizons in Translational Medicine - February 8, 2015 Category: Research Source Type: research

Identification & characterization of tumor cells isolated from body fluids
Publication date: January 2015 Source:New Horizons in Translational Medicine, Volume 2, Issue 2 Author(s): Gottfried Köhler The appearance of malignant cells in body fluids like urine, blood or body-cavity fluids are a clear indication for the existence of a tumor and urine or body-cavity fluid cytology are routine diagnostics today. Cytologic examination of the cellular features of fluids is a valuable adjunct to patient diagnosis and the staging and management of tumors. The German-language literature contains the earliest references to the cytology of malignant cells in fluid specimens. Preparation of the specimen...
Source: New Horizons in Translational Medicine - February 8, 2015 Category: Research Source Type: research

Bioinformatics challenges in the adoption of next generation sequencing for translational molecular diagnostics
Publication date: January 2015 Source:New Horizons in Translational Medicine, Volume 2, Issue 2 Author(s): Puthen Veettil Jithesh Next generation sequencing (NGS) technologies are now widely used in medical research. NGS provides an unprecedented opportunity for high throughput analysis of genetic variations warranting their use in molecular diagnostics. However, among other obstacles, their adoption in clinics poses challenges in the provision for accurate and timely data analysis. A bioinformatics workflow for the analysis of the large amount of data from raw reads to final annotated variants suggesting their functi...
Source: New Horizons in Translational Medicine - February 8, 2015 Category: Research Source Type: research

Metabolic phenotyping in mouse and man: Mind the differences!
Publication date: January 2015 Source:New Horizons in Translational Medicine, Volume 2, Issue 2 Author(s): Denise Sonntag , Guido Krebiehl , Torben Friedrich Metabolic phenotyping comprises the quantification of endogenous metabolites in biofluids, cells, and tissues. It provides insights into normal as well as aberrant metabolic pathways and biological processes, which is important for the understanding of disease phenotypes. It also allows the identification of biological markers, which can serve as early disease indicators and therapeutic markers for the evaluation of treatment effects. As metabolic markers are n...
Source: New Horizons in Translational Medicine - February 8, 2015 Category: Research Source Type: research

A big data platform to enable integration of high quality clinical data and next generation sequencing data
Publication date: January 2015 Source:New Horizons in Translational Medicine, Volume 2, Issue 2 Author(s): Joel Haspel Today, personalized medicine is closer to reality than ever before through targeted treatment, however, the substantial increase in data correspondingly requires scalable systems to continue to effectively manage the data and to remain current with advancing technology. As organizations move to advance translational research to achieve personalized medicine, researchers and clinicians must manage informatics, however, there is a shortage of fully integrated informatics solutions that integrate, store,...
Source: New Horizons in Translational Medicine - February 8, 2015 Category: Research Source Type: research

A newly human developed diabody against CD99 delivers a lethal signal through p53 pathway reactivation in Ewing sarcoma cells and synergistically acts with doxorubicin
We present here a new engeneered human bivalent single chain fragment variable diabody (C7 scFv diabody) directed against CD99, a transmembrane protein whose high expression characterizes Ewing sarcoma. The triggering of CD99 with C7scFv diabody induces rapid and massive Ewing sarcoma cell death through MDM2 ubiquitination and p53 reactivation. Accordingly, the most CD99-responsive Ewing sarcoma cells have transcriptional active p53 and greatly benefit from MDM2 degradation. CD99 triggering also potentiates the cytotoxic effect of doxorubicin in vitro and in vivo and reactivates p53 to a much greater degree, which in turn ...
Source: New Horizons in Translational Medicine - February 8, 2015 Category: Research Source Type: research

Cancer prevention strategies in different countries: Qualitative and quantitative differences
This study represents a preliminary report regarding a novel linguistic approach to assess the efficacy of cancer prevention strategies. The conclusions indicate significant and relevant quantitative differences between communication strategies having language differences. (Source: New Horizons in Translational Medicine)
Source: New Horizons in Translational Medicine - February 8, 2015 Category: Research Source Type: research

RLIP76 protein reduces 4-HNE generated during oxidative stress and results in protection in well characterized animal models of acute radiation syndrome
Conclusions Combined data of many studies show that when compared to controls, treated mice exposed to LD50s, LD70s, or LD90s resulted in survival ranging from 50-80% over the controls. RLIP76-PL is a strong candidate to protect the population from acute radiation exposure. (Source: New Horizons in Translational Medicine)
Source: New Horizons in Translational Medicine - February 8, 2015 Category: Research Source Type: research

Targeting microRNAs: Towards a new tailored therapy for hepatocellular carcinoma
Publication date: January 2015 Source:New Horizons in Translational Medicine, Volume 2, Issue 2 Author(s): Raffaele Baffa Hepatocellular carcinoma (HCC) remains a significant unmet medical need with very limited therapeutic options available. Although microRNA-21 (miR-21) has been shown to be upregulated in HCC, its contribution as an onco-miR to the maintenance of tumorigenic phenotype in liver cancer remains poorly understood. We have developed potent and specific single-stranded oligonucleotide inhibitors of miR-21 (anti-miR-21) and used them to interrogate dependency on miR-21 in a panel of 20 commercially availab...
Source: New Horizons in Translational Medicine - February 8, 2015 Category: Research Source Type: research