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

[摘要] Neurofibromatosis type I is a common tumor predisposing diseasein humans. Surgical therapy can be applied only in selectedpatients with resectable masses. Hence, development of newtherapies for this disease is urgent. We used human neurofibromaimplants in mice with severe combined immunodeficiency (SCID) asa model to test the toxicity and potential efficacy ofpirfenidone, a new therapeutic agent. Two hundred twelve humanneurofibromas were transplanted into various locations in 59experimental animals, and 30 mice with implants received oralpirfenidone for up to six weeks. Survival of neurofibromas inanimals treated with pirfenidone was lower than in the controlgroup(P=.02). Tumors did not change histologic appearance orvascularization in response to pirfenidone. Treatment withpirfenidone, a new antifibrotic agent, inhibits survival of sometumors without causing toxicity in animals.

[学科分类] 基础医学