Acute lymphoblastic leukaemia of childhood represents one of the most dramatic examples of the success of modern oncology. In particular now in the different countries we are able to cure at least 80-85% of patients with the front line regimens and another 5-10% of children can be salvaged by rescue therapies including stem cell transplantation. This dramatic improvement in the outcome of patients has been mainly the result of international co-operation, conduction of randomised clinical trials and integration of biology knowledge into the therapeutic strategies of these patients.
Nowadays in particular, clearance of leukaemia cells measured through the surrogate marker of minimal residual disease and information deriving from the presence of recurring genetic lesions permits to stratify the patients into different groups of risk, modulating the intensity of therapy according to the need of being more or less aggressive. Despite this dramatic improvement there is still a need to find innovative approaches for treating some specific subgroups of patients who still have an unsatisfactory or even dismal outcome. This is a wonderful period for clinicians because we can count not only on the classical mainstay of chemotherapy rather than stem cell transplantation but also because we have the possibility of integrating approaches of immunotherapy in the treatment of the majority of patients with acute lymphoblastic leukaemia. Indeed, 80% of patients have a leukaemia deriving from B-cell precursors and these patients can benefit from treatment with either monoclonal antibodies or with T-lymphocytes genetically modified for being redirected against the tumour target.
The most exciting development certainly represents the bio-immunotherapy from one side because I am convinced that these approaches of immunotherapy will significantly contribute to further improve the outcome of patients, and maybe that they will even substitute in certain patients stem cell transplantation, and by the possibility of targeting with molecular directed drugs specifically recurrent genetic lesions. In particular, we now know that a subgroup of patients, namely those with the so-called Ph-like acute lymphoblastic leukaemia, could benefit from treatment with tyrosine kinase inhibitors or JAK1 or JAK2 inhibitors.
The main message that I’d like to transmit is that we have to continue to collaborate together to exchange data and information, to implement well-conducted clinical trials with the great goal that we have to pursue of maintaining or even improving the cure rate that we have obtained while decreasing the long-term toxicity that still some of our patients experience in the long term.