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HPS Network collaborates with the Rare Lung Disease Consortium to accelerate research

Nov 8, 2017

The Hermansky-Pudlak Syndrome Network (HPS Network) has formed a collaboration with the Rare Lung Disease Research Consortium to accelerate research on Hermansky-Pudlak Syndrome. “We are very excited about this project that will enhance our research efforts on the lung disease of HPS,” says HPS Network President and Founder Donna Appell, “It will offer our community opportunities to participate in research that will help prepare us for future treatment trials and enable the engagement of more experts as they get to know us.”

The five-year project will set up research centers in strategic locations, making it possible to participate in research closer to home. Until now, clinical research on HPS has always been conducted at the Clinical Center on the National Institutes of Health campus in Bethesda, Md. NIH will continue to enroll patients, both new and old, and their dedication to finding a treatment will be uninterrupted.  To help with the research, the new centers in the Rare Lung Disease Consortium will enable the HPS community to expand existing research to follow the natural history of a wider range of patients. It will also help lay the groundwork to accelerate recruitment for future clinical trials.

The first HPS-related centers will be established at Vanderbilt University and Columbia University Medical Center. Dr. Lisa Young at Vanderbilt University was responsible for this opportunity and is the Primary Investigator and Dr. David Lederer at Columbia University was a key collaborator on this grant. The large number of patients with HPS in the greater New York City area also made Columbia a logical place to establish one of the first centers. The centers will not be open until 2015 and anyone interested in participating in this project should contact the HPS Network (1 (800) 789-9HPS or info@hpsnetwork.org) for more information or a referral.

This project was made possible by a grant from the National Institutes of Health through the Office of Rare Diseases (ORD) and the National Center for Advancing Translational Science (NCATS) in a program called the Rare Clinical Diseases Research Network which sponsors 22 consortiums. Our “Lung” consortium was also funded by the National Heart Lung and Blood Institute of NIH.

The HPS Network collaborated with HPS researchers to secure this opportunity, and will continue to play an active role throughout the project. Soon, Donna Appell, Founder and President of the HPS Network, and Valerie Friedman, Director of Medical Affairs for the HPS Network, will take classes so that they are able to work more closely with the researchers to serve patients in the program.

The National Center for Advancing Translational Sciences (NCATS) is a distinctly different entity in the research ecosystem. Rather than targeting a particular disease, NCATS focuses on what is common across diseases and how to make the research process more efficient. The Center emphasizes innovation and deliverables, relying on the power of data and new technologies to develop, demonstrate and disseminate improvements in translational science that bring about tangible improvements in human health.

The Rare Disease Clinical Research Network is designed to advance collaborative multisite studies on more than 200 rare diseases. The consortia provide a cost-sharing infrastructure and a robust data collection source that enables scientists to better understand, diagnose and treat rare diseases.

In addition to Hermansky-Pudlak Syndrome (HPS) the Rare Lung Disease Consortium will also study Pulmonary Alveolar Proteinosis and Lymphangioleiomyomatosis.