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The starting point: search for a new treatment approach

From idea to effective therapy (2/6)

From Idea to Patient Step I
When discovering a potential active substance, researchers find a target in the disease process and an active substance that has an impact on it

 "The first step in developing a new drug is to find a treatment approach for a disease" explains Dr. Marc Nolte, who heads preclinical pharmacology and toxicology at CSL R&D in Marburg. " This requires a detailed understanding of the disease in order to identify where in the disease process an active substance could have a positive impact. That is the task of basic research, where we collaborate a lot with researchers at universities or other research institutions." 

A good example is hemophilia A. It is a hereditary disease that leads to prolonged bleeding after injury or surgery. Spontaneous bleeding in joints, muscles, brain or other internal organs can lead to serious complications. It has been found that the bleeding results from a deficiency of functional clotting factor 8, which is normally present in the blood. The treatment approach is therefore to replace the missing clotting factor. A suitable substance had to be found for this purpose.

In hemophilia, the cause of the disease is clear. In other diseases, such as immune thrombocytopenia (ITP), it is more complex. And sometimes it is simply not known how the disease develops. In such cases, several years often pass before a treatment approach is found. 


" The first step in developing a new drug is to find a treatment approach for a disease. "

Dr. Marc Nolte, Head of Pharmacology & Toxicology, CSL R&D Marburg

Once researchers have found promising drug candidates, the journey continues for them on their way to becoming a new drug.      

Next page > A first milestone: A candidate becomes a test drug

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