京大：iPS細胞から血小板を作り投与：’Megakaryon’（動画）：
Kyoto Univ: Platelets made from iPS cells､administered: ‘Megakaryon’:
京都大学：血小板由iPS细胞制成，并经管理：Venture’Megakaryon’
ー止血に必要な血小板を大量生産ー
ー年内にも治験開始へー
京大のベンチャー：
iPS細胞から血小板という血液の成分を作り出します。
これを患者に投与する治療法の治験計画が開始しました。
「Megakaryon」：
京都市のバイオベンチャー企業「Megakaryon」が4月26日、発表しました。
- 日本の政府機関調査が終わったことから、
- 医療機関審査を経たうえで、
- 年内に治験を始めたいとしています。
iPS細胞から血小板作製：
この会社では、iPS細胞から血液の成分の一つで、止血に必要な血小板を作り出します。
「血小板減少症」患者の治療法について、国の承認を得るため、治験計画を進めています。
京大がiPS細胞を提供：
治験計画では、
- 第三者のiPS細胞から、
- 血小板を作り出し、
- 成人の男女10人に投与して、
1年間かけ、安全性や有効性を確認します。
血小板の大量生産：
従来、治療に使う血小板は、献血に頼っていました。
iPS細胞から作り出すことができれば大量生産も可能になります。
医療 | NHKニュース
https://www3.nhk.or.jp/news/html/20210426/k10012997531000.html
Megakaryon Corporation announces the completion of 30-day review of Clinical trial notification submitted for human iPS cell-derived HLA homozygous platelets (MEG-002).
26th April 2021
Overview
Megakaryon Corporation
today announces the completion of 30-day review of the clinical trial notification submitted for human iPS cell-derived HLA homozygous platelets (Development Code: MEG-002)
by Japan’s Pharmaceutical and Medical Devices Agency (PMDA).
Megakaryon Corporation
has planned this trial MEG-002 in collaboration with Kyoto University Hospital,
Center for iPS Cell Research and Application, Kyoto University(CiRA) and CiRA Foundation(CiRA_F).
We will continue to work closely with related parties and work diligently toward conducting the clinical trial.
Background
The Department of Hematology, Kyoto University Hospital (Professor Akifumi Takaori)and CiRA (Professor Koji Eto)
have conducted clinical research for the transfer of autologous iPS cell-derived platelets to a thrombocytopenia patient to verify the safety of iPS cell-derived platelet preparations.
The clinical trial
announced today is for an allogeneic transplant, which will enable industrialization of iPS cell-derived platelet and subsequent availability to a large number of people.
Summary of the clinical trial
In the clinical trial, we will confirm the safety and estimate the efficacy of MEG-002 to thrombocytopenia patients.
MEG-002 is prepared from an iPS cell provided by CiRA (currently CiRA_F) and consists of platelets with the HLA type most common in Japanese people.
The technology for producing platelets from iPS cells invented by Professor Koji Eto
is used for the development of MEG-002. The clinical trial will be conducted at multiple medical institutions, including the Department of Hematology, Kyoto University Hospital using product manufactured by CiRA_F.
Support for the clinical trial
The practical application of human iPS cell-derived HLA homozygous platelets
will be implemented with the support of the 5th Cyclic Innovation for Clinical Empowerment (CiCLE) program of Japan Agency for Medical Research and Development (AMED).
Megakaryon Corporation
was established in 2011 with the aim of utilizing technologies invented by Professor Koji Eto and others for producing platelets from human iPS cells for clinical application.
By developing large-scale manufacturing of human iPS-derived platelets with no risk of infection, we aim to supply platelets to medical facilities around the world.
Human iPS-derived platelets
Human iPS cell-derived platelets are produced by maturing megakaryocytes cultured from a master cell bank (MCB).
MCB is made from cryopreservable immortalized megakaryocyte precursor cells obtained by introducing three genes into hematopoietic precursor cells differentiated from human iPS cells.
Platelets, Thrombocytopenia
Platelets, also known as thrombocytes, are major blood components that play a crucial role in hemostasis.
Upon endothelial damage, platelets are activated leading to adhesion and aggregation at the wound site, thereby stopping bleeding.
Thrombocytopenia is a condition in which the number of platelets in the blood is low.
If the platelet count drops below a certain level or if there is a high risk of bleeding, treatment with blood transfusion platelet preparations is given.
http://www.megakaryon.com/en/dataroom/pdf/mega_release_20210426_en.pdf