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Takeda findings may minimise haemophilia treatment side effects

Research from Takeda and Institute Krems Bioanalytics of IMC Krems will help treat patients.

Immune responses against factor VIII (FVIII) – used to treat haemophilia A – are influenced by the microenvironment of antigen-presenting cells.

The findings have emerged from a scientific study conducted in partnership between the Institute Krems Bioanalytics of IMC University of Applied Sciences Krems (IMC Krems) and Takeda.

Haemophilia A is a hereditary bleeding disorder caused by the absence or deficiency of active clotting FVIII in the blood. A major complication of this therapy is the development of a neutralising immune reaction in 30% of patients, therefore rendering the administered FVIII ineffective. The research group, led by the institute deputy head, Dr Christian Lubich, set out to address the ongoing issue.

During the study, a cell culture model was used to investigate the impact of the microenvironment on factor VIII uptake and processing within antigen-presenting cells. It was discovered that observed alterations in protein processing might have profound effects on immune responses to FVIII.

The novel data provided will form the basis for developing future FVIII therapies that elicit minimal immune responses in patients.

Dr Lubich explained: “The experimental design of our cell culture study aimed at taking a closer look at the response of different immune cells in the context of FVIII administration and thereby gaining better insight into the underlying processes.”

He added: “Immunopetidomics is a pioneering method for investigation of the composition and dynamics of proteins presented by antigen-presenting cells with the aid of mass spectrometry. This method can be used, for example, to determine peptides of a pathogen, a tumour cell or, as in this case, a biotherapeutic agent that elicits an immune response.”

IMC Krems specialises in innovative bioanalytical services and the institute focuses on tailor-made immunogenicity assessments.