Survival of human neurofibroma in immunodeficient mice and initial results of therapy with pirfenidone

Dusica Babovic-Vuksanovic, Lydia Petrovic, Bruce E. Knudsen, Timothy B. Plummer, Joseph E. Parisi, Srdan Babovic, Jeffrey L. Platt

Research output: Contribution to journalArticlepeer-review

15 Scopus citations


Neurofibromatosis type I is a common tumor predisposing disease in humans. Surgical therapy can be applied only in selected patients with resectable masses. Hence, development of new therapies for this disease is urgent. We used human neurofibroma implants in mice with severe combined immunodeficiency (SCID) as a model to test the toxicity and potential efficacy of pirfenidone, a new therapeutic agent. Two hundred twelve human neurofibromas were transplanted into various locations in 59 experimental animals, and 30 mice with implants received oral pirfenidone for up to six weeks. Survival of neurofibromas in animals treated with pirfenidone was lower than in the control group (P = .02). Tumors did not change histologic appearance or vascularization in response to pirfenidone. Treatment with pirfenidone, a new antifibrotic agent, inhibits survival of some tumors without causing toxicity in animals.

Original languageEnglish (US)
Pages (from-to)79-85
Number of pages7
JournalJournal of Biomedicine and Biotechnology
Issue number2
StatePublished - Jun 2004

ASJC Scopus subject areas

  • Biotechnology
  • Molecular Medicine
  • Molecular Biology
  • Genetics
  • Health, Toxicology and Mutagenesis


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