The development of novel therapeutic strategy for pediatric heart failure patients using ventricular assist devices and cardiac regnerative therapy.

• Project/Area Number: 24592061
• Research Category: Grant-in-Aid for Scientific Research (C)
• Allocation Type: Multi-year Fund
• Section: 一般
• Research Field: Thoracic surgery
• Research Institution: Osaka University
• Principal Investigator: UENO Takayoshi 大阪大学, 医学(系)研究科(研究院), 寄附講座准教授 (60437316)
• Co-Investigator(Kenkyū-buntansha): MIYAGAWA Shigeru 大阪大学, 大学院医学系研究科, 特任准教授(常勤) (70544237) ; IDE Haruki 大阪大学, 大学院医学系研究科, 特任助教 (90600122)
• Project Period (FY): 2012-04-01 – 2015-03-31
• Project Status: Completed (Fiscal Year 2014)
• Budget Amount: ¥5,200,000 (Direct Cost: ¥4,000,000、Indirect Cost: ¥1,200,000); Fiscal Year 2014: ¥2,080,000 (Direct Cost: ¥1,600,000、Indirect Cost: ¥480,000); Fiscal Year 2013: ¥1,040,000 (Direct Cost: ¥800,000、Indirect Cost: ¥240,000); Fiscal Year 2012: ¥2,080,000 (Direct Cost: ¥1,600,000、Indirect Cost: ¥480,000)
• Keywords: 再生医療 / 小児心不全 / 幹細胞治療 / 筋芽細胞シート / 小児拡張型心筋症 / 小児補助人工心臓 / 医師主導治験 / ヒト幹細胞を使用した臨床研究 / 医師主導型治験 / 対面助言 / 難治性疾患

Outline of Final Research Achievements

To establish novel therapeutic strategy for pediatric severe heart failure, we have completed pre-clinical studies regarding cardiac regenerative therapy. We have proved the safety and feasibility of novel synthetic prostacyclin agonist therapy for DCM rat and juvenile autologous skeletal muscle stem cell therapy for young ICM animal model. Those results implied the stem cell therapy for young patients will have greater therapeutic potentials than adult patients. We experienced a case of myoblast sheet implantation for DCM pediatric patient. No severe adverse events were found and her cardiac function and performance status improved during follow up period. Now we have been preparing for PI initiated clinical trial for approval to expand indication of the therapeutic strategy to pediatric heart failure patients.

Research Products

• [Presentation] 遠隔期からみた小児心臓移植待機患者に対する補助人工心臓の有用性の検討 (2015)
  • Author(s): 平 将生
  • Organizer: 第45回日本心臓血管外科学会学術集会
  • Place of Presentation: 国立京都国際会館
  • Year and Date: 2015-02-16 – 2015-02-18
• [Presentation] Surgical management for secondary pulmonary hypertension ina pediatric restrictive cardiomyopathy patient (2014)
  • Author(s): 平 将生
  • Organizer: 第18回日本心不全学会学術集会
  • Place of Presentation: 大阪国際会議場
  • Year and Date: 2014-10-10 – 2014-10-12
• [Presentation] 幼若ブタ虚血性心筋症モデルに対する自己骨格筋細胞シート移植の有用性の検討 (2014)
  • Author(s): 小澤 秀登
  • Organizer: 第13回再生医療学会
  • Place of Presentation: 国立京都国際会館
• [Presentation] 小児重症心不全に対する補助人工心臓使用経験 (2014)
  • Author(s): 平 将生
  • Organizer: 第42回人工心臓と補助循環懇話会学術集会
  • Place of Presentation: 新潟県 ナスパニューオータニ
• [Presentation] Juvenile Skeltal Stem Cell has Greater Therapeutic Potentials of Regenerative Therapy than Adult Shee for Treating Chronic Myocardial Infarction in Infarct Pig (2013)
  • Author(s): Ozawa Hideto
  • Organizer: American Heart Association 2013
  • Place of Presentation: Dallas Convention Center, USA
• [Presentation] "Novel Synthetic Prostacyclin Agonist, ONO1301, Inhibits Proliferation of Pulmonary Arterial Smooth Muscle Cells by Suppressing Raf signal pathway (2012)
  • Author(s): 平 将生
  • Organizer: American Heart Association 2012
  • Place of Presentation: Los Angels