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Journal of Biomedicine and Biotechnology
Volume 2004, Issue 2, Pages 79-85
http://dx.doi.org/10.1155/S1110724304308107
Research article

Survival of Human Neurofibroma in Immunodeficient Mice and Initial Results of Therapy With Pirfenidone

1Department of Medical Genetics, Mayo Clinic, 200 First Street SW, Rochester, MN 55902, USA
2Department of Surgery, Mayo Clinic, 200 First Street SW, Rochester, MN 55902, USA
3Transplantation Biology Program, Mayo Clinic, 200 First Street SW, Rochester, MN 55902, USA
4Department of Laboratory Medicine and Pathology, Mayo Clinic, 200 First Street SW, Rochester, MN 55902, USA

Received 27 September 2003; Revised 16 December 2003; Accepted 22 December 2003

Copyright © 2004 Hindawi Publishing Corporation. This is an open access article distributed under the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.

Abstract

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.