黄斑変性に対する胚性幹細胞試験: 予備報告

Steven D Schwartz¹, Jean-Pierre Hubschman, Gad Heilwell

¹Jules Stein Eye Institute Retina Division, Department of Ophthalmology, David Geffen School of Medicine, University of California, Los Angeles, CA 90095, USA.

Lancet (London, England)

|January 28, 2012

まとめ

ヒト胚性幹細胞由来網膜色素表皮質 (hESC-RPE) 細胞を,マキュラ・ディストロフィー症の患者に移植した. hESC-RPE細胞は,初期の臨床試験で安全性と潜在的視力改善を示した.

科学分野:

オフタルモロジック (眼科)
再生医学は,再生医療である.
幹細胞生物学幹細胞生物学

背景:

人間の胚性幹細胞 (hESCs) は13年前に発見されました.
本報告書は,ヒト患者に対するhESC由来細胞の初めての移植について詳しく述べています.
この研究は,失明状態の治療のためのhESC由来網膜色素上皮質 (RPE) に焦点を当てています.

研究の目的:

hESC由来RPEのサブレテナル移植の安全性と耐性を評価する.
スターガルトの黄斑変性および乾燥した年齢関連の黄斑変性を持つ患者におけるhESC-RPEの潜在的な有効性を評価する.
hESC-RPE移植のための臨床プロトコルを作成する.

主な方法:

2つの前向きな臨床試験が開始されました (ClinicalTrials.gov:NCT01345006,NCT01344993).
>99%の純粋で,制御されたhESCで分化されたRPE細胞の網膜下移植.
術前および術後の眼科検査は,視覚の敏度,光素血管撮影,OCT,視野検査を含む.

主要な成果:

移植されたhESC-RPE細胞はホストのRPE層に統合され,持続しました.
4か月以内の患者では,過剰増殖,腫瘍発生性,または免疫拒絶の兆候は見られなかった.
スターガルトの患者は手動きから20/800まで改善し,AMDの患者は21から28のETDRS文字まで改善した.

結論:

hESC由来のRPE細胞は,マキュラ・ディストロフィー症の患者に安全でよく耐えます.
初期の証拠は,視力の改善と光受容体の救済の可能性を示唆しています.
将来の治療戦略は,病気の進行に早期に介入することを目的としています.

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