A potential therapeutic breakthrough awaits pediatric and adult patients with acute myeloid leukemia (AML), one of the most aggressive blood cancers. In fact, this year, coordinated by the Bambino Gesù Hospital in Rome, the first clinical trial in Europe of gene therapy with Car-Natural killer cells (Car-Nk) will start. It is an advancement of immunotherapy compared to the recent Car-T therapy. The difference lies in the fact that instead of using T lymphocytes (which often cause uncontrolled immune reactions), the Italian procedure “recruits” cells (Nk) taken from a healthy donor; these are reprogrammed to intercept the tumor target and destroy it.
Designed in the Vatican hospital in the capital, the method, which in the preclinical phase has given excellent results on animals, involves researchers from the new national network of institutes specialized in the oncohematological field, “Palm”, supported with a loan of over 3 million euros by the Umberto Veronesi Foundation. The 5-year project does not only involve the research and development of innovative therapies: new diagnostic methods will also be investigated for this rare disease which, in our country, affects 70 people a year in the 0-18 age group. The activities of the Palm network will immediately contribute to improving the treatment of AML in pediatric patients, not only in Italy. In addition to the Bambino Gesù, the diagnostic laboratory of the Oncohaematological clinic of the Padua University Hospital, the department of Experimental Oncology of the European Institute of Oncology in Milan and the Leukemia department of the “MD Anderson Cancer Center” in Houston (USA).
The researchers of the network will have a vast field of action: from the identification of new molecular alterations to the discovery of the mechanisms responsible for resistance to treatments or disease relapses, up to the trials of Car-NK cells for patients with relapsed or refractory AML. There is still a lot to understand about this disease given that it presents recurring molecular alterations which, in a third of cases, are not identified. A limit that today can be overcome with new generation sequencing technologies, available in the Institutes of the Network. Technologies that will also serve to better define the prognosis, the sensitivity to treatments and to discover tumor targets to be hit with targeted therapies.
“Investigating the heterogeneity of cells – the researchers argue – is one of the keys to clarifying the behavior of the tumor and to directing therapies”. This investigation will be performed, for all cases of pediatric AML diagnosed in Italy, in the AIEOP centers (Association of Pediatric Hematology and Oncology). «The research of the Palm Network and the availability of Car-Nk immunotherapy – explain Bambino Gesù and the Veronesi Foundation – will contribute decisively to the success of the protocol for the treatment of AML which will involve other European countries (Italy and Germany lead partner; Austria , the Czech Republic, Slovenia, Slovakia, Switzerland and Poland). This is the first transnational experience of this type for the Lma. Among the same countries, studies will be activated which will be accessible to more than 200 children a year: a large case series» which will make it possible to reduce the trial times (about 3 years compared to 6 for studies conducted at the national level alone).
«Scientific research is the basis, the heart of medicine – comments Mariella Enoc, president of Bambino Gesù -. A continuous challenge that must be supported and strengthened. Thanks to the commitment of the Veronesi Foundation and the work of the many researchers who place their knowledge at the service of science, today we are closer to defining effective treatments for children with cancer, to innovative treatments that open up new possibilities for their future ». The new research platform launched with the Child Jesus, adds Paolo Veronesi, president of the Veronesi Foundation, and professor of General Surgery at the State University of Milan, is part of a process that has seen the Foundation engaged for years in financing «scientific research and the best treatments for young cancer patients, through the activation of treatment protocols that comply with the highest international standards”.
For his part, Franco Locatelli, rhead of the area of Pediatric Oncohematology and cell and gene therapy of the Child Jesus and full professor of Pediatrics at the Catholic University, it is said «particularly happy for the start of this project which has all the characteristics to contribute significantly to further improve the diagnostic and therapeutic approaches in the field of child and adolescent AML. The collaboration between Aieop and the Veronesi Foundation represents a model of how virtuous synergies of interaction between the scientific world and institutions that aim to support biomedical research can translate into initiatives of extraordinary importance for the treatment of oncological diseases».