Research And Grants

A Novel Formulation to Allow the Simultaneous Targeting of MYC Expression and Copper Chelation to Treat DIPG Tumor and Overcome Multidrug Resistance

Diffuse Intrinsic Pontine Glioma (DIPG) is a devastating brain cancer that primarily affects children between the ages of 5 and 10. With a poor prognosis, most children diagnosed with DIPG survive only about 9 to 12 months. Current treatments like radiation therapy offer only temporary relief, leaving families and doctors in desperate need of better options.

Our Innovative Approach

Our research is exploring a novel approach to treat DIPG by combining two promising strategies: copper chelation and gene silencing.

1. Copper Chelation: Cancer cells, especially in brain tumors, often contain high levels of copper, which they utilize to grow and resist treatments. We use a drug called TETA to strip the excess copper from the cancer cells, making them weaker and more susceptible to treatment.
2. Gene Silencing: The MYC gene is a major player in tumor growth and aggressiveness. By using small molecules called siRNA, we aim to turn off this gene, thereby slowing down or stopping the tumor growth.

The Delivery System

To get these treatments directly to the tumor cells, we use structures made from red blood cells, known as extracellular vesicles (EVs). These EVs act like delivery trucks, carrying our treatments safely through the body to the brain tumor, ensuring they reach their target effectively.

What We Expect

Combining these two approaches, we hope to:

• More effectively stop tumor growth.
• Increase the death of cancer cells.
• Reduce the tumor cells’ ability to survive.
• Prevent the cancer cells from developing resistance to treatment.

Progress So Far

We have made exciting progress from the start of our study in March 2024.

1. Producing EVs: We successfully created EVs loaded with anti-MYC siRNA.
2. Delivery to Cancer Cells: These EVs effectively delivered the siRNA to DIPG cells in the lab, significantly reducing MYC gene activity.
3. Reducing Tumor Growth: This led to a 50% reduction in cancer cell growth. Combining TETA with MYC silencing showed an even greater effect, making the treatment more powerful.
4. Developing Tools: We designed a high-throughput device to improve the efficiency of loading red blood cells with our therapeutic molecules, aiming to increase production and reduce costs. This plug-and-play device is capable of collecting blood, loading red blood cells with therapeutics, and releasing extracellular vesicles (EVs) into the patients' veins, embodying a circular personalized medicine approach

The Potential Impact

If successful, our approach could revolutionize DIPG treatment, offering a new, targeted therapy that minimizes damage to healthy cells and reduces side effects. This method could also be adapted to treat other types of cancer by changing the drug and RNA content inside the EVs.

Our vision is to provide more effective and personalized cancer treatments, offering new hope to patients who currently have few options. This research holds the promise of improving outcomes for children with DIPG.