Cancer treatment using antisense oligonucleotides to modulate alternative splicing
Centre for Genomic Regulation, Spain
Lung cancer is the leading cause of cancer mortality, accounting for nearly one in five cancer deaths. The most frequent subtype of this cancer is lung adenocarcinoma (40%), for which there is still no effective treatment. An innovative pharmacological approach to tackle tumour progression is a technology based on antisense oligonucleotides, which can correct alternative splicing alterations found on cancer.
To bring a new-targeted therapy for lung adenocarninoma to the market, based on antisense oligonucleotides (AON) acting selectively on key cancer related genes, an emerging target for cancer treatment.
Problem to Solve
Lung adenocarcinoma has high incidence and mortality rates, and is an important and challenging objective for innovative treatments. The lack of effective therapies and the resistance that has been acquired to existing treatments are obstacles that have yet to be overcome. To make progress on developing lung cancer treatments, new drugs should be improved in terms of specificity and side effects.
This project uses an innovative AON approach to target lung cancer. The use of a specific set of AON, that correct the alternative splicing of key cancer-related genes, has shown antitumor properties in a mouse model of lung cancer.
Level of Innovation
This is the first therapy for lung cancer that uses splicing-modifying AONs, paving the way for other potential cancer treatments based on this technology.