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Brain Malignancy Steering Committee clinical trials planning workshop: report from the Targeted Therapies Working
Brian M Alexander1, Evanthia Galanis1, W K Alfred Yung1
1Dana-Farber/Brigham and Women's Cancer Center, Boston, Massachusetts (B.M.A., L.T., K.L.L., P.Y.W.); Mayo Clinic, Rochester, Minnesota (E.G., K.V.B., B.P.O., J.N.S.); The University of Texas M.D. Anderson Cancer Center, Houston, Texas (W.K.A.Y., J.F.D., D.A.B.); St. Jude Children's Research Hospital, Memphis, Tennessee (J.M.B.); University of California, Los Angeles, California (T.F.C.); Memorial Sloan-Kettering Cancer Center, New York, New York (J.T.H., I.K.M.); National Cancer Institute, Bethesda, Maryland (B.M., A.T.-A.); Princess Margaret Cancer Centre, Toronto, Ontario, Canada (W.M.); Henry Ford Hospital, Detroit, Michigan (T.M.); University of California, San Diego, La Jolla, California (P.S.M.); University of California, San Francisco, California (M.D.P.); Johns Hopkins University, Baltimore, Maryland (X.Y.).
Abstract:
Glioblastoma is the most common primary brain malignancy and is associated with poor prognosis despite aggressive local and systemic therapy, which is related to a paucity of viable treatment options in both the newly diagnosed and recurrent settings. Even so, the rapidly increasing number of targeted therapies being evaluated in oncology clinical trials offers hope for the future. Given the broad range of possibilities for future trials, the Brain Malignancy Steering Committee convened a clinical trials planning meeting that was held at the Udvar-Hazy Center in Chantilly, Virginia, on September 19 and 20, 2013. This manuscript reports the deliberations leading up to the event from the Targeted Therapies Working Group and the results of the meeting.
Insights
Glioblastoma treatment options remain limited, prompting a 2013 meeting to discuss future clinical trials. This report details the targeted therapies working group
Area of Science:
- Neuro-oncology
- Clinical trial design
- Malignant brain tumors
Background:
- Glioblastoma is the most common primary brain malignancy with a poor prognosis.
- Current treatments offer limited options for both newly diagnosed and recurrent disease.
- The development of targeted therapies presents a promising avenue for improving patient outcomes.
Purpose of the Study:
- To report on the deliberations of the Targeted Therapies Working Group.
- To summarize the results of a clinical trials planning meeting held in 2013.
- To outline future directions for glioblastoma clinical trials.
Main Methods:
- Review of existing treatment strategies for glioblastoma.
- Discussion and planning for future oncology clinical trials.
- Analysis of targeted therapies relevant to brain malignancies.
Main Results:
- Identification of key challenges in glioblastoma treatment.
- Recommendations for the design of future clinical trials.
- Emphasis on the potential of targeted therapies.
Conclusions:
- Despite aggressive therapies, glioblastoma prognosis remains poor.
- Targeted therapies offer hope for improved treatment efficacy.
- Strategic planning of clinical trials is crucial for advancing glioblastoma care.

