First Effective Treatment Against a Dangerous Form of Anemia

Vienna, June 9, 2007 – Without prior warning, quick and fatal. That is usually how thrombosis (blood clot) develops when they block the blood stream at a particularly critical place, such as near the heart or in the brain. Until recently, a person suffering from paroxysmal nocturnal hemoglobinuria, or PNH for short, stood a 45 percent risk of his/her life ending this way.

This danger is now dramatically reduced. A new drug known as eculizumab reduces the occurrence of thrombosis by no less than 85 percent. This finding was made in a multicenter study at hospitals, which was presented today at the 12th Congress of the European Hematology Association (EHA). More than 7,000 experts in blood and bone marrow diseases are gathering for this congress in Vienna from June 7 to 10. Professor Peter Hillmen from Leeds Teaching Hospital in Leeds (GB), the lead author in the study: “Eculizumab is a genuine breakthrough to a new generation of intelligent drugs against PNH.”

New active ingredients act directly for the first time This was made possible by a new ingenious mode of action. It may not cure the disease but it does interrupt the disastrous causal chain the disease triggers at a critical point. That makes eculizumab the first approved drug that acts directly against PNH.

Paroxysmal nocturnal hemoglobinuria is a form of hemolytic anemia, i.e. anaemia due to pathological destruction of red blood cells (erythrocytes). It can be traced to a mutation of the stem cells in the blood-forming bone marrow, which is acquired over the course of a person's life. In producing red blood cells, the bone marrow leaves a particular protein out. As a result, the red cells are 'treated like enemies'. Proteins in the immune system, especially the protein C5, perforate the cell membranes of the red blood corpuscles. As a consequence, these cells can no longer transport oxygen through the body. Instead, they dissolve and “explode” into the blood stream. This anemia results in exhaustion, pallor, shortness of breath, and a dark coloration particularly of morning urine.

This symptom gives the disease its name (hemoglobinuria means “red coloring in urine”) and is the outward sign of a dangerous process. Not only are the destroyed red blood cells unable to perform their job, they inundate the body with large amounts of their disintegration products. These products arrive in the urine at a time when the processing capacities of the liver and kidney are exhausted.

High risk of thrombosis

These patients also have a high risk of thrombosis. Dr. Peter Hillmen: “Until now the only way we could counter this danger was to use anticoagulants as a prophylactic in cases of PNH. These agents increased the risk of a fatal hemorrhage, however, without preventing the occurrence of thrombosis. They occurred anyway in 30 percent of the patients. Eculizumab has a totally different mode of action. It occupies the protein C5, so the latter has no more free capacity to attack the erythrocytes.”

85% decrease in vascular occlusion

The success of this drug is striking. The TRIUMPH Study published last year already showed that eculizumab stabilizes the hemoglobin values in about half of patients treated with it and reduces the need for blood transfusions by an average of 10 per patient and year to zero. Now this active agent has also been shown to bring about an 85 percent reduction in the risk of vascular occlusions.
This success is not achieved entirely without side effects. When the C5 proteins are put in check, the body no longer has them as a weapon to combat genuine foes. This increases the danger of infection from certain pathogens. However, this trade-off adds a number of additional years to a patient’s life. Dr Hillmen: “If this treatment eliminates 85% of the events that previously took the life of 45% of PNH patients, the logical result is greater life expectancy, even if there are not yet any studies on this aspect.”

Contact:

B&K Medien- und Kommunikationsberatung GmbH Dr. Birgit Kofler; Daniela Pedross, MA.
Porzellangasse 35/Top 3, A-1090 Vienna
Phone: 0043-(0)676-6368930; Fax: 0043-01-319-43-78-20
E-Mail: kofler@bkkommunikation.at; pedross@bkkommunikation.at

About EHA

The European Hematology Association (EHA) aims to promote excellence in clinical practice, research and education in European hematology.
Today, EHA – with over 2600 active members from 95 countries – is a consolidated organization that pursues a large and growing number of projects and programs. An Executive Board and Councilors elected by the membership form the governmental body responsible for the strategy and organization of the association.

About the EHA Annual Congress

The EHA annual congress is organized every June in a major European city. Over the years the congress has become the meeting place for hematologists in all fields of the speciality. The congress program has sessions on clinical and laboratory hematology and covers all the major hematological subspecialties, including hemato-oncology, red cell disorders, hemostasis, thrombosis, pediatric hematology and transfusion medicine.