MicroRNA drugs for the treatment of neuroblastoma

MicroRNA drugs for the treatment of neuroblastoma

Recipient: Associate Professor Alexander Swarbrick
Institute: Garvan Institute of Medical Research
Funding: $308,270 July 2018 to September 2021

This project aims to develop safe and effective treatments to replace, or markedly improve, conventional chemotherapy for children with neuroblastoma.

Neuroblastoma typically affects children aged one to five, who often present with metastatic poor-prognosis disease, which has a less than 50 per cent five-year survival rate. Many patients depend on high dose multi-layered chemotherapy for their treatment. This only works for some. For many, the treatment itself impacts heavily on quality of life, with many survivors suffering chronic health problems, including cardiovascular, hearing defects and secondary cancers.

Behind the science: Associate Professor Alexander Swarbrick

MicroRNAs are small RNA molecules produced by every cell. Instead of making proteins like most genes, they work by regulating other genes, like a capacitor in an electrical circuit. The Swarbrick lab has discovered several microRNAs that very effectively kill neuroblastoma cells, either alone or in synergy with low doses of chemotherapy.

Behind the science: Dr Holly Holliday

The direct outcome from this research will be the identification of new microRNA-based drugs for neuroblastoma. If successful, a microRNA-based therapy will be ready for clinical trials in children within four years.

“Survival rates for this disease are intolerable. We are committed to changing these statistics.” - Associate Professor Alexander Swarbrick