Stanford Researchers find promising drug pair for DIPG treatment

Researchers at some of the top children’s cancer research centers in America have a new approach to treating pediatric brain tumors, and it is raising hopes that a new drug treatment for DIPG could be possible.

Pediatric brain tumors are among the most dangerous cancers and diseases children get. While common childhood cancers like lymphoma have seen improved survival in recent decades, brain tumors remain difficult to treat in children. 

A young boy with his brain glowing white.

By and large the brain tumors that impact children are different from those that strike adults. The most common children’s brain tumors are known as diffuse midline gliomas (DMG), DMGs include diffuse intrinsic pontine glioma (DIPG), thalamic glioma and spinal cord glioma. DMGs are highly resistant to treatment: to date no child has survived DIPG. 

But a new pair of drugs—panobinostat and marizomib—have shown promise by working together, killing cancer cells and turning off a gene mutation that helps the cancer grow. As a strategy, this is not unusual. Very few cancers can be treated effectively by a single drug alone. But for DIPG, which so far has been studied less than many other cancers, researchers first had to conduct an exhaustive search just to find a pair of drugs that might work well together. 

A group of researchers at Stanford University, led by pediatric neuro-oncologist Michelle Monje, teamed up with researchers at the National Institutes of Health to find such a pair. With the help of robots that can run tests far faster than human lab assistants, they explored of over 9,000 drug pairs on lab-grown strains of DIPG and identified a leading candidate. 

They focused on drugs that were approved or close to approved for human patients, attacked cancers by different mechanisms, and were known to cross the blood brain barrier. And this pair emerged as the most promising. 

According to Monje, the combination is now advancing towards clinical trials for children with DIPG. 

She cautions that this will not likely be enough to cure DIPG. A multi-pronged approach will ultimately be necessary, involving immunotherapy and a better understanding of the tumor microenvironment of DIPG.

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