In July 2023, Enzyvant, now Sumitomo Pharma America, a subsidiary of Sumitomo Pharma, announced an update on their Phase 2 ELEVATE 2 study of Rodatristat Ethyl for PAH. The Phase 2b study failed to lower pulmonary vascular resistance, as measured by right heart catheterization, at Week 24. Sumitomo undertook a very rigorous evaluation of Rodatristat Ethyl in PAH. This included evaluating the molecule in animal models, as well as in both single and multi-dose studies in healthy people. The structure of their Phase 2 study was excellent, incorporating the best learnings from the past 2 ..read more

Across America a woman’s right to terminate an unwanted pregnancy is under threat. Without diving into politics or religion I want to impress on the PAH community why this may spell disaster for our PAH patients. The Supreme Court is on the verge of reversing half a century of legal precedent that has protected a woman’s right to choose. At the same time, several states have passed or are about to pass laws that criminalize abortion under any circumstances. Even states that preserve access to pregnancy termination in the case that the mother’s life is in jeopardy are creating new hoops that w ..read more

Imatinib is a tyrosine kinase inhibitor that has been on the market for more than 20 years to treat a type of leukemia.  It acts by turning off tyrosine kinases.   You can think of tyrosine kinases as critical switches in cells that turn on and off critical pathways.  Too much of an “on” signal leads to dysregulated cell proliferation and growth—such as seen in the pulmonary arteries of PAH patients or in the bone marrow of patients with leukemia.  Imatinib revolutionized the treatment for chronic myelogenous leukemia.  A decade ago, we participated in a groundbre ..read more

For the past 30 years, all our pulmonary arterial hypertension medications were described as vasodilators:   First prostanoids (epoprostenol), then endothelin receptor antagonists (bosentan, ambrisentan, macitentan) and PDE5 inhibitors (sildenafil and tadalafil).  These medications work through different mechanism but all lead to the vasodilation or relaxation of blood vessels in the lungs.  Unfortunately, they all share a similar side effect profile:  headaches, flushing, nasal congestion, diarrhea.  Progress with these three families of ..read more

Please forgive the hiatus in posting new content. When I am not caring for pulmonary hypertension and pulmonary fibrosis patients I am also an intensive care unit doctor and have been inundated caring for many thousands of patients with severe COVID. My team and I have cared for well over 8,000 patients with severe COVID during the past two years. Over the past year it has become crystal clear that vaccines work. The overwhelming majority of patients dying from COVID are unvaccinated and unboosted. In the absence of underlying diseases that impair the immune system or medications that suppres ..read more

Pulmonary hypertension (elevated pressure within the blood vessels of the lungs) is organized into five groups: Group 1:  Pulmonary arterial hypertension (PAH) Group 2:  Pulmonary venous hypertension (due to problems on the left side of the heart) Group 3:  Pulmonary hypertension due to lung disease Group 4:  Pulmonary hypertension due to blood clots in the lungs Group 5:  Pulmonary hypertension due to blood problems and various other causes Within group 3, there are three main sub-categories.  Chronic obstructive pulmonary disease ..read more

For Pulmonary Arterial Hypertension (PAH) patients who remain very short of breath despite maximal medical therapy, lung transplantation may be an option.  Maximal medical therapy usually means continuously infused therapy (Remodulin, Flolan, Veletri) and multiple oral medications.  First, it is important to know that lung transplantation is not a cure.  It is the exchange of one disease for another. The old disease is your PAH and the new lung disease is called lung transplantation.  Much like PAH, it is no cake walk.  You will have to take m ..read more

An important cause of PAH is liver disease.  Up to 5% of patients with cirrhosis (scarring in the liver) or portal hypertension (high pressure in the blood vessels of the liver) can develop PAH.  All causes of cirrhosis and portal hypertension can develop PAH.   Causes The most common causes of cirrhosis (scarring of the liver that leads to liver failure) are hepatitis (Hepatitis C in the United States and Hepatitis B in other countries), chronic alcohol abuse and non-alcoholic steatohepatitis (also called fatty liver).  There are many other causes of ch ..read more

In a surprising development, Complexa abruptly terminated their phase 2 study in PAH.  They seem to have taken a page from Reata’s playbook.  The news blindsided the PAH community and is a reminder that the development of a successful medication is a long hard road.  The company did not provide much explanation.   This is the second study this year that has been stopped abruptly.  Reata abruptly terminated their phase 3 study of bardoxolone methyl after many years of investigation.  Both molecules acted to improve energy use by cells ..read more

During the past few months, we have not had quite as much time as usual to address our readers questions. Apologies for that.  When I am not caring for pulmonary hypertension patients, I wear a critical care doctor’s hat and have been taking care of a growing number of gravely ill COVID patients.  At Inspire Pulmonary Media and PulmonaryhypertnesionRN.com we appreciate our readers and hope that you are safe during this stressful time.   What is borderline pulmonary hypertension? Usually we encounter this term in an echocardiogram report.  The echoca ..read more