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 causes the appearance of fibrous tissue in the bone marrow, which changes its composition, as well as the enlargement 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, showed that the 21 patients who underwent the phase I clinical trial with a drug that inhibits transforming growth factor TGF-β 1/3 were no longer dependent on platelet transfusions 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 count in 81% of patients, three of whom reached 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”.
UCBM researchers played a key role in identifying the mechanism by which platelet counts were restored in treated patients by testing the maturation of megaryocytes, highly specialized hematopoietic cells responsible for producing 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.
