I recently spoke with Dr. Rao at the University of Texas Health Science Center at San Antonio about his 2024 research project funded by The Cure Starts Now. His work focuses on medulloblastoma, the most common cancerous brain tumor in children, and explores a new way to make these tumors more responsive to treatment.
Before diving into his findings, Dr. Rao shard something many families already know. While progress has been made, there is still a long way to go.
Why New Approaches Matter
Some children respond well to treatment. Others, especially those with more aggressive forms, face a higher risk of the tumor coming back, often within just a few years.
Even when treatment works, therapies like radiation and chemotherapy can have lasting effects on a child’s development and quality of life. For families, this creates a difficult balance between treating the tumor and protecting the child’s future.
That is why researchers are looking for approaches that are not only more effective, but also more precise.
A Closer Look Inside Tumor Cells
Dr. Rao’s research focuses on what helps tumor cells survive. At the center of this work is a protein called SKP2. Proteins act like tiny workers inside cells, helping control how they grow and function. Dr. Rao’s team found that medulloblastoma cells rely heavily on this protein.
Dr. Rao explained that SKP2 appears to play two roles. It helps tumors grow, and it makes them less responsive to radiation, one of the main treatments children receive. This makes it an important target for improving outcomes.
The Tumor’s Fuel Source
As Dr. Rao’s research progressed, the team uncovered a connection between SKP2 and how tumor cells use nutrients. They found that SKP2 makes tumors more dependent on an amino acid called glutamine.
Glutamine is something all cells use. But medulloblastoma cells seem to need much more of it than normal cells. Dr. Rao described this as a dependency.
In simple terms, glutamine acts like fuel. Tumor cells rely on a constant supply to grow and survive. If that supply can be disrupted, the tumor may become weaker.
A New Treatment Idea
This discovery points to a different way of thinking about treatment.
Instead of only trying to destroy tumor cells directly, researchers are exploring whether they can cut off what those cells need to survive. By blocking SKP2 signals or limiting how tumor cells use glutamine, it may be possible to slow tumor growth and make the cancer more sensitive to radiation.
Building on Existing Therapies
One encouraging part of this work is that researchers may not have to start from the beginning. There are no effective drugs yet that directly target SKP2, especially ones that can safely reach the brain. However, there are already drugs in development that block how cells use glutamine. Some of these drugs have been tested in adults with cancer. That means researchers already understand more about their safety.
Dr. Rao’s team tested one of these drugs in medulloblastoma models and saw promising results. The drug was able to reach the brain, slow tumor growth, and improve response to radiation.
What Researchers Are Seeing
To better understand how this approach might work in patients, Dr. Rao’s team studied tumor samples taken directly from children during surgery.
These models closely reflect what happens in the body. Across these studies, the findings suggest this approach may be especially promising in more aggressive forms of medulloblastoma, which are often the hardest to treat.
What Comes Next
I asked Dr. Rao what this could mean for families in the near future.
He explained that one of the most hopeful aspects of this research is how quickly it may move forward. Because glutamine-targeting drugs are already being tested in adults, pediatric clinical trials could begin within the next one to two years.
These trials will involve multiple hospitals working together. In pediatric cancer, collaboration is essential. No single center sees enough patients to answer these questions alone.
Dr. Rao’s team is working with institutions such as MD Anderson Cancer Center and other collaborating centers to help bring this approach into clinical testing.
We look forward to continuing to follow this work as it moves forward.
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