Promising New Treatment for Paroxysmal Nocturnal Hemoglobinuria

by Lauren Taylor from In The Cloud Copy

Paroxysmal nocturnal hemoglobinuria (PNH) is a very rare disease of the blood in which the affected individuals red blood cells break apart. This breaking apart is called “hemolysis.” This process occurs because the individual is missing a certain protein on the surface of the blood cell that protects them from the body’s own immune system. The process of the red blood cell breaking apart ultimately causes the hemoglobin to be released. The hemoglobin is the element of the red blood cell that carries oxygen throughout the body. The release and absence of hemoglobin is the cause of the majority of PNH symptoms.

PNH is very rare, affecting 1-1.5 persons per million, with most affected being younger adults. The median diagnosis age is 35-40 years of age. Symptoms of the disease are variable from person to person and it is not rare for an individual to go undiagnosed for months to years. The most commonly seen symptoms include significant fatigue or weakness, easily bleeding and bruising, shortness of breath, difficulty controlling bleeding, severe headache, recurring infections, and blood clots.

Current treatment options for PNH include medical therapy or bone marrow transplants. Currently the only medication approved by the U.S. Food and Drug Administration is a drug called eculizumab (Soliris). This medication works by binding to the proteins in the blood that destroy red blood cells. This works to reduce the risk of thrombosis and improve a PNH patient’s quality of life. An allogenic (donor) bone marrow transplant is the only cure for PNH and works to replace all of the patient’s bone marrow stem cells with those of a donor. This is indicated when patients don’t respond to medical therapy or are having severe reductions in cell counts.

New Treatment Options for PNH

A new medication for treatment of PNH is showing promising results from drug maker Novartis. LNP023, an investigational oral treatment, was tested in patients that were currently being treated with eculizumab and experiencing active hemolysis. Patients in the clinical trial who were given LNP023 in addition to the eculizumab showed notable decreases in the biomarkers of intravascular hemolysis and lactate dehydrogenase, while also showing noteworthy increases in their hemoglobin levels. Patients showed increases of 2.87 g/dL in hemoglobin levels compared to their baseline levels when taking just eculizumab. All but two of the patients enrolled in the trial saw hemoglobin levels of greater than 12 g/dL without the need for a red blood cell transfusion, which all were required prior to starting LNP023.

Further study information shows that seven patients were able to discontinue their eculizumab completely and remain just on the LNP023 after six months of stability with LNP023 as add-on therapy. These seven patients all had stable hemoglobin levels with no biomarker changes for disease activity as well as no apparent breakthrough hemolysis signs or symptoms. No infections or serious thromboembolic events were recorded due to this new treatment in study participants.

These promising results could truly be changing the course of this disease and how it is treated. These results show that monotherapy with oral LNP023 has potential and could change the way in which PNH is treated for future patients.

Learn more about these findings here.

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