Lilah's Fund researchers develop and test new treatments for neuroblastoma patients in conjunction with the Canadian Stem Cell Network, the Ontario Institute of Cancer Research, SickKids, the Scripps Institute and the Vermont Cancer Centre, February 2010

Lilah’s Fund helps fund the neuroblastoma drug discovery project at SickKids Hospital in Toronto.  Neuroblastoma is one of the “big three” kids cancers which also include leukemia and brain tumors, and is one of the most fatal. No drugs presently exist to specifically treat neuroblastoma.

This unique and innovative project uses neuroblastoma stem cells isolated from patients at SickKids In order to find new drugs and treatments. Cancer stem cells are the cells within tumors that are resistant to present treatments and are responsible for cancers reoccurring after treatment. They can be thought of as “queen bees.” In the hive, only killing the queen and not the workers will end the hive. In cancer, present treatments are thought to target the workers and not the queen, accounting for the poor cure rate of neuroblastoma in children.

With funding from Lilah’s Fund and other family-based neuroblastoma research funds (Including the James Fund, Sam’s Day, and Shania’s Sunflower of Hope), The Kaplan lab has identified the queen bee or cancer stem cells in neuroblastoma and along with colleagues at SickKids, including Drs. Sylvain Baruchel and Meredith Irwin, have been discovering drugs used for other conditions in children that can kill the cancer stem cells. One of the drug combinations identified, rapamycin and vinblastine, has been administered to patients in the US and Canada, and will be assessed in a Phase I clinical trial at SickKids and the Vermont Cancer Centre beginning in 2010. This work was awarded a prize for best research at the International Advances in Neuroblastoma Research meeting in Japan in 2008.  Kaplan lab scientists have been recognized for these studies by the American Association of Cancer Research and the Canadian Cancer Society.

Lilah’s Fund specifically supports the research of Dr. Kristen Smith and Dr. Natalie Grinshtein in the Kaplan laboratory at SickKids. Dr. Smith is spending the next year in the laboratory of Dr. Peter Schultz at the Scripps Institute in San Diego to test 3 million drugs and chemicals for their ability to kill neuroblastoma stem cells from patients. This collaborative project between Scripps Institute, the Canadian Stem Cell Network, and SickKids is the largest effort worldwide to discover new drugs to treat neuroblastoma, and the only one that targets neuroblastoma stem cells from individual patients. Dr. Grinshtein works closely with the Ontario Institute of Cancer Research to discover new drugs to treat neuroblastoma and is currently testing several encouraging “leads”.  If successful, we hope to begin additional clinical trials in the next year.

We are grateful for the support of Lilah’s Fund, which has truly made a huge impact on our ability to perform novel and groundbreaking research.

David Kaplan, Ph.D
Senior Scientist, Cell Biology Program, and
Co-head, SickKids Comprehensive Cancer Centre