International R&D core of gene therapy and cell therapy for cancer & intractable diseases

The IMSUT Hospital has been playing a lead role in gene therapy and hematopoietic stem cell transplantation in Japan. In order to strengthen this clinical development even further, IMSUT established the Center for Gene & Cell Therapy (CGCT) in 2014. CGCT particularly focuses on the development of gene therapy/cell therapy for intractable cancer and chronic diseases, e.g. oncolytic virotherapy for brain tumor, T-cell therapy, gene therapy for ALS, sarcopenia, and myasthenia or neurodegenerative disorders and hemophilia using AAV vectors, and cell therapy for muscular dystrophy using mesenchymal cells.

IMSUT Hospital, Advanced Clinical Research Center, Center for Stem Cell Biology and Regenerative Medicine

1. Nitahara-Kasahara Y, Kuraoka M, Oda Y, Hayashita-Kinoh H, Takeda S, Okada T. Enhanced cell survival and therapeutic benefits of IL-10-expressing multipotent mesenchymal stromal cells for muscular dystrophy. Stem Cell Res Ther 12:105, 2021.
2. Nitahara-Kasahara Y, Kuraoka M, Posadas GH, Hayashita-Kinoh H, Maruoka Y, Nakamura Takahashi A, Kimura K, Takeda S, Okada T. Dental pulp stem cells can improve muscle dysfunction in animal models of Duchenne muscular dystrophy. Stem Cell Res Ther 12:78, 2021.
3. Kato T, Nakamori M, Matsumura S, Nakamura M, Ojima T, Fukuhara H, Ino Y, Todo T, Yamaue H: Oncolytic virotherapy with human telomerase reverse transcriptase promoter regulation enhances cytotoxic effects against gastric cancer. Oncol Lett. 21:490, 2021.
4. Shiga M, Miyazaki J, Tanuma K, Nagumo Y, Yoshino T, Kandori S, Negoro H, Kojima T, Tanaka R, Okiyama N, Fujisawa Y, Watanabe M, Yamasaki S, Kiyohara H, Watanabe M, Sato TA, Tahara H, Nishiyama H, Yano I. The liposome of trehalose dimycolate extracted from M. bovis BCG induces antitumor immunity via the activation of dendritic cells and CD8+ T cells. Cancer Immunol Immunother. 2021.
5. Ishino R, Kawase Y, Kitawaki T, Sugimoto N, Oku M, Uchida S, Imataki O, Matsuoka A, Taoka T, Kawakami K, van Kuppevelt TH, Todo T, Takaori-Kondo A, Kadowaki N: Oncolytic Virus Therapy with HSV-1 for Hematological Malignancies. Mol Ther. 29:762-774, 2021.
6. Ishino R, Kawase Y, Kitawaki T, Sugimoto N, Oku M, Uchida S, Imataki O, Matsuoka A, Taoka T, Kawakami K, van Kuppevelt TH, Todo T, Takaori-Kondo A, Kadowaki N: Oncolytic Virus Therapy with HSV-1 for Hematological Malignancies. Mol Ther. 29:762-774, 2021.
7. Ueta R, Sugita S, Minegishi Y, Shimotoyodome A, Ota N, Ogiso N, Eguchi T, Yamanashi Y. DOK7 Gene Therapy Enhances Neuromuscular Junction Innervation and Motor Function in Aged Mice. iScience. 23(8):101385, 2020.
8. Hayashita-Kinoh H, Guillermo PH, Nitahara-Kasahara Y, Kuraoka M, Okada H, Chiyo T, Takeda S, Okada T. Improved transduction of canine X-linked muscular dystrophy with rAAV9-microdystrophin via multipotent MSC pretreatment. Mol Ther Methods Clin Dev. 2020, 20:133-141.
9. Hayashi, Y., Goyama, S., Liu, X., Tamura, M., Asada, S., Tanaka, Y., Fukuyama, T., Wunderlich, M., O'Brien, E., Mizukawa, B., Yamazaki, S., Matsumoto, A., Yamasaki, S., Shibata, T., Matsuda, K., Sashida, G., Takizawa, H. and Kitamura, T. Antitumor immunity augments the therapeutic effects of p53 activation on acute myeloid leukemia. Nat Commun. 10:4869. 2019.

1. Yuji Yamanashi, et al., inventors; DNA Encoding Polypeptide Capable of Modulating Muscle-specific Tyrosine Kinase Activity. Patent No.520460. Issue date: Apr 26, 2007.
2. Nahomi Nakatani, Yoshihiro Yoshikawa, Arinobu Tojo, Akio Shirasu, Tokiko Nagamura, inventors; Production method and expansion devices for regulatory T cell. Patent No. 010523. Issue date: Jan 21, 2009.
3. Shinichi Muramatsu, inventors; Adeno-associated virus virion for gene transfer into nervous system cells. Patent No. 215618. Issue date: Oct 22, 2014.
4. Kiwamu Imagawa, Takashi Okada, Yuko Kasahara, inventors; Therapeutic agent containing dental pulp-derived pluripotent stem cells for muscular dystrophy. Patent No. 559128. Issue date: Nov 13, 2015.
5. Takashi Okada, Shin-ichi Takeda, Hiromi Kinoh, inventors; Drug delivery particles and their manufacturing methods. Patent No. 165350. Issue date: Aug 26, 2016.
6. Takashi Okada, inventors; Nucleic acid-encapsulating AAV empty vector particles. Patent No. 215618. Issue date: Jan 29, 2018.
7. Tomoki Todo, inventors; Oncolytic virus therapy with suppression of tumor swelling. Patent No. 511025. Issue date: Mar 28, 2019.