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Resistance to methotrexate and multidrug resistance in childhood malignancies

D Niethammer1, H Diddens, V Gekeler

  • 1Department of Pediatrics, University of Tübingen, F.R.G.

Insights

Drug resistance in pediatric cancers is a major challenge. This study identifies gene amplification for dihydrofolate reductase and MDR1 gene expression as key causes of resistance in children, offering insights for treatment strategies.

Area of Science:

  • Oncology
  • Molecular Biology
  • Pharmacology

Background:

  • Drug resistance, both primary and acquired, significantly impedes effective cancer chemotherapy.
  • Understanding the molecular mechanisms of resistance is crucial for developing improved therapeutic strategies in pediatric malignancies.

Purpose of the Study:

  • To investigate the molecular basis of methotrexate (MTX) resistance and multidrug resistance (MDR) in human pediatric cancer cell lines and in children with resistant cancers.
  • To identify specific genetic alterations and gene expression patterns associated with drug resistance in pediatric malignancies.

Main Methods:

  • Analysis of gene amplification for dihydrofolate reductase (DHFR) in cell lines and patient samples.
  • Assessment of multidrug resistance 1 (mdr1) gene expression, coding for P-glycoprotein, in relation to MDR.
  • Evaluation of the impact of drug withdrawal and reintroduction on mdr1 gene expression in cell cultures.

Main Results:

  • Gene amplification of DHFR was confirmed as a cause of MTX resistance in both pediatric cancer cell lines and children.
  • Increased expression of the mdr1 gene, encoding P-glycoprotein, was associated with MDR in cell lines and pediatric patients.
  • Reversible modulation of mdr1 gene expression was observed in response to drug exposure in cell culture, highlighting dynamic resistance mechanisms.

Conclusions:

  • This study provides the first data in children demonstrating DHFR gene amplification and mdr1 gene overexpression as causative factors for drug resistance in cancer.
  • The findings underscore the clinical relevance of these molecular mechanisms and suggest potential therapeutic avenues, such as high-dose MTX with leucovorin rescue, to overcome resistance.

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