People in the Foreground
Our research goals are oriented towards the people for whom we strive to attain these.
For example, we would like to explain what our research work and corresponding goals are for the hereditary disease hemophilia – also called the “blood disease.”
Those afflicted by hemophilia – usually men – suffer all their lives long from blood clotting problems, because the activity of the blood clotting factor VIII or IX is reduced or may even be totally absent. The consequence of this is that blood clots are formed very slowly, if at all, and it is very difficult to stop bleeding.
The Situation Today
The classical treatment approach has been substitution therapy with coagulating factors obtained from donor blood. In the past, those afflicted were exposed to a high risk of infection. And in the 1970s and 80s, blood donations were often not examined sufficiently for their level of infectiousness. Many cases of HIV or hepatitis C-infected hemophiliacs were the result. Today, this risk can be averted through careful donor selection and cost-intensive virus-deactivation procedures, but the danger of infection through viruses and pathogens yet to be discovered still remains. Another problem is the unbalanced relationship between the demand and offer for human plasma. Only about one out of every five hemophilia patients can be treated through substitution therapy.
The Situation Tomorrow
One of our research goals is to improve the quality of care to hemophilia patients.
A serious complication in the management of hemophilia is the formation of antibodies, which significantly hinders the treatment process. Octapharma is one of the first companies investigating the use of a human cell line for production of therapeutic coagulation proteins, with the goal that the manufactured pharmaceutical products are as close as possible to those produced physiologically in plasma, in regards to structure, efficacy and tolerability. We hope that this approach can reduce the formation of problematic antibodies.