The results of a study published in the International Journal of Molecular Sciences suggest a possible new compound for treating hemophilia A in patients who cannot use Factor VIII infusions.

The main complication in therapy for hemophilia A is that many patients generate autoantibodies against the injected Factor VIII, which ultimately has little effect. Therefore, it is urgent to study the coagulation mechanisms that will allow us to find new therapies for these patients.

In this regard, researchers from the University of Cádiz UCA) and Puerta del Mar Hospital (HUPM), affiliated with the Cádiz Institute for Biomedical Research and Innovation Cádiz INiBICA), in collaboration with researchers from the Virgen del Rocío Hospital (HUVR) in Seville, have recently described how phosphate polymers (polyphosphates) could form the basis for the design of new therapies to treat hemophilia A.

In previous studies, researchers had found that one of the factors in blood, called von Willebrand factor (vWF), can be modulated by phosphate polymers (polyphosphates) that are produced naturally in the body.

In this study, it has been determined that the modulation of FvW by polyphosphates occurs only in the absence of another blood factor, called Factor VIII. Factor VIII is precisely what is missing in patients with hemophilia A.

The Authors experiments on plasma from healthy individuals, patients with hemophilia A of varying severity, and patients with different types of von Willebrand disease. They also studied molecular binding in vitro, where they found that polyphosphate and FVIII compete to bind to FvW. These findings were published this week in the International Journal of Molecular Sciences.

Based on the results obtained, the Authors that polyphosphate—which is a natural coagulant—could be used in the design of new therapies to treat hemophilia A that replace FVIII when the latter cannot be used, such as in patients who have developed autoantibodies.

This work was carried out by the Phosphate Metabolism research group atHUPM, in close collaboration with professors from the Department of Biomedicine, Biotechnology, and Public Health (UCA) and the Department of Hematology at HUVR. The researchers involved were Marcela Montilla (HUPM, now a professor at the Cooperative University of Colombia), Isabel Atienza-Navarro (HUPM), Francisco José García-Cozar (UCA), Carmen Castro (UCA), and Francisco Javier Rodríguez-Martorell (HUVR), led by Professor Félix A. Ruiz (Nutrition Department, UCA).

Bibliographic Reference: Montilla, M.; Atienza-Navarro, I.; García-Cozar, F.J.; Castro, C.; Rodríguez-Martorell, F.J.; Ruiz, F.A. Polyphosphate Activates von Willebrand Factor Interaction with Glycoprotein Ib in the Absence of Factor VIII In Vitro. Int. J. Mol. Sci. 2022, 23, 14118. https://doi.org/10.3390/ijms232214118.

🔗 Más información: Polyphosphate Activates von Willebrand Factor Interaction with Glycoprotein Ib in the Absence of Factor VIII In Vitro