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The Desmoid Tumor Research Foundation

May 2019

Feasibility of pre-operative mTOR inhibitor Sirolimus in children and young adults with desmoid tumor

Author Information

Verwys S(1), Magyar C(2), Glick K(1), Hawkins D(3), Sharma A(4), Weigel B(5), Chastain K(6), Khoury J(7), Manalang M(8), Dry S(2), Federman N(2), Weiss A(1).

(1)Maine Medical Center, Portland, ME. (2)The David Geffen School of Medicine at UCLA, Los Angeles, CA. (3)Seattle Children's Hospital, Seattle, WA. (4)Rutgers Cancer Institute of New Jersey, New Brunswick, NJ. (5)Univ of Minnesota, Masonic Cancer Center, Minneapolis, MN. (6)Mercy Hospital, Univ of Missouri-Kansas City, Kansas City, MO. (7)The Univ of Texas, MD Anderson Cancer Center, Houston, TX. (8)Marshfield Clinic, Marshfield, WI.

Publication

MaineHealth Knowledge Connection

Summary

Desmoid tumor represents an intermediate grade neoplasm with a striking predilection for locally invasive growth and recurrence following resection. More effective, well-tolerated non-surgical treatment options are needed. Current approaches: If feasible, watchful waiting is the preferred approach, 20-30% spontaneous regression. In situations where treatment is indicated, the following approaches are utilized:vSurgery is the primary approach if minimal morbidity is anticipated, Medical therapies, Cytotoxic drugs, Tyrosine kinase inhibitors, Hydroxyurea, Gamma secretase inhibitors. mTOR Inhibitor Rationale: Desmoid tumor is well-known to be associated with deregulation of the APC/beta-catenin pathway, Deregulation of the mTOR cell proliferation/survival pathway may play an important role in tumor biology when the APC/beta-catenin pathway is disrupted, The mTOR inhibitor sirolimus is attractive as a potential targeted therapy for desmoid tumor, Well-tolerated in children and young adults, Can be given orally in tablet or liquid formulation.

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