Event Recap: Research Town Hall and 2020 Award Announcement

August 24,2020

On July 11, 2020, we held our very first community-focused Virtual Research Town Hall for patients and members of the community with presentations from leading physicians and researchers, including our 2019 research award winner. We also shared a patient’s perspective on participating in research, behind-the-scenes tours of laboratories and announced our 2020 research award winner. More than 109 patients, families, healthcare professionals and leading
members of industry registered from 10 countries. We thank everyone for their participation– speakers and participants. Thank you for making this a successful event.

Below is a brief summary of the Research Town Hall with links to the videos on our YouTube channel.

In Part 1, Dr. Patricia George opened up the Research Town Hall discussing a brief history of pulmonary hypertension (PH), definitions of the different diagnostic classifications, or groups, of PH, and described the framework of research (basic, translational, clinical), and different phases in clinical trials.

In Part 2, Dr. Hap Farber gave us his review of the most significant clinical trial findings in PH in the past year, and interesting molecules/treatments coming on the market. He discussed the INCREASE trial looking at inhaled treprostinil in pateints with PH and interstitial lung disease, the first successful clinical trial looking at vasodilator therapy in patients with ILD. He discussed findings from the TRITON trial looking at triple upfront vasodilator therapy. And he discussed the PULSAR trial, which studied sotateracept, a new molecule that helps restore BMP signaling and targets the pulmonary vascular remodeling side of PAH. He also discussed emerging therapies, pediatric research update, and stem cells.

In Part 3, Dr. Ray Benza described how PAH is a hemodynamic disorder, affecting the way the heart circulates blood. The gold standard for diagnosis of PAH is the right heart catheterization, and while the data might be helpful if we could get the data more frequently, it is challenging to do these on a weekly or monthly basis. He described the CardioMEMS sensor as a device that is implanted directly into the side of the pulmonary artery, allowing the patient to measure hemodynamics on a daily basis at home using a special sensor. This has been shown to reduce hospitalizations in patients with congestive heart failure, and also shows promise in the management of patients with PAH. He also talked about surgical right ventricular assist devices, including ECMO and a right ventricular balloon pump device.

In Part 4, Ms. Jan Janus, who lives with heritable pulmonary arterial hypertension (HPAH) gave us her perspective as a participant in clinical trials.

In Part 5, Dr. Yen-Chun (Charly) Lai talked about preclinical research in PH, and described how researchers use cells and animal models. She clearly presented the different types of PH, and then focused in on PH associated with heart failure with preserved ejection fraction (HFpEF), which is elevated pressures in the lungs associated with increased pressures from the left side of the heart. She talked about a type of PH-HFpEF associated with metabolic syndrome, and explained her novel animal model of this disease.

In Part 6, Dr. Vineet Agrawal, our 2019 Research Award Winner, presented his research into PH- HFpEF, a disease with currently no effective therapy. He discussed the role of natriuretic peptide clearance receptor (NPRC) in PH, and how this may be a treatment target in this disease. He then went on to discuss the concept of a virtual clinical trials to take knowledge gained from the animal model and see how NPRC mutants were associated with PH-HFpEF in
humans.

In Part 7, we brought you into the labs with video tours of research labs. Dr. Vineet Agrawal gave us a tour of the Hemnes Lab at Vanderbilt University, and Dr. Rebecca Vanderpool gave us a tour of her lab at the University of Arizona.

In Part 8, Dr. George gave a brief history of Team PHenomenal Hope, from RAAM 2014 through the present day. She also thanked Kayla Frattini for her amazing PH5K fundraiser. The Pittsburgh PH5K was the first major event to seed our research fund, which is now in the second year of funding promising PH research. She described the process of choosing the best research projects, the concept of the study section, and finally announced the 2020 Award
Winner, Paula Menezes.

We look forward to our next Research Town Hall in 2021, and can’t wait to learn more exciting
findings from this next year in pulmonary hypertension.

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