Targeted Drug Therapy for Neuroblastoma

Newer drugs that target neuroblastoma cells more specifically than standard chemo drugs are now being used in treatment.

ALK inhibitors

Some high-risk neuroblastomas have changes in the ALK gene that help them grow. For children with these tumors, drugs that target changes in the ALK gene may be helpful. These drugs include:

• Crizotinib (Xalkori)
• Lorlatinib (Lorbrena)
• Ceritinib (Zykadia)

Lorlatinib is now being studied as part of the initial treatment for some children with high-risk neuroblastoma. Other drugs that target cells with ALK changes, such as ceritinib, and combination treatments are also being studied.

Other kinase inhibitors

The presence of MYCN oncogene amplification in neuroblastomas has been found to impact how well neuroblastoma treatments work. Studies looking at adding dasatinib (Sprycel, a tyrosine kinase inhibitor) and sirolimus (rapamycin, an mTOR inhibitor) to treatment in relapsed MYCN-amplified tumors have shown better results. Research to understand the MYCN oncogene and how it works has led to new drugs to target its impact on neuroblastoma treatment response.

In some neuroblastomas, the cells have an overactive Aurora A kinase signaling pathway, which helps the cells grow. Drugs that target the Aurora A kinase pathway, such as LY3295668 erbumine, are being studied in clinical trials for relapsed or difficult-to-treat neuroblastoma.

Many other targeted drugs are now being studied for use against neuroblastoma as well.