Using a DNA molecule as a new and safer treatment for neuroblastoma

The challenge

Neuroblastoma is a type of childhood cancer that affects the nervous system. Despite aggressive treatments, only around half of children with relapsed or high‑risk disease survive. Those who do survive often face long‑term health issues caused by treatment, including learning difficulties, stunted growth and an increased risk of secondary cancers. Finding effective, kinder and less toxic treatments remains a major priority.

Adult clinical trials have shown that a molecule called aptamer AS1411 can slow cancer growth and is very well tolerated, with few side effects. AS1411 is a man‑made piece of DNA that sticks to a protein called nucleolin, which neuroblastoma cells rely on for growth. This blocks an important fuel source for the tumour. Because normal cells do not depend on nucleolin in the same way, treatments that target it could cause fewer side effects. Early laboratory tests in childhood neuroblastoma have shown similar promising results.

The project

With this funding, Professor Arturo Sala’s team at Brunel University want to fully test aptamer AS1411 in neuroblastoma. They will study its effects across multiple lab‑grown neuroblastoma cell types, to ensure the treatment could help patients with different genetic backgrounds. The team will also test AS1411 alongside existing chemotherapies to find the most effective combination for children with recurrent or high‑risk disease.

The best combination will be tested in living models of neuroblastoma to confirm whether it could be safe and effective for patients. The resulting tumours will be analysed down to individual cells to see whether AS1411 triggers changes that could make it work well alongside immunotherapies.

Throughout the project, the researchers will also be investigating how aptamer AS1411 fights cancer. This will help them understand which patients are most likely to benefit from the treatment.

The impact

The researchers believe that aptamer AS1411 is a promising option for neuroblastoma, as it has already been shown to be safe and well tolerated in adults, even at high doses. If successful, this project could allow the treatment to move into a clinical trial relatively quickly. Ultimately, it may bring us closer to safer and kinder treatment options for children facing this devastating disease.