The researchers coordinated by professor Anna Rita Migliaccio. Collaboration with 11 US centers

di Beatrice Passarelli

A group of researchers coordinated by Professor Anna Rita Migliaccio, contract professor of Histology and Embryology at theUniversità Campus Bio-Medico di Roma and senior investigator at the Altius Institute for Biomedical Sciences in Seattle, in collaboration with 11 US research centers and with the University of Florence, Azienda Ospedaliero Universitaria Careggi and the Istituto Superiore di Sanità, contributed to a clinical study on patients suffering from advanced myelofibrosis.

The disease involves the appearance of a fibrous tissue in the bone marrow, which modifies its composition, in addition toingredness of the spleen and liver and produces symptoms such as tiredness, muscle pain, fever, night sweats, itching and even thrombosis.

The results, published in the prestigious journal Clinical Cancer Research, demonstrated that the 21 patients subjected to the phase I clinical trial with a transform inhibitor druging growth factor TGF-β 1/3 were no longer dependent on platelet transfusion during the treatment period and, in some cases, were able to suspend transfusions, with a clear improvement in their quality of life. The treatment period in fact induced an increase in platelet counts in 81% of patients, three of whom achieved normal levels of platelets in the blood. The treatment also revealed no significant toxicities. “This is an exceptional result – explained Professor Anna Rita Migliaccio – because none of the experimental treatments evaluated so far for this disease have had an effect on the number of platelets”.

At the end of the treatment Ucbm have played a fundamental role in identifying the mechanism through which the platelet count was restored in treated patients by verifying the maturation of megariocytes, highly specialized hematopoietic cells responsible for the production of platelets.

This study therefore highlighted how the action of the treatment, by interrupting the effects of TGFβ1, promotes the production of platelets in patients with myelofibrosis, curing at least in part their complex symptoms.