Small Molecules To Treat Pediatric Cancers

Targeted therapies for high-risk leukemia in children have yet to be developed, but scientists at Université de Montréal and its affiliated Institute for Research in Immunology and Cancer are working on large-scale drug discovery project to get there.

In the laboratory run by Brian Wilhelm, UdeM doctoral student in cancer genomics Safia Safa-Tahar-Henni has tested more than 11,000 molecules for their ability to inhibit the survival and growth of human leukemia cells.

Her results were published last October in a study in the journal Leukemia.

Cells were sourced from samples from patients with leukemia, laboratory-generated models of human leukemia and established human leukemia cell lines - and striking differences were observed between how the latter responded versus the others.

"We found that using leukemic cell lines can lead to misleading results in drug discovery," said Wilhelm. "These cell lines do not grow in the same way as cells taken from patients."

The efficacy of drug-discovery experiments therefore greatly depends on the source of the cells used in the assays, he said.

In all, the scientists found 12 molecules with anti-leukemic potential.

"The molecules we have identified are not only capable of killing various leukemia cells, but also multiple cells of myeloma, another type of deadly blood cancer," said Wilhelm.

"What's more, these molecules were selected specifically because they eliminate cancer cells without affecting normal cells. This nuance is crucial for the development of targeted treatments that cause fewer side effects, which are terrible with the standard chemotherapy currently available."

The IRIC scientists are now developing dozens of new versions of these selected molecules for further study, and hope to someday make significant advances in the identification of more effective anti-cancer treatments.

About this study

"Comparative small molecule screening of primary human acute leukemias, engineered human leukemia and leukemia cell lines," by Safa-Tahar-Henni et al, was published Oct. 29, 2024 in Leukemia.

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