Overview of Current Targeted Anti-Cancer Drugs for Therapy in Onco-Hematology

Stefania Crisci, Filomena Amitrano, Mariangela Saggese, Tommaso Muto, Sabrina Sarno, Sara Mele, Pasquale Vitale, Giuseppina Ronga, Massimiliano Berretta, Raffaele Di Francia

Research output: Contribution to journalArticlepeer-review


The upgraded knowledge of tumor biology and microenviroment provides information on differences in neoplastic and normal cells. Thus, the need to target these differences led to the development of novel molecules (targeted therapy) active against the neoplastic cells' inner workings. There are several types of targeted agents, including Small Molecules Inhibitors (SMIs), monoclonal antibodies (mAbs), interfering RNA (iRNA) molecules and microRNA. In the clinical practice, these new medicines generate a multilayered step in pharmacokinetics (PK), which encompasses a broad individual PK variability, and unpredictable outcomes according to the pharmacogenetics (PG) profile of the patient (e.g., cytochrome P450 enzyme), and to patient characteristics such as adherence to treatment and environmental factors. This review focuses on the use of targeted agents in-human phase I/II/III clinical trials in cancer-hematology. Thus, it outlines the up-to-date anticancer drugs suitable for targeted therapies and the most recent finding in pharmacogenomics related to drug response. Besides, a summary assessment of the genotyping costs has been discussed. Targeted therapy seems to be an effective and less toxic therapeutic approach in onco-hematology. The identification of individual PG profile should be a new resource for oncologists to make treatment decisions for the patients to minimize the toxicity and or inefficacy of therapy. This could allow the clinicians to evaluate benefits and restrictions, regarding costs and applicability, of the most suitable pharmacological approach for performing a tailor-made therapy.
Original languageEnglish
Article number414
Number of pages28
JournalMedicina (Kaunas, Lithuania)
Issue number8
Publication statusPublished - Jul 28 2019


  • Tyrosine kinase inhibitors
  • anticancer mAbs
  • personalized medicine
  • pharmacogenomics
  • tailored therapy
  • Antineoplastic Agents/pharmacology/*therapeutic use
  • Antineoplastic Agents, Immunological/pharmacology/therapeutic use
  • Humans
  • MicroRNAs/pharmacology/therapeutic use
  • Oncolytic Viruses
  • RNA, Small Interfering/pharmacology/therapeutic use
  • Review Literature as Topic
  • Targeted Gene Repair/*methods/statistics & numerical data


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