- acute myeloid leukemia (AML)
- aplastic anemia
- myelodysplastic syndromes (MDS)
- myeloproliferative neoplasms (MPN)
- paroxysmal nocturnal hemoglobinuria (PNH)
In 2008 Dr. Risitano received a grant for the research project, "Genetic Fingerprint of Complement and Complement-related Genes in PNH: Relationship with Pathophysiology, Clinical Manifestations (including Thrombosis) and Response to Eculizamab PNH Foundation."
We recently caught up with Dr. Risitano and asked how the grant helped him add to the understanding of bone marrow failure disease and where his current research is.
The grant contributed to allowing Dr. Risitano to perform research that has unraveled novel mechanisms of disease in PNH. Risitano and his team were first able to demonstrate that an additional mechanism of hemolysis may emerge during eculizumab treatment; it can be designated as "C3-mediated extravascular hemolysis.” They have subsequently extended their study, to provide possible explanations for the heterogeneity of response to eculizumab treatment, based on inherited factors. These studies were pivotal because they are leading to novel strategies of complement inhibition which are currently in preclinical and even clinical development. They anticipate that in a few years this will translate to substantial improvement in the treatment of PNH, with obvious benefits for both the patients and their families.
Dr. Risitano says that this grant was pivotal for his career development, since it provided him with independent funding needed to perform individual research plans. Indeed, based on coming data and burning issues, the grant funded a whole research line rather than a single small project. Thus, in the end, the scientific impact of the research was even larger than initially anticipated. The opportunity to do this research was translated into an opportunity for him to become a KOL in the field. More than six years later this award has been pivotal even for the further steps he is doing now in his career.
- accepting new patients