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

Scientists have combined two cancer drugs to create a treatment that can be used for children with brain tumours.
Diffuse intrinsic Pontine glioma, a rare type of brain tumor in children, can evolve to resist treatment. The disease is incurable and many children who are affected by it die within months.

A team of scientists and doctors from the Institute of Cancer Research (ICR), London has discovered that combining a skin and blood cancer drug can be very effective in fighting DIPG. Cancer experts called the breakthrough promising and it was published in Cancer Discovery.

Professor Chris Jones of paediatric brain tumor biology at the ICR said that we now have a better understanding of how DIPG brain tumours can become resistant to single drugs and the mechanisms by which they can be mutated. This has enabled us to identify what we believe could be a new, successful combination treatment for this horrible disease.

Prof Chris Jones: We are hopeful that the new treatment will soon enter clinical trials. Photograph by ICR London/PA

The new research found that the combination of two cancer drugs, trametinib and dasatinib, slowed down the growth of DIPG tumors. These treatments are called MEK inhibitors.

Targeted drugs are often effective on their own, but cancers can develop resistance to them. They had never been considered for children with DIPG, most of whom die within one year.

Researchers tested trametinib on mice and found it to have little effect. To treat DIPG cells in the laboratory, they tried trametinib and dasatinib together. The ICR stated that they found that combing these two drugs with different mechanisms of action slowed down the growth of tumours.

Combining the effects of both drugs together produced a greater effect than expected. Combining the two drugs resulted in a reduction of more than 60% in growth of DIPG cancer cells that were grown on brain tissue from mice.

Jones said that although our findings need to be confirmed in the laboratory, because we are using approved drugs we know are safe, it is not too soon before the new treatment enters clinical trials.

We are encouraged by these promising results to continue analysing patient samples and modeling their treatment response. It shows how specific some treatments were that we needed to develop.

Professor Kristian Helin is the chief executive officer of the ICR. He stated that cancer's ability to develop resistance to treatment is one the greatest challenges in developing effective targeted therapies for this disease.

We must continue to find ways to improve cancer's ability to evolve and adapt in children's cancers like DIPG, so that we can offer new treatments to young patients with cancer who desperately need them.

A variety of cancer charities funded the research, including Christophers Smile and Abbies Army, Islastones Foundation and the CRIS Cancer Foundation.