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Current and Developing Pharmacologic Agents for Improving Skeletal Health in Adults with Osteogenesis Imperfecta
Winnie Liu1, Lindsey Nicol2, Eric Orwoll3
1Department of Medicine, Division of Endocrinology, Diabetes & Clinical Nutrition, Oregon Health & Science University, Portland, OR, USA. lwin@ohsu.edu.
Abstract:
Osteogenesis imperfecta (OI) is a genetic disorder characterized by increased bone fragility largely caused by defects in structure, synthesis, or post-translational processing of type I collagen. Drugs currently used to improve skeletal health in OI were initially developed to treat osteoporosis and clinical trials are ongoing to study their effectiveness in OI adults. Additionally, novel bone-protective agents are in preclinical studies and various phases of OI clinical trials. This review summarizes current knowledge on available pharmacologic agents and current drug trials involving OI participants. A PubMed online database search of all study types published in the English language using the terms "osteogenesis imperfecta," "OI," and "brittle bone disease" was performed in August 2022. Articles screened were restricted to adults. A ClinicalTrials.gov database search of all studies involving "osteogenesis imperfecta" was performed in August 2023. Although clinical trial data are limited, bisphosphonates and teriparatide may be useful in improving bone mineral density. As of yet, no clinical trials are available that adequately evaluate the usefulness of current therapies in reducing fracture risk. Several therapeutics, including teriparatide, setrusumab, anti-TGF-β antibodies, and allogeneic stem cells, are being studied in clinical trials. Preclinical studies involving Dickkopf-1 antagonists present promising data in non-OI bone disease, and could be useful in OI. Research is ongoing to improve therapeutic options for adults with OI and clinical trials involving gene-editing may be possible in the coming decade.
Insights
Osteogenesis imperfecta (OI) treatments are evolving. Current osteoporosis drugs may help bone density, but new agents and gene therapies are under investigation for brittle bone disease in adults.
Area of Science:
- Medical Science
- Genetics
- Pharmacology
Background:
- Osteogenesis imperfecta (OI) is a genetic disorder causing brittle bones due to type I collagen defects.
- Current treatments for OI were repurposed from osteoporosis, with ongoing trials for adult efficacy.
- Novel bone-protective agents are in preclinical and clinical development for OI.
Purpose of the Study:
- To review current pharmacologic agents for OI.
- To summarize ongoing drug trials for adult OI participants.
- To explore emerging therapeutic options for brittle bone disease.
Main Methods:
- PubMed database search (August 2022) for "osteogenesis imperfecta," "OI," and "brittle bone disease" in English, adult studies.
- ClinicalTrials.gov search (August 2023) for "osteogenesis imperfecta" studies.
- Literature review and synthesis of available data.
Main Results:
- Limited clinical trial data exist for current OI therapies.
- Bisphosphonates and teriparatide show potential for improving bone mineral density.
- Several agents (teriparatide, setrusumab, anti-TGF-β antibodies, stem cells) are in clinical trials.
- Dickkopf-1 antagonists show promise in preclinical studies for bone disease.
Conclusions:
- Further research is needed to evaluate current therapies' effectiveness in reducing fracture risk in OI adults.
- Multiple novel therapeutics are under investigation, offering hope for improved OI management.
- Gene-editing therapies may become a future treatment option for osteogenesis imperfecta.
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