Welcome! We created this “who is who” interview series to help our community connect better and learn about SilkFusion, its partners and its experts.
|Job title||Research Director|
|Organization||INSERM and Gustave Roussy Cancer Campus|
SilkFusion covers the critical need of functional platelets, independent from donations, for transfusions as well as for clinical and physiological studies.
The aim of Silkfusion is not only to develop the platelet production at large scale for transfusion purpose, but also to propose a personalized medicine for patients suffering of severe inherited thrombocytopenia. The only treatment of patients with severe inherited thrombocytopenias today is the use of thrombopoetin (TPO) analogues romiplostim and eltrombopag independently of the genetic defect. These TPO-receptor agonists enhance the production of platelet precursors, megakaryocytes, but do not correct the intrinsic defect in platelet generation at the origin of these thrombocytopenia explaining that not all patients respond to these drugs. Therefore, one of the Silkfusion aims is to propose personalized treatment. In this way, the first objective is to decipher the physiopathological mechanisms of those inherited thrombocytopenia for which no efficient specific treatment exists in order to identify druggable targets.
For this purpose, Silkfusion has developed a bank of patient-derived iPSC to model these diseases in order to understand the mechanisms of the thrombocytopenia. Could you tell us about this point?
Yes. It will be possible to perform drug screening as the 3D silk-based scaffold allows the in vitro production and precise quantification of functional platelets. Our goal is to miniaturize this scaffold to facilitate drug screening to rescue platelet production in the different types of inherited thrombocytopenia.
What is your role in SilkFusion project?
The role of my team in Silkfusion is to generate the iPSC lines from patients with inherited thrombocytopenia carrying different genetical alterations and study the physiopathological mechanisms leading to these diseases with the aim to identify new druggable targets. Once derived, these iPSC lines are phenotypically and genetically characterized and bio-banked by ISENET.