Modified blood-thinning drug could target neuroblastoma

In collaboration with the Press Association

A drug commonly used to prevent blood clots could be adapted to treat neuroblastoma, a form of childhood cancer, according to a US study.

“There’s still a long way to go before this could become a drug that would be given to patients" - Professor Andy Pearson, Cancer Research UK

Researchers at Duke Medicine found that a modified version of the blood-thinning drug heparin slowed the growth of tumours in mice.

Professor Andy Pearson, a Cancer Research UK expert in neuroblastoma, who was not involved in the research, said that the results showed potential for future study.

“Finding new ways to treat neuroblastoma is hugely important, and this interesting approach using a modified version of an established drug opens up a new area of exploration for potential therapies,” he said.
Many solid tumours are surrounded by a type of connective tissue – known as ‘stroma’ – that helps some cancers become more aggressive.

But according to lead author Professor Gerard C. Blobe from Duke, having a lot of stroma around the tumour is beneficial for patients with neuroblastoma. 

"Most of the time we think of stroma in solid tumours as a bad thing that helps cancers become more invasive. 

“In neuroblastoma, it's the opposite: having a lot of connective tissue around the tumour does something favourable for patients," he said.

In the study, published in the Journal of Clinical Investigation, the researchers found that the stroma surrounding neuroblastoma tumours releases a type of molecule that changed the way the cancer cells behave.

The molecules, called heparan sulfate proteoglycans (HSPGs), made neuroblastoma cells behave more like normal nerve cells, preventing them from growing.

HSPGs have a similar molecular shape to heparin, which for decades has been used as an anti-clotting agent to treat a number of blood conditions.

Testing whether heparin could mimic the effects of HSPGs, the researchers found that the drug slowed the growth of tumours in mice. But the blood-thinning effects of heparin meant that it also caused severe bleeding.

The team then identified a variant of the drug, which when tested on cells grown in the lab, was still able to halt their growth while avoiding the side-effects associated with heparin.

Professor Pearson, who works at The Institute of Cancer Research, London and Royal Marsden NHS Foundation Trust, cautioned that the US study is still a way off being translated into something that could benefit patients.

“There’s still a long way to go before this could become a drug that would be given to patients. But this early work with mice refines our understanding of how neuroblastoma cells interact with the normal cells around them, and how this communication could be exploited to slow the growth of tumour cells.”

Neuroblastoma develops from cells called neuroblasts that are left behind following a baby’s development in the womb. Around 90 children are diagnosed with the disease each year in the UK.


  • Knelson E.H, et al. (2014). Stromal heparan sulfate differentiates neuroblasts to suppress neuroblastoma growth, Journal of Clinical Investigation, DOI: