Combined treatment with mesenchymal stem cells and therapeutic hypothermia for neonatal hypoxic ischemic encephalopathy: a phase 1/2 randomized trial

Neonatal hypoxic ischemic encephalopathy (nHIE) is a serious disease that causes severe and chronic neurological damage. Hypothermia therapy improves patients' outcomes albeit with some limitations, but combining it with treatment with cord blood cells (analogous to mesenchymal stem cells [MSCs]) reportedly improves its effectiveness. TEMCELL HS Inj. (Temcell), a human bone marrow-derived MSC product used for acute graft-versus-host disease, seems an appropriate candidate for this combination therapy. Therefore, we performed a randomized, parallel-group study to compare combined treatment with Temcell and hypothermia versus hypothermia therapy-alone to evaluate the safety and efficacy of Temcell in nHIE patients. The primary endpoint was treatment response defined as an overall developmental quotient of ≥ 85 at 18 months of age. Fourteen patients were enrolled and randomized, with 7 assigned to each group. Both groups had similar demographic characteristics and nHIE severity. Treatment response was observed in 4 of the 6 (66.7%) patients in the Temcell combination group, and in 4 of the 7 patients (57.1%) in the hypothermia therapy-alone group. No marked differences in safety profile were observed between the groups. These results indicate that the efficacy of Temcell combined with hypothermia is comparable to therapeutic hypothermia for patients with nHIE.Clinical Trial Registration: jRCT1080224818.

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Publication Details

Journal
Scientific Reports
Published
2025-05-10
DOI
https://doi.org/10.1038/s41598-025-98504-z
Citations
2
Primary Topic
Neonatal and fetal brain pathology
Type
article
Field-Weighted Citation Impact
6.99

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article

Combined treatment with mesenchymal stem cells and therapeutic hypothermia for neonatal hypoxic ischemic encephalopathy: a phase 1/2 randomized trial

Hirofumi Ide, Kazuko Wada, Seiji Yoshimoto, Yukimichi Nakano et al.
2 citations
Scientific Reports
Neonatal and fetal brain pathology
6.99
article

Combined treatment with mesenchymal stem cells and therapeutic hypothermia for neonatal hypoxic ischemic encephalopathy: a phase 1/2 randomized trial

Hirofumi Ide, Kazuko Wada, Seiji Yoshimoto, Yukimichi Nakano, Natsuki Ohkawa, Masato Kantake, Kazumichi Fujioka, Yasumasa Yamada, Satoshi Kusuda, Toshiya Nishikubo, Hideaki Hirai, Kiyoaki Sumi, Takeo Mure, Akihito Takeuchi, Tomoaki Ioroi, Naoko Takasao, Yutaka Yamamoto, Yuji Sato, Tatsuyoshi Yamamoto, Norihisa Wada, Kenji Tada, Yoshinori Katayama
article en
2 citations

Abstract

Neonatal hypoxic ischemic encephalopathy (nHIE) is a serious disease that causes severe and chronic neurological damage. Hypothermia therapy improves patients' outcomes albeit with some limitations, but combining it with treatment with cord blood cells (analogous to mesenchymal stem cells [MSCs]) reportedly improves its effectiveness. TEMCELL HS Inj. (Temcell), a human bone marrow-derived MSC product used for acute graft-versus-host disease, seems an appropriate candidate for this combination therapy. Therefore, we performed a randomized, parallel-group study to compare combined treatment with Temcell and hypothermia versus hypothermia therapy-alone to evaluate the safety and efficacy of Temcell in nHIE patients. The primary endpoint was treatment response defined as an overall developmental quotient of ≥ 85 at 18 months of age. Fourteen patients were enrolled and randomized, with 7 assigned to each group. Both groups had similar demographic characteristics and nHIE severity. Treatment response was observed in 4 of the 6 (66.7%) patients in the Temcell combination group, and in 4 of the 7 patients (57.1%) in the hypothermia therapy-alone group. No marked differences in safety profile were observed between the groups. These results indicate that the efficacy of Temcell combined with hypothermia is comparable to therapeutic hypothermia for patients with nHIE.Clinical Trial Registration: jRCT1080224818.

Scientific ReportsVol. 15(1)
Aichi Medical University (JP), Kindai University Hospital (JP), JCR Pharmaceuticals (Japan) (JP), Chibune General Hospital (JP), Nara Medical University Hospital (JP), Kobe Children's Hospital (JP), Juntendo University Shizuoka Hospital (JP), Himeji Red Cross Hospital (JP), Gifu Prefectural General Medical Center (JP), Okayama Medical Center (JP), Toujinkai Hospital (JP), Juntendo University Nerima Hospital (JP), Takatsuki General Hospital (JP), Kobe University (JP)
JCR Pharmaceuticals, Keio University, Jichi Medical University
Good health and well-being
Openalex Percentile: Top 4%
Neonatal and fetal brain pathology
6.99
DOIOpen Access PDFOpenAlex
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