Scientists Uncover How Retinoic Acid ‘Treats’ Neuroblastoma

Scientists at St. Jude Children’s Research Hospital have solved a decades-old mystery surrounding the drug retinoic acid’s effectiveness in treating neuroblastoma, a deadly childhood cancer. 

While retinoic acid has long been known to improve survival rates for high-risk neuroblastoma patients after chemotherapy, its effectiveness against primary tumors remained unclear. 

In a new study published in Nature Communications, the researchers revealed that retinoic acid works by “hijacking” a normal developmental pathway to trigger cancer cell death in metastasized neuroblastoma.

The team discovered that retinoic acid’s impact heavily depends on the tumor’s cellular microenvironment—specifically the bone marrow, where neuroblastoma cells often spread. 

In this microenvironment, the bone morphogenetic protein (BMP) signaling pathway is highly active, making cancer cells more vulnerable to retinoic acid. “Cells expressing genes from the BMP signaling pathway were very sensitive to retinoic acid,” said co-first author Min Pan, PhD. 

This insight helps explain why retinoic acid is effective during post-chemotherapy consolidation but not against primary tumors.

Using gene editing technology, the researchers identified how the BMP signaling pathway collaborates with retinoic acid to promote cancer cell death. 

This discovery offers new therapeutic possibilities, as it highlights a “hijacking” of normal developmental processes that could be exploited in other cancers. 

“We are the first to uncover such an example of “hijacking” a normal embryonic developmental process preserved in cancer that we can exploit therapeutically,” said senior author Paul Geeleher, PhD. This work opens the door to new, more effective and less toxic cancer treatments.

Note: This news summary was generated by AI based on a published press release, followed by a review from human editors.