Stem cells screen drugs for kids’ rare tumor

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A team at Johns Hopkins University in Baltimore has transformed stem cells into a particularly nasty form of pediatric brain cancer, medulloblastoma. They then used those cells to figure out what drugs might defeat the tumor and found one existing drug, approved for advanced breast cancer, that seemed to be a good candidate.

While about two-thirds of medulloblastoma patients do well with standard therapy, those in a class called “group 3” often do not survive. But the rarity of that condition, meant the researcher could not use what has become a common route to determining effective drugs: comparing the genetic profile of the cancer with the genetic profile of banks of cancer cells that have already have been tested against existing cancer drugs. There are not enough Group 3 samples in the banks to take that route.

So, the Hopkins team used a two-step process for the drug search. They first inserted genes associated with the Group 3 cancers into stem cells and let the cells begin to transform into tumors. After making sure their stem cell tumors genetically looked and behaved like medulloblastoma the researchers compared genetic “signatures” from those cells with the signatures of cells in the large databases of other cancers.

We wanted to find whether the cells we created matched any of these existing signatures, because if they did, then we would have some idea of what kinds of drugs are more or most likely to kill these cells. We didn’t have to do the laborious screening to test 100,000 compounds against our own cells.”
- Eric Raabe

Raabe suggested this system might work to create a short cut to finding best therapies for other rare tumors as well.

Published by CIRM – The Stem Cellar