Heart drug could help kids with brain tumours

Scientists at the University of Nottingham have discovered that repurposing a heart drug could significantly increase the survival rate for children with ependymoma — a particularly aggressive type of brain tumour.

Published in the journal Scientific Reports, the findings suggest that co-treatment with a drug normally used to treat cardiac hypertrophy can overcome chemotherapy resistance and increase survival in over a third of ependymoma patients.

Ependymomas are the second most common malignant brain tumours in children. They can occur across all age groups, but the outcome for children is lower than in their adult counterpart. The poorest survival is seen in infants, with the five-year prognosis at just 42–55%.

The use of chemotherapy in children with ependymomas has had variable levels of success, leading to the frequent belief that ependymomas are chemoresistant tumours, since over half of tumours cannot be cured by chemotherapy alone. The Nottingham researchers set out to determine the nature of this chemoresistance.

The scientists showed that, in patients treated with chemotherapy alone, the presence of a chemotherapy drug-pumping protein called ABCB1 was associated with a significantly poorer outcome. Tumours that expressed ABCB1 were less likely to respond to chemotherapy and more likely to be locally invasive.

The authors then used a heart drug to inhibit ABCB1 function in cells taken from patients’ tumours. The heart drug was able to stop ABCB1 pumping chemotherapy drugs out of the tumour cells, making them more sensitive to chemotherapy and less able to migrate.

With ABCB1 expressed in over one-third of patients’ tumours, the hope is that all such patients could potentially benefit from repurposing the heart drug in future clinical trials. As noted by study leader Dr Beth Coyle, “We are hopeful that by combining this repurposed drug with current treatments, we can give new hope for long-term survival to patients with these devastating brain tumours.”

Image credit: ©stock.adobe.com/au/Frantab

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