Dual-drug treatment offers hope to children with rare brain cancer | Medical research

Scientists have successfully combined two existing cancer drugs to create a treatment for children diagnosed with deadly brain tumors.

Diffuse intrinsic pontine glioma (DIPG), a rare and fast-growing type of brain tumor in children, can mutate and evolve to withstand treatment with a single drug. There is currently no cure and many children who have the disease die within months.

Now a team of scientists and doctors from the Institute of Cancer Research (ICR), London, has found that taking a skin cancer drug and a blood cancer drug together can be effective in fighting DIPG. The breakthrough, described as “promising” by cancer experts, was revealed in the journal Cancer Discovery.

Prof Chris Jones, professor of childhood brain tumor biology at the ICR, said: “We now have a much better understanding of the ways in which DIPG brain tumors can mutate and how they can develop resistance to treatment with a single drug. It has allowed us to to identify what we hope can become a successful new combination treatment for this terrible disease.”

Prof Chris Jones: ‘We hope it won’t be too long before the new treatment enters clinical trials’. Photo: ICR London/PA

In the new study, lab tests found that the combination of two existing cancer drugs — dasatinib for leukemia and trametinib for melanoma — slowed the growth of DIPG tumors. The treatments are known as MEK inhibitors.

These targeted drugs have often been found to work well on their own at first, only for cancers to develop resistance to treatment. Until now, they had not previously been studied for children with DIPG – most of whom currently die within a year of diagnosis.

Researchers evaluated trametinib in mice and found that it had little effect on its own. They then tried using dasatinib alongside trametinib to treat DIPG cells in the lab. They found that the combination of these two drugs, each with a different mechanism of action, “slowed down tumor growth,” according to the ICR.

The combination “had a much greater effect than would have been expected by adding the effects of the two drugs together,” it added. The combination succeeded in reducing the growth of DIPG cancer cells grown on mouse brain tissue by more than 60%.

“Our findings will need further validation in the lab, but because we’re using existing approved drugs that we know are safe, we hope it won’t be too long before the new treatment enters clinical trials,” Jones said.

“These promising results have encouraged us to continue to analyze patient samples and model their treatment response, as it shows how specific are some of the treatments we need to develop.”

Prof Kristian Helin, the chief executive of the ICR, said: “The ability of cancer to evolve to become resistant to treatment is one of the greatest challenges we face in creating effective targeted cancer therapies.

“It is vital that we continue to find ways to overcome cancer’s ability to adapt and evolve in childhood cancers such as DIPG – so that we can introduce new treatments to young cancer patients who so desperately need them.”

The research was funded by several cancer charities, including Christopher’s Smile, Abbie’s Army, Islastones Foundation, the CRIS Cancer Foundation, Cancer Research UK, Children with Cancer UK and the Ollie Young Foundation.

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