Novel therapeutic strategy based on precision medicine in bone and soft tissue sarcomas(Fostering Joint International Research)

2019 Fiscal Year Final Research Report

• Project/Area Number: 15KK0353
• Research Category: Fund for the Promotion of Joint International Research (Fostering Joint International Research)
• Allocation Type: Multi-year Fund
• Research Field: Orthopaedic surgery
• Research Institution: Juntendo University
• Principal Investigator: Suehara Yoshiyuki 順天堂大学, 医学部, 准教授 (70509405)
• Project Period (FY): 2016 – 2019
• Keywords: 骨軟部腫瘍 / 骨肉腫 / プレシジョンメディシン / がんクリニカルシークエンス / MSK-IMPACT / チロシンキナーゼ阻害剤

Outline of Final Research Achievements

The prognosis of bone and soft tissue sarcomas are still extremely poor. As sarcomas is a rare cancer, the development of novel treatments has been delayed due to the limited amount of data and the associated negative efforts. In recent years, precision medicine based on a cancer clinical sequence, which uses a next-generation sequencer has been developed in across all cancer types. Additionally, in Japan, cancer gene panel tests were recently approved in government health care.
In this study, we mainly performed genome-based profiling of bone soft tissue sarcomas using MSK-IMPAC, which is a cancer clinical sequence and approved by FDA. These studies elucidated the frequency of potential actionable gene alteration based on OncoKB in each bone and soft tissue sarcomas as well as identified novel therapeutic targets and strategies based on gene profiling in osteosarcoma.

Free Research Field

整形外科

Academic Significance and Societal Importance of the Research Achievements

本研究結果は、現在までに不明であったがんクリニカルシークエンスに基づいた骨軟部肉腫の治療適応可能遺伝子変化の頻度を明らかにした。骨肉腫にいては新規標的治療を同定しその適応対象が約40％の骨肉腫患者に存在することを明らかにした。特に染色体4q12領域のKIT、KDR、PDGFRA遺伝子、染色体6p12-21領域のVEGFA遺伝子の標的治療薬（阻害剤）は、臨床試験を経て新規治療法の開発へ繋がる可能性を大きく含んでおり、それらはゲノムベースアルゴリズムの基に理論的に薬剤を使用するがん個別化医療で展開することが可能となる 。