The Current Landscape of Early Drug Development for Patients With Sarcoma

Am Soc Clin Oncol Educ Book. 2017;37:807-810. doi: 10.1200/EDBK_174701.


Until recently, advancements in the treatment of patients with adult soft tissue sarcomas have been relatively slow. This is, in part, due to their heterogeneity and rarity. A better understanding of the biology and differences among the various histologies has led to substantial growth in novel strategies. In addition to novel cytotoxic chemotherapies, agents targeting platelet-derived growth factor receptor-α (PDGFRα), mTOR, and angiogenesis are areas of active investigation. Additionally, with the success of checkpoint inhibitors in other malignancies and early encouraging results of checkpoint inhibitors in some sarcoma subtypes, this approach is being widely investigated in various sarcomas. As we increasingly recognize and treat each sarcoma histology as a separate disease, it is important to spread awareness of the exciting clinical trials available to our patients with these rare malignancies.

Publication types

  • Review

MeSH terms

  • Adult
  • Antineoplastic Agents / therapeutic use*
  • Drug Discovery / trends
  • Humans
  • Neovascularization, Pathologic / drug therapy*
  • Neovascularization, Pathologic / genetics
  • Neovascularization, Pathologic / pathology
  • Receptor, Platelet-Derived Growth Factor alpha / antagonists & inhibitors
  • Receptor, Platelet-Derived Growth Factor alpha / genetics
  • Sarcoma / drug therapy*
  • Sarcoma / genetics
  • Sarcoma / pathology
  • TOR Serine-Threonine Kinases / antagonists & inhibitors
  • TOR Serine-Threonine Kinases / genetics


  • Antineoplastic Agents
  • MTOR protein, human
  • TOR Serine-Threonine Kinases
  • Receptor, Platelet-Derived Growth Factor alpha