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Molecular mechanism of antifolate transport-deficiency in a methotrexate-resistant MOLT-3 human leukemia cell line

M Gong1, J Yess, T Connolly

  • 1Medicine and Pediatric Branches, National Cancer Institute, Bethesda, MD 20892,USA.

Blood
|April 1, 1997
PubMed

Insights

Methotrexate resistance in leukemia cells is linked to mutations in the reduced folate carrier 1 (RFC1) gene, affecting methotrexate transport. These RFC1 gene mutations inactivate the transporter, causing drug resistance.

Area of Science:

  • Molecular Biology
  • Cancer Research
  • Genetics

Background:

  • Methotrexate (MTX) resistance in cancer cells can arise from altered drug uptake.
  • Previous studies indicated changes in MTX transport kinetics in resistant cell lines.
  • The reduced folate carrier 1 (RFC1) gene is crucial for MTX transport.

Purpose of the Study:

  • To investigate if alterations in RFC1 gene transcription or translation cause MTX transport deficiency.
  • To determine the role of RFC1 gene mutations in MTX resistance in human T-cell leukemia cell lines.

Main Methods:

  • Studied methotrexate-resistant MOLT-3 cell lines (MOLT-3/MTX200 and MOLT-3/MTX10,000).
  • Analyzed RFC1 gene transcription and protein expression using RNA and FACS analysis.
  • Determined RFC1 gene nucleotide and genomic sequences to identify mutations.
  • Reconstituted RFC activity via retroviral vector transduction.

Main Results:

  • RFC1 RNA levels were unchanged in resistant cell lines.
  • No detectable RFC1 protein was found in highly resistant MOLT-3/MTX10,000 cells.
  • MOLT-3/MTX10,000 cells possessed three RFC1 alleles: wild-type, premature stop codon at codon 40, and premature stop codon at codon 25.
  • Only one-third of RFC1 cDNA subclones from MOLT-3/MTX10,000 cells contained the wild-type sequence.
  • MOLT-3/MTX200 cells were heterozygous for a codon 40 mutation and homozygous for wild-type at codon 25.
  • Reconstitution of RFC activity reversed MTX resistance.

Conclusions:

  • Acquisition of MTX transport deficiency in MOLT-3/MTX10,000 cells results from inactivating mutations in RFC1 gene alleles.
  • Mutations leading to premature stop codons in RFC1 are responsible for MTX resistance.
  • RFC1 gene mutations play a critical role in methotrexate resistance mechanisms.

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