"The therapeutic landscape is evolving rapidly, to the point that the dream of one day curing all patients with pediatric acute leukemia no longer seems like an unattainable pipe dream."
Franco Locatelli, a professor at the Catholic University of the Sacred Heart and head of the Clinical and Research Area of Oncohematology, Cellular Therapy, Gene Therapies, and Hematopoietic Transplantation at the Bambino Gesù Hospital in Rome, is accustomed to weighing every word. Sixty-five years old, president of the Superior Council of Health until last year—a role that made him, despite himself, a familiar face on TV during the Covid era—and a pioneer in blood cancer research, he has examined and treated thousands of children and adolescents throughout his career, living daily with their suffering and that of their families. A situation that, inevitably, convinces you to never give false hope.
This is why his prediction, more than a wish, is now a concrete goal supported by data: the cure rate for leukemia today is over 80 percent, compared to just a couple of decades ago, it barely exceeded 60. This extraordinary achievement is the fruit of scientific research, which has never stopped exploring new avenues, such as CAR-T, the revolutionary technique in which the patient's white blood cells are genetically transformed into true killers that recognize and destroy only tumor cells.
With the treatments available today, would Carlo Acutis, the teenager who died in 2006 and was proclaimed a saint last September, have been saved?
Acutis died of acute promyelocytic leukemia, which we now cure in 97-98 percent of cases without a single milligram of chemotherapy thanks to the combined use of a vitamin A precursor and an arsenic derivative. So yes, he would have had a much better chance of survival. Also because when I started doing this work, in cases like his, we lost a quarter of the patients in the first 48-72 hours. This data gives us an idea of just how extraordinary the progress has been: just think that today we can cure between 85 and 90 percent of children with acute lymphoblastic leukemia and between 75 and 80 percent of those with acute myeloid leukemia.
Why are positive results less impressive in adults?
"Molecular lesions in leukemia cells differ significantly between adults and children, and this helps explain the differences in the likelihood of recovery. Furthermore, young people tolerate treatment better than adults over 50."
How is immunotherapy changing the prognosis of leukemia patients?
When we talk about immunotherapy, we mean both the use of targeted antibodies and CAR-T cells, which are genetically modified immune system components that are redirected to a target on the surface of tumor cells, where they act as veritable snipers. Until a couple of years ago, CAR-T cells were only used on the B-cell form of acute lymphoblastic leukemia, but today we're starting to see initial results in acute myeloid leukemia as well, and it's a technique that's increasingly being refined.
What are the main benefits of new therapies compared to traditional chemotherapy?
"Meanwhile, it limits a whole series of side effects, such as a decrease in white blood cells and platelets, nausea, and vomiting. Immunotherapy reduces the risk of infections, cardiac failure, pancreatitis, and endocrine dysfunction. However, chemotherapy has not exhausted its role and remains essential."
Then there is the bone marrow transplant, what importance does it have in the treatment of blood cancers?
It continues to play a central role, especially in patients who have relapsed or are refractory, or who have biological characteristics that, from the moment of diagnosis, predict a high probability of failure with non-transplant treatments. It is also an essential tool in other blood diseases, such as congenital defects of the immune system or bone marrow aplasia. It is therefore essential to foster a culture of donation, which poses no risk. In Italy, although we are not in a bad situation, we can certainly do more, so we must continue to raise awareness among all potential bone marrow donors, that is, healthy individuals between the ages of 18 and 35. With this gesture of biosolidarity, we can offer a concrete chance of recovery to an adult or child in need of a transplant.
Funding research requires a lot of money. How are we doing in Italy on this front?
"Today, the largest resources available—about three-quarters—come from private institutions like AIRC, the Umberto Veronesi Foundation, and the AIL. The state, however, should do much more, perhaps even setting aside funds specifically for pediatric oncology, which is currently not happening. The more we understand the mechanisms responsible for the development of leukemia cells, the more selective we can be with targeted therapies, which is why investing in research is crucial."
New therapies, but also family support. How important is this, especially for children or adolescents?
"Very much so. When a child gets sick, the whole family gets sick, so healing one child means healing the whole family. In all pediatric oncology centers, there are now volunteer associations and teams of psychologists whose mission is to support parents, siblings, and grandparents. Juvenal said, 'maxima debetur puero reverentia' (the child is owed the utmost care), a maxim we must all always uphold."
For over 40 years, you've worked every day alongside the pain and suffering of others, always in the National Health Service because you've always refused to pursue private practice. Have you ever faltered?
If by faltering you mean being tempted to quit this job, the answer is no. Luckily, that's never happened to me. Of course, I've suffered and continue to suffer when faced with certain situations where the outcome isn't as hoped, but every child we fail to heal is a motivation for me to continue to do better and to ensure the same unfortunate outcome doesn't happen again. The truth, ultimately, is that in my professional life, I consider myself privileged because I do a job I deeply love and because I receive so much from it every day, on a human level.