A Glimmer of Hope for a Difficult Disease
Childhood brain tumors, especially a type known as diffuse midline glioma or diffuse intrinsic pontine glioma, have long been a challenging issue for doctors.
These tumors have a low survival rate, with most patients living only between 11-15 months after diagnosis.
They grow in crucial areas of the brain, making them particularly hard to treat. Up until now, over 250 clinical trials had tried and failed to find a solution.
However, a new compound, ONC201, is offering a ray of hope. In recent trials, this compound almost doubled the survival rate for affected patients.
From an Unexpected Source
Initially, ONC201 wasn’t even aimed at treating these tumors. It was designed for dopamine receptors, common in many types of tumors.
But then, researchers noticed something: ONC201 can penetrate the blood-brain barrier, a major hurdle when making drugs for brain diseases.
In early trials on a different type of brain tumor, glioblastoma, ONC201 didn’t show great results.
But for some patients with a certain mutation in their DMG tumor, the drug had better outcomes. This prompted a more focused trial on young patients with this mutation.
Why Does ONC201 Work?
A crucial question was, “Why did ONC201 work for these specific patients?” To find out, the team, including Dr. Koschmann and Dr. Venneti from the University of Michigan, studied the fluid surrounding the brain of patients.
The results were surprising. ONC201 was entering tumor cells and affecting their energy centers, the mitochondria. This change led to an increase in a specific chemical, L-2HG.
The more L-2HG increased, the less the tumor cells divided. Essentially, ONC201 was switching off a harmful change in the tumor cells caused by their mutation.
Looking Forward
It’s early days, but the results are promising. More trials are in progress, combining ONC201 with other treatments. The hope is to improve its effectiveness and counter any resistance the tumor might develop against ONC201.
For families with loved ones affected by this aggressive tumor, this drug provides some hope.
Dr. Koschmann remarked on the significance, stating that while the improvement is a good start, the goal is to make even bigger advancements in the future.
Dr. Venneti shared his optimism, saying, “We are really excited about this study and envision ONC201 becoming the standard of care for these patients in the near future.”
If you care about brain health, please read studies about low choline intake linked to higher dementia risk, and how eating nuts can affect your cognitive ability.
For more information about brain health, please see recent studies that blueberry supplements may prevent cognitive decline, and results showing higher magnesium intake could help benefit brain health.
The study was published in Cancer Discovery.
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