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Avances en el tratamiento con oligonucleótidos antisentido para el cáncer
Lu Zhu1,2, Pariha Muhtar1,2,3, Susumu Goyama2,4
1Division of Cancer RNA Research, National Cancer Center Research Institute, 5-1-1 Tsukiji, Chuo-ku, Tokyo 104-0045, Japan.
Japanese journal of clinical oncology
|February 3, 2026
Resumen
Los oligonucleótidos antisentido (ASO) son una clase prometedora de ARN terapéuticos. La investigación está acelerando el desarrollo de ASO para el cáncer al dirigirse al empalme de ARN aberrante, con muchos ensayos clínicos en curso.
Área de la Ciencia:
- Ciencias Biomédicas
- Biología Molecular
- Oncología
Sus antecedentes:
- Los ARN terapéuticos, incluidos los oligonucleótidos antisentido (ASO), representan un avance significativo en el desarrollo de fármacos, y varios ya están aprobados para uso clínico.
- Si bien la mayoría de las terapias ASO aprobadas se dirigen a afecciones no malignas como las enfermedades neurodegenerativas, solo una está aprobada actualmente para el tratamiento del cáncer.
- El empalme de ARN aberrante se reconoce cada vez más como un factor clave en el desarrollo del cáncer, lo que destaca una oportunidad terapéutica.
Objetivo del estudio:
- Revisar los avances recientes en la comprensión de la biología del cáncer relevante para las terapias basadas en ASO.
- Destacar el progreso en el desarrollo de enfoques dirigidos al ARN para el tratamiento del cáncer.
- Subrayar el potencial de los ASO terapéuticos en oncología.
Principales métodos:
- Revisión de la literatura de publicaciones científicas recientes y datos de ensayos clínicos.
- Análisis del papel del empalme de ARN aberrante en la patogénesis del cáncer.
- Síntesis de la investigación actual sobre el desarrollo de ASO para indicaciones oncológicas.
Principales resultados:
- Casi la mitad de los ARN terapéuticos actuales modulan el empalme del ARN.
- Más de 250 ensayos clínicos han investigado agentes de oligonucleótidos para diversas moléculas asociadas con el cáncer.
- Los resultados preliminares de los ensayos clínicos para terapias contra el cáncer basadas en ASO muestran resultados alentadores.
Conclusiones:
- El desarrollo de ASO terapéuticos para el cáncer está preparado para acelerarse debido al papel del empalme de ARN en la oncogénesis.
- La investigación en curso y los ensayos clínicos están allanando el camino para nuevos tratamientos contra el cáncer dirigidos al ARN.
- La tecnología ASO ofrece una vía prometedora para abordar las necesidades no satisfechas en la terapia contra el cáncer.
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