| Project/Area Number |
18K15715
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| Research Category |
Grant-in-Aid for Early-Career Scientists
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| Allocation Type | Multi-year Fund |
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| Review Section |
Basic Section 52050:Embryonic medicine and pediatrics-related
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| Research Institution | Shimane University |
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Principal Investigator |
Oda Yasuaki 島根大学, 医学部, 助教 (70602473)
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| Project Period (FY) |
2018-04-01 – 2021-03-31
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| Project Status |
Completed (Fiscal Year 2020)
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| Budget Amount *help |
¥4,160,000 (Direct Cost: ¥3,200,000、Indirect Cost: ¥960,000)
Fiscal Year 2020: ¥520,000 (Direct Cost: ¥400,000、Indirect Cost: ¥120,000)
Fiscal Year 2019: ¥650,000 (Direct Cost: ¥500,000、Indirect Cost: ¥150,000)
Fiscal Year 2018: ¥2,990,000 (Direct Cost: ¥2,300,000、Indirect Cost: ¥690,000)
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| Keywords | 低ホスファターゼ症 / 再生医療 / 間葉系幹細胞 / 細胞移植 / iPS細胞 / 難病 |
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| Outline of Final Research Achievements |
Hypophosphatasia is a rare bone disease that presents with severe osteogenesis imperfecta due to mutations in the ALPL gene. In this study, using with hypophosphatasia model mice (Alpl -/- mice), we developed a basic technology for cell transplantation therapy using ultra-high-purity mesenchymal stem cells (MSC), which have extremely high differentiation, proliferation, and migration ability.
Mouse bone marrow cells and human ultra-high purity MSCs were transplanted into X-ray irradiated Alpl -/- mice. Two month later, I prepared frozen slices of mouse femur to examine alkaline phosphatase (ALP) activity, which is an indicator of bone-producing osteoblasts. ALP activity derived from human ultra-high purity MSC was detected only in the transplanted group. And the survival rate has also improved significantly.
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| Academic Significance and Societal Importance of the Research Achievements |
現在、低ホスファターゼ症の治療薬は酵素製剤（製品名：ストレンジック）のみであるが、生涯にわたって投薬が必要であり、投薬を続けることで中和抗体（薬の効果を打ち消してしまう抗体ができること）が出現する懸念がある。
本研究成果は、ヒト超高純度MSC移植という、低ホスファターゼ症に対する新たな治療法の選択肢を提案すると共に、併用することでより相乗的な効果が期待される。ヒト高純度MSC移植のみで治療が可能になれば、根治療法となり得る可能性も秘めている。低ホスファターゼ症のみならず、他の骨系統疾患にも適応できると考えられるため、学術的にも社会的にも大きな意義があると考える。
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