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PFF-supported PRECISIONS Study Aims to Bring Personalized Treatment to IPF – Pulmonary Fibrosis News

Posted: November 7, 2019 at 3:45 am

The Pulmonary Fibrosis Foundation (PFF) announced a research initiative that aims to expand precision medicine strategies for diagnosing and treatingidiopathic pulmonary fibrosis (IPF).

The study, called PRECISIONS (for Prospective tReatmentEffiCacy inIPF uSIng genOtype forNacSelection), is being funded by a$22 milliongrant from the National Institutes of HealthandThree Lakes Partners, a philanthropic organization dedicated to pulmonary fibrosis care and research.

PRECISIONShas three primary goals.First, it aims to determine whether N-Acetyl-cysteine (NAC) is an effective treatment for individuals with IPF who have a particular variant of the geneTOLLIP, known to play a role in lung immunity.

NAC is an antioxidant, and it has been suggested in can lessen lung damage in IPF, although its efficacy is far from clear, with studies finding conflicting results.Previous researchsuggested that this may be because NAC is only effective in individuals with particular genotypes. Specifically, the variantTOLLIPrs3750920T/T, which is present in around a quarter of people with IPF, is associated with better clinical responses to NAC. PRECISIONS aims to directly test this association.

The second goal of PRECISIONS is to develop blood-based assays to help distinguish IPF from other lung diseases with similar symptoms. This will be accomplished by using unbiased analyses of transcriptomics (which genes are off and on in cells), proteomics (global analysis of proteins in the blood), and other large-scale molecular analyses, in order to identify molecular signatures that are linked with IPF. Such biomarkers could help in predicting an individuals disease course and response to therapy.

PRECISIONSthird and final goal is to identify genetic variants that influence an individuals risk of developing IPF, which may prove useful in monitoring people at a high risk of the disease.

This innovative study highlights the value of a partnership between a broad range of investigators, the PFF, a philanthropic organization (Three Lakes Partners), and the National Heart, Lung, and Blood Institute (NHLBI). Most importantly, it seeks to provide patients with interstitial lung disease (ILD) access to personalized medicine in which the right medication is used for the right patient,Fernando Martinez, MD, one of the researchers leading the project, said ina press release.

The project will rely on the PFF Patient Registry and Biorepository, which have collected clinical data and biological samples from over 2,000 pulmonary fibrosis patients.

The PFF Patient Registry will serve as an invaluable tool to facilitate more efficient enrollment into the NAC pragmatic trial, and to further define the genetic risk factors influencing the development and potential progression of the disease, saidGregory Cosgrove, MD, chief medical officer at PFF. It will hopefully allow for the identification of important biomarkers to assist in the diagnosis and care of patients with PF.

Added Imre North, MD, a co-principal investigator on the project:PRECISIONS has the potential to really change the scientific landscape over how we view IPF and ILDs by providing molecular classifications while determining if a pharmacogenetically driven treatment can change outcomes.

Marisa holds an MS in Cellular and Molecular Pathology from the University of Pittsburgh, where she studied novel genetic drivers of ovarian cancer. She specializes in cancer biology, immunology, and genetics. Marisa began working with BioNews in 2018, and has written about science and health for SelfHacked and the Genetics Society of America. She also writes/composes musicals and coaches the University of Pittsburgh fencing club.

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Patrcia holds her PhD in Medical Microbiology and Infectious Diseases from the Leiden University Medical Center in Leiden, The Netherlands. She has studied Applied Biology at Universidade do Minho and was a postdoctoral research fellow at Instituto de Medicina Molecular in Lisbon, Portugal. Her work has been focused on molecular genetic traits of infectious agents such as viruses and parasites.

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PFF-supported PRECISIONS Study Aims to Bring Personalized Treatment to IPF - Pulmonary Fibrosis News

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