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Updated: Dec 21, 2025
Analyzing Tumor and Tissue Distribution of Target Antigen Specific Therapeutic Antibody
Published on: May 16, 2020
[Anti-FGF23 antibody therapy for patients with tumor-induced osteomalacia]
Yuka Kinoshita1, Seiji Fukumoto
1Division of Nephrology & Endocrinology, Department of Medicine, The University of Tokyo Hospital, Japan.
Abstract:
Tumor-induced osteomalacia (TIO) is a disease caused by fibroblast growth factor 23 (FGF23) secreted from the causative tumor. This disease is cured by complete surgical removal of the tumor. However, there are several difficult cases in which the responsible tumors cannot be found, are incompletely removed, or relapse after the surgery. Anti-FGF23 antibody is being studied as a novel therapy for FGF23-related hypophosphatemic diseases. The efficacy of anti-FGF23 antibodies were confirmed using a murine model of X-linked hypophosphatemic rickets (XLHR) , which is the most common heritable form of FGF23-related hypophosphatemic disease. In addition, results of phase I study of single injection of humanized anti-FGF23 antibody for adult patients with XLHR were recently published and the safety and effectiveness of this antibody was shown. This antibody therapy may be useful for patients with TIO with similar pathogenesis to that of XLHR.
Insights
Tumor-induced osteomalacia (TIO) is a rare bone disease caused by excess fibroblast growth factor 23 (FGF23). Anti-FGF23 antibody therapy shows promise for TIO patients, particularly when surgery is not fully effective.
Area of Science:
- Endocrinology
- Oncology
- Metabolic Bone Disease
Background:
- Tumor-induced osteomalacia (TIO) is a rare paraneoplastic syndrome characterized by excessive fibroblast growth factor 23 (FGF23) production by a tumor.
- While surgical tumor resection is curative, challenges arise from occult tumors, incomplete removal, or recurrence, necessitating alternative treatments.
- FGF23-related hypophosphatemic diseases share a common pathogenesis involving FGF23 dysregulation.
Purpose of the Study:
- To explore the potential of anti-FGF23 antibody therapy as a novel treatment for Tumor-induced osteomalacia (TIO).
- To evaluate the efficacy and safety of anti-FGF23 antibodies in FGF23-related hypophosphatemic conditions.
Main Methods:
- Confirmation of anti-FGF23 antibody efficacy in a murine model of X-linked hypophosphatemic rickets (XLHR).
- Review of Phase I clinical trial data for humanized anti-FGF23 antibody in adult XLHR patients.
Main Results:
- Anti-FGF23 antibodies demonstrated efficacy in a preclinical model of FGF23-related hypophosphatemic disease.
- Phase I human trials showed the safety and effectiveness of a single injection of humanized anti-FGF23 antibody in XLHR patients.
Conclusions:
- Anti-FGF23 antibody therapy is a potential novel treatment for Tumor-induced osteomalacia (TIO).
- This therapeutic approach may benefit TIO patients with similar FGF23-driven pathology to XLHR.
