Author: Ikeda, Kei ; Miyashita, Kazuya ; Watanabe, Suzuka ; Yokote, Koutaro ; Teramoto, Naoya ; Maezawa, Yoshiro ; Yamaguchi, Takashi ; Ogura, Masatsune ; Kobayashi, Junji ; Ishikawa, Takahiro ; Sato, Tetta ; Kami, Saori ; Aono, Kazuto ; Murakami, Masami ; Yamaguchi, Ayano ; Igarashi, Katsushi ; Nakajima, Katsuyuki ; Kaneko, Hiyori ; Miyabayashi, Makoto ; Hiraga, Chihiro

BACKGROUND:

Glycosylphosphatidylinositol-anchored high-density lipoprotein binding protein 1 (GPIHBP1) autoantibody syndrome is a rare autoimmune cause of severe hypertriglyceridemia and acute pancreatitis.

OBJECTIVE:

We report 2 patients who achieved long-term remission through distinct therapeutic approaches.

METHODS:

Case 1 received prednisolone, azathioprine, and hydroxychloroquine, maintaining remission and experienced an uneventful pregnancy while continuing hydroxychloroquine monotherapy for 4 years. Case 2, who suffered recurrent pancreatitis, was treated with rituximab and achieved sustained remission without maintenance therapy for 3 years.

RESULTS:

Epitope mapping identified distinct antibody epitopes in the 2 cases. The antibody from Case 1 targeted the Ly6/uPAR (LU) domain of GPIHBP1, whereas the antibody from Case 2 recognized an epitope involving not only the LU domain but also the region bordering the acidic domain.

CONCLUSION:

This is the first report demonstrating successful long-term remission of GPIHBP1 autoantibody syndrome, including during pregnancy. The clinical significance of epitope heterogeneity among GPIHBP1 autoantibodies remains to be clarified.

01 Jan 2026·Journal of Atherosclerosis and Thrombosis

A Case of Familial Chylomicronemia Syndrome Caused by a Novel Homozygous GPIHBP1 Mutation Successfully Treated with the Selective PPARα Modulator Pemafibrate

Article

Author: Yamanishi, Masayuki ; Hanada, Hiroyuki ; Yamaguchi, Atsuhiro ; Takano, Toru ; Yamashita, Shizuya ; Miyashita, Kazuya ; Komuku, Shiho ; Masuda, Daisaku ; Kawachi, Yusuke ; Otsuki, Tomoko ; Chung, Yumi

AIM:

Familial chylomicronemia syndrome (FCS) is a rare disorder characterized by the accumulation of chylomicrons in the circulation due to genetic defects or autoantibodies affecting lipoprotein lipase (LPL), the key enzyme responsible for the metabolism of chylomicrons and very-low-density lipoproteins (VLDL), or its associated proteins, including apolipoprotein (apo) C-II, apoA-V, glycosylphosphatidylinositol-anchored high-density lipoprotein-binding protein 1 (GPIHBP1), and lipase maturation factor 1 (LMF1). This condition is associated with markedly elevated serum triglyceride (TG) levels and, in severe cases, recurrent episodes of pancreatitis and eruptive xanthomas. Among these etiologies, genetic deficiency of GPIHBP1 is exceedingly rare. We aimed to clarify the clinical and genetic basis and treatment strategies of a patient with GPIHBP1 deficiency.

METHODS:

A 47-year-old woman with a childhood diagnosis of FCS presented with severe epigastric pain and was admitted to Rinku General Medical Center for emergency management of acute pancreatitis secondary to severe chylomicronemia. She had a history of severe hypertriglyceridemia complicated by pancreatitis during her first pregnancy and extreme hypertriglyceridemia exceeding 6,000 mg/dL during her second pregnancy without the development of pancreatitis. DNA sequence analysis was performed as a clinical diagnostic test and outsourced to KUBIX Inc. (Hakusan, Ishikawa, Japan).

RESULTS:

Laboratory evaluation revealed marked hypertriglyceridemia (1,262 mg/dL) with a type V hyperlipoproteinemia pattern. Serum preheparin LPL mass was markedly reduced, and circulating GPIHBP1 was undetectable. Genetic analysis identified a novel homozygous frameshift mutation in the GPIHBP1 gene (NM_178172.4:c.20del [p.Val7AlafsTer73]), which has not been previously reported in public databases. No pathogenic variants were detected in other LPL-related genes. Acute pancreatitis improved rapidly with fasting and intravenous fluid therapy. Strict dietary fat restriction, followed by the addition of the selective PPARα modulator pemafibrate, successfully maintained TG levels below 500 mg/dL during follow-up.

CONCLUSION:

We report an extremely rare case of FCS caused by a novel homozygous mutation in the GPIHBP1 gene, in which pregnancy served as a major trigger for severe hypertriglyceridemia. Early diagnosis and strict lipid control are essential to prevent recurrent pancreatitis in patients with GPIHBP1 deficiency.

01 Aug 2025·JOURNAL OF GASTROENTEROLOGY

Molecular imaging of gastrointestinal stromal tumor using anti-c-KIT antibody and its fragments

Article

Author: Yasushi Sato ; Naoki Muguruma ; Tatsuya Segawa ; Takanori Miyake ; Mamoru Shimizu ; Takanori Kashihara ; Shota Fujimoto ; Tetsuji Takayama ; Koichi Okamoto ; Yutaka Kawano

BACKGROUND:

Gastrointestinal stromal tumors (GISTs) are malignant subepithelial tumors, known for their poor prognosis due to distant metastasis. Because GIST is covered by a normal mucosal layer, effective tissue biopsy under conventional endoscopy is difficult, thereby leading to delayed diagnosis and a dismal prognosis. We performed molecular imaging of GIST targeting c-KIT using fluorescence-labeled anti-c-KIT antibody/fragments and fluorescent endoscopy.

METHODS:

Mouse anti-human c-KIT monoclonal antibody, its F(ab')₂ and Fab fragments were labeled with AF680. Two GIST cell lines (GIST-T1, GIST-882M) were used for experiments. Antibodies were intravenously administered to mice xenografted with GIST-T1 or GIST-882M, and each tumor was observed using IVIS Spectrum and self-developed simple fluorescent endoscopy.

RESULTS:

The GIST-T1 cell live imaging revealed strong signals on cell membranes after 1 min incubation, and thereafter, they aggregated and internalized inside the cells within 130 min in all antibody/fragment groups. In vivo mouse experiments, AF680-labeled IgG slowly accumulated in tumors peaking at 24 h after injection. However, AF680-labeled F(ab')₂ and Fab rapidly accumulated in tumors peaking at 1-2 h, and completely cleared from the body within 24 h. Fab showed the strongest fluorescence intensity in tumors. Fluorescence endoscopy could clearly detect GIST xenograft tumors 1-2 h after AF680-labeled F(ab')₂ and Fab injection.

CONCLUSIONS:

AF680-labeled antibody/fragments showed clear and specific fluorescence signals in GIST xenografts in mice. Particularly, AF680-labeled Fab showed the strongest signal intensity at 1-2 h post-administration and rapid clearance, suggestive of the safety. This approach may enable molecular imaging diagnosis of GIST by endoscopy in outpatient settings in the future.

News (Medical) associated with Immuno-Biological Laboratories Co., Ltd.

26 Oct 2009

·www.astellas.com

Astellas to Terminate the Development of Anti-Human Osteopontin Antibody ASK8007for RA

Japan, October 26, 2009 - Astellas Pharma Inc. (“Astellas”; headquarters: Tokyo; President and CEO: Masafumi Nogimori) today announced that it has decided to terminate the development of ASK8007 (development code), anti-human osteopontin antibody for rheumatoid arthritis (RA). It was co-developed with Chemo-Sero-Therapeutic Research Institute (“KAKETSUKEN”; headquarters: Kumamoto, Kumamoto Prefecture). Astellas and Immuno-Biological Laboratories Co., Ltd. (headquarters: Takasaki, Gunma Prefecture) concluded a worldwide licensing agreement which granted Astellas the exclusive rights to develop, manufacture, and market anti-human osteopontin antibodies, for therapeutic use in March 2006. Astellas and KAKETSUKEN carried out Phase I for ASK8007 for RA; however, have concluded terminating the development for the field of RA by evaluating the result of the study comprehensively. Astellas is now considering a possible application of it to other indications than RA. ####

License out/inPhase 1

100 Deals associated with Immuno-Biological Laboratories Co., Ltd.

100 Translational Medicine associated with Immuno-Biological Laboratories Co., Ltd.

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