Drug repositioning is a strategy to identify new uses for approved or investigational drugs that are used off-label outside the scope of the original medical indication.
In this review, we report the most relevant studies about drug repositioning in hematology, reporting the signalling pathways and molecular targets of these drugs, and describing the biological mechanisms which are responsible for their anticancer effects.
Although the majority of studies on drug repositioning in hematology concern acute myeloid leukemia and multiple myeloma, numerous studies are present in the literature on the possibility of using these drugs also in other hematological diseases, such as acute lymphoblastic leukemia, chronic myeloid leukemia, and lymphomas. Numerous anti-infectious drugs and chemical entities used for the therapy of neurological or endocrine diseases, oral antidiabetics, statins and medications used to treat high blood pressure and heart failure, bisphosphonate and natural substance such as artemisin and curcumin, have found a place in the treatment of hematological diseases. Moreover, several molecules drastically reversed the resistance of the tumor cells to the chemotherapeutic drugs both in vitro and in vivo.