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Research finds potential drug targets for rare pediatric cancer

Investigators at the University of Calgary‘s Cumming School of Medicine (ALTA, Canada) have made progress in understanding neurocutaneous melanocytosis (NCM), an extremely rare childhood cancer. The study, recently published in Neuro-Oncology, uncovered potential drug targets for the disease by using a molecular analysis of patient tumor cells grown in animal models.

NCM is a malignant cancer characterized by an excessive growth of melanin-producing cells in both the skin and the brain. Approximately 1% of the population is born with congenital melanocytic nevi, a precursor to NCM. In Canada, only one or two patients are diagnosed with NCM per year. Currently, the symptomatic form of this disease carries an extremely poor prognosis with little or no benefit offered by current chemotherapy and radiation regimens.

The study aimed to describe target drugs and potential new treatments for children affected by NCM. Lead author Aru Narendran from Alberta Children’s Hospital explained that the new knowledge gained from the study will be a catalyst to generate further information about this rare cancer.

“While there is unfortunately no known effective treatment for NCM, this study is an important step in the search for new and effective agents,” added co-lead author Ronald Anderson from Alberta Children’s Hospital.

The experimental approach taken by the team was aimed towards gaining maximal biological and drug sensitivity information in very rare tumors where sufficient quantities of specimens are not available.

“This is important because we have shown that real efforts can be made to understand the biology of even extremely rare cancers that have not been studied adequately by researchers in the past,” commented Narendran. “In a wider sense, it sets up precedence to search for effective treatments for conditions that are extraordinarily uncommon in children.”

Sources: Ruan Y, Kovalchuk A, Jayanthan A et al. Druggable targets in pediatric neurocutaneous melanocytosis: Molecular and drug sensitivity studies in xenograft and ex vivo tumor cell culture to identify agents for therapy. Neuro-Oncology, doi: 10.1093/neuonc/nou310 (Epub ahead of print) (2015); University of Calgary press release