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Donors Fuel Promising Cancer Research

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Mark R. Kelley, Ph.D.,

Gifts from Riley Children’s Foundation donors have led to a significant grant in support of promising Riley cancer research.

Wells Center for Pediatric Research cancer investigator Mark R. Kelley, Ph.D., and his team have been awarded a $2.8 million dollar grant from the National Cancer Institute (NCI). This funding will allow them to advance studies using the drug APX3330, which appears to be able to attack certain cancers while preventing a common chemotherapy side effect, peripheral neuropathy. The goal of this research is to see if this drug can give cancer patients better long-term outcomes with fewer chemotherapy-induced nerve damage problems such as pain, tingling and muscle weakness.

“What we’re seeing is we can target tumors and slow their growth and at the same time protect the neurons,” explains Dr. Kelley. Early studies have shown the drug to be effective in attacking cancer without causing the nerve damage that is common with platinum-based chemotherapy drugs. As Dr. Kelley explains, APX3330 takes a “sniper shot” at a protein known as Ape 1, which is responsible for “turning on” multiple transcription factors that allow cancer to grow. The drug causes a domino effect. By shutting down Ape 1 it stops the rest of the process that leads to cancer growth, while at the same time protecting neurons from damage. “The mice don’t get the neuropathy, and we see less DNA damage in the neurons,” says Dr. Kelley. 

The drug gained FDA approval last summer and Phase I trials will begin soon in patients. It must be tested in adults first, but has great future potential in pediatric cancers such as osteosarcoma, neuroblastoma and Malignant Peripheral Nerve Sheath Tumors (MPNST).

Pediatric Research and Patient Care is the number one fundraising priority for Riley Children’s Foundation, which facilitated the initial donations made from the Tom and Julie Wood Family Foundation and Tom Wood Lexus. The $42,000 philanthropic investment provided both bridge funding and seed funding to get this research to the next level, and ultimately led to the $2.8 million dollar grant—a 67-fold return on investment. “Riley’s mission is exemplified by Dr. Mark Kelley,” says Bart Schlosser with Tom Wood Lexus. “His passion toward pediatric oncology research is beyond compelling, especially when the research already done has save so many children’s lives. A remedy is so close, and Mark’s National Cancer Institute grant puts it ever so closer.”

“In many ways it’s the perfect storm,” says Dr. Kelley. “The National Cancer Institute is very interested in symptom management and quality of life. We have a drug that is being shown to attack hard-to-treat cancers with fewer toxic side effects. And we had the donor funding we needed to get our studies off the ground so they could gain federal support. It’s a great example of the progress that we can make in advancing medicine when donors in the community get behind us.”

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