Development of the novel biopharmacy for pulmonary hypertension

2023 Fiscal Year Final Research Report

• Project/Area Number: 21K19469
• Research Category: Grant-in-Aid for Challenging Research (Exploratory)
• Allocation Type: Multi-year Fund
• Review Section: Medium-sized Section 53:Organ-based internal medicine and related fields
• Research Institution: Tohoku University
• Principal Investigator: SATOH KIMIO 東北大学, 高度教養教育・学生支援機構, 非常勤講師 (80436120)
• Project Period (FY): 2021-07-09 – 2024-03-31
• Keywords: 肺高血圧

Outline of Final Research Achievements

Pulmonary arterial hypertension (PAH) is a highly fatal disease where lung transplantation is the only means of saving patients. However, due to the limited number of donors and the progression of right heart failure, many patients succumb to the disease. There are many severe cases where multi-drug therapy is ineffective, highlighting the urgent need for the development of fundamental treatments. Our previous research identified a group of genes with over fivefold increased expression in the pulmonary artery smooth muscle cells of PAH patients, which are involved in smooth muscle proliferation and endothelial cell dysfunction. Based on this, the development of new drugs with mechanisms different from existing therapies is anticipated. Specifically, the inhibition of newly discovered proteins A and B has shown to improve PAH, leading to the advancement of new treatment development.

Free Research Field

循環器内科学

Academic Significance and Societal Importance of the Research Achievements

本研究による肺動脈性肺高血圧症（PAH）の研究成果は、肺高血圧症の病態解明という点でも大きな意義を持つと考えられる。まず、PAHの病態メカニズムに関与する遺伝子群（蛋白Aや蛋白B）を特定し、これらが肺動脈血管平滑筋増殖や内皮細胞機能障害を引き起こすことを明らかにした。この発見は、PAHの発症と進行の根本原因を解明し、新たな治療ターゲットを提供した。さらに、遺伝子発現変動の網羅的解析を通じて、従来の血管拡張薬とは異なる作用機序を持つ新規治療薬の開発が可能となった。この成果は、PAH治療のパラダイムシフトを促進し、将来的な研究と臨床応用における重要な基盤を築くと考えられる。