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Javed Khan M.D.
Funded: 07-01-2026
through 06-30-2028
Funding Type: Research Grant
Institution Location:
Bethesda, MD
Institution: National Cancer Institute, National Institutes of Health
affiliated with NIH Clinical Center
Rhabdomyosarcoma (RMS) is an aggressive childhood cancer that resembles developing muscle but cannot mature. The most dangerous form is driven by the PAX3::FOXO1 gene fusion, which forces uncontrolled growth. Better treatments are urgently needed because current therapy cures fewer than one in three children whose cancer returns. Dr. Khan and colleagues research targets FGFR4, a protein expressed at very high levels in RMS but not in normal tissues. A highly specific antibody against FGFR4 enables precise delivery of drugs into cancers. Dr. Khan and team will use this antibody in two ways: (1) to create an antibody-drug conjugate that delivers a drug blocking EP300, a key partner PAX3::FOXO1 uses to activate cancer genes: and (2) to build an antibody-PROTAC shuttle that brings a protein-destroying drug into FGFR4-positive cells to eliminate EP300. By disabling EP300 only in cancer cells, Dr. Khan and team will halt RMS growth while sparing healthy tissue and pave the way for safer, more effective treatments.