Related Experiment Video
Updated: May 20, 2026
Protocol and Guidelines for Point-of-Care Lung Ultrasound in Diagnosing Neonatal Pulmonary Diseases Based on International Expert Consensus
Published on: March 6, 2019
Clinical expert panel on monitoring potential lung toxicity of inhaled oligonucleotides: consensus points and
Eric W Alton1, Homer A Boushey, Holger Garn
1Imperial College, London, United Kingdom.
Abstract:
Oligonucleotides (ONs) are an emerging class of drugs being developed for the treatment of a wide variety of diseases including the treatment of respiratory diseases by the inhalation route. As a class, their toxicity on human lungs has not been fully characterized, and predictive toxicity biomarkers have not been identified. To that end, identification of sensitive methods and biomarkers that can detect toxicity in humans before any long term and/or irreversible side effects occur would be helpful. In light of the public's greater interests, the Inhalation Subcommittee of the Oligonucleotide Safety Working Group (OSWG) held expert panel discussions focusing on the potential toxicity of inhaled ONs and assessing the strengths and weaknesses of different monitoring techniques for use during the clinical evaluation of inhaled ON candidates. This white paper summarizes the key discussions and captures the panelists' perspectives and recommendations which, we propose, could be used as a framework to guide both industry and regulatory scientists in future clinical research to characterize and monitor the short and long term lung response to inhaled ONs.
Insights
Oligonucleotides (ONs) show promise for respiratory diseases but lung toxicity needs characterization. This paper proposes a framework for monitoring inhaled ONs to ensure patient safety and identify early toxicity biomarkers.
Area of Science:
- Pharmacology
- Toxicology
- Respiratory Medicine
Background:
- Oligonucleotides (ONs) are a developing drug class for treating diseases via inhalation.
- The lung toxicity of inhaled ONs and predictive biomarkers remain uncharacterized.
- Sensitive detection methods are needed to identify ON-induced lung toxicity before irreversible effects.
Framework:
- Expert panel discussions convened by the Oligonucleotide Safety Working Group (OSWG) focused on inhaled ON toxicity.
- Assessed strengths and weaknesses of various monitoring techniques for clinical evaluation of inhaled ON candidates.
- Proposed a framework for industry and regulatory scientists to guide future research on inhaled ON lung response.
Implementation:
- The white paper summarizes expert perspectives and recommendations.
- Focuses on characterizing short and long-term lung responses to inhaled ONs.
- Aims to establish standardized monitoring protocols for inhaled ONs.
Implications:
- Provides a guide for clinical research on inhaled ON safety.
- Facilitates early detection of potential lung toxicity.
- Supports the safe development of inhaled oligonucleotide therapeutics.
