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Precision Therapy for Brain Tumors in Hereditary Syndromes
Gerald C Wallace1, Madeleine Tjoelker2, Kaitlyn Bartley3
1Neurology Residency Program, Medical College of Georgia, 1120 15th Street, Augusta, GA, 30912, USA.
Opinion Statement:
Nervous system tumors arising in the setting of monogenic, hereditary cancer predisposition syndromes are unique in that the initiating genetic event in tumor formation is known. This knowledge provides a powerful treatment approach if the alteration or pathway can be targeted with a therapeutic agent. A reasonable argument can be made for the use of targeted agents in these tumor patients, even though many of them have FDA approval only for other tumor types. It is our practice to use and employ targeted therapy when standard treatments have failed or represent an unattractive option. Over time, however, targeted therapies will likely become first-line options.
Insights
Targeted therapies offer a promising treatment for nervous system tumors in hereditary cancer syndromes by targeting known genetic alterations. These agents are increasingly used when standard treatments fail, potentially becoming first-line options.
Area of Science:
- Oncology
- Genetics
- Neurology
Background:
- Monogenic hereditary cancer predisposition syndromes involve known initiating genetic events in tumor formation.
- Nervous system tumors in these syndromes present a unique opportunity for targeted treatment strategies.
Purpose of the Study:
- To evaluate the utility of targeted therapeutic agents for nervous system tumors in hereditary cancer predisposition syndromes.
- To discuss the current practice and future potential of targeted therapies in this patient population.
Main Methods:
- Review of current clinical practices and literature regarding targeted therapy use.
- Analysis of the rationale for employing targeted agents despite potential off-label indications.
Main Results:
- Targeted agents can be effectively utilized in patients with known genetic alterations driving tumor formation.
- Current practice involves using targeted therapy when standard treatments are inadequate or undesirable.
Conclusions:
- Targeted therapies represent a powerful treatment modality for nervous system tumors in hereditary cancer syndromes.
- The use of targeted agents is likely to expand, potentially becoming a first-line treatment option in the future.
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