A new targeted therapy for acute myeloid leukaemia

International study, coordinated for Italy by the haematologists of Tor Vergata, published in the New England Journal of Medicine
 
A molecular "target" drug, a new targeted therapy, will soon be available for about a third of the patients with acute myeloid leukaemia. In addition to conventional chemotherapy, this drug (midostaurin) has produced a significant benefit in the survival of patients with this disease, who exhibit specific mutations of a receptor, called FLT3.
 
The results of the international clinical study showing the benefits of this therapy have just been published on the New England Journal of Medicine, the most prestigious medical journal in the world. The randomized controlled trial assessed the effectiveness and tolerability of a FLT3 inhibitor against placebo in patients aged 18 to 60, in combination with chemotherapy.
 
The study saw the participation of 12 national co-operative groups and was supported by the National Cancer Institute in the USA and Novartis in Europe. The important Italian contribution is witnessed by the participation of 25 Centres that, through the cooperative group GIMEMA (Italian Group for the Study of Adult Haematological Diseases), enrolled 105 of the 717 patients studied. Co-authors of the newly published scientific work, as representatives of Italian haematologists, are Prof. Francesco Lo Coco, who has centralized at the Tor Vergata's laboratories the diagnostics and therefore the selection of the suitable patients, for all the Centres GIMEMA, and Prof. Sergio Amadori, Vice President of GIMEMA and Coordinator of the study Group of Acute Leukaemia.
 
It is worth noting that, thanks to this work, "midostaurin" has recently been approved in the USA by the Food and Drug Administration and will soon be approved as a new drug for the treatment of patients with acute myeloid leukaemia in Europe as well.
This has not happened for nearly 20 years!

Prof. William Arcese
Director
Haematology and Stem Cell Transplantation