Fighting Osteosarcoma with a Bold Idea

When Lily Guenther, M.D., was just starting her career in childhood cancer research at Dana-Farber Cancer Institute, Rally Foundation for Childhood Cancer Research gave her something big, support to chase a bold idea.

She was studying osteosarcoma, a rare and aggressive bone cancer that mostly affects kids and teens. It’s especially hard to treat when it spreads to other parts of the body. This is called metastatic cancer, and sadly, many kids already have metastatic osteosarcoma when they’re first diagnosed.

Thanks to Rally Foundation’s early support, Dr. Guenther ran a special experiment called a CRISPR screen. That’s where scientists turn off one gene at a time in cancer cells to find weak spots. She wanted to see if a drug called a PARP inhibitor could work better when combined with other drugs.

PARP inhibitors are medicines that stop cancer cells from fixing their own damaged DNA. If cancer cells can’t repair themselves, they’re more likely to die. But these drugs don’t always work well by themselves, so Dr. Guenther tested combinations to make them stronger.

Here’s what she found: when osteosarcoma cells were treated with both PARP inhibitors and something called ATM inhibitors (which also block DNA repair), the cancer cells had a much harder time surviving.

Today, Dr. Guenther leads her own lab at St. Jude Children’s Research Hospital. Her team is testing these drug combinations in the lab and working with computer experts to find the best matches. She’s even teaming up with doctors to try to bring this treatment to real kids in a clinical trial.

This all started with early funding from Rally.

This is why early-stage research funding matters.

This is the power of philanthropic seed investing.

This is Rally.