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Molecular targeted therapies in advanced or metastatic chordoma patients: facts and hypotheses
Loïc Lebellec1, Sébastien Aubert2, Fahed Zaïri3
1Medical Oncology Department, Centre Oscar Lambret, Lille, France.
Abstract:
Chordomas, derived from undifferentiated notochordal remnants, represent less than 4% of bone primary tumors. Despite surgery followed by radiotherapy, local and metastatic relapses are frequent. In case of locally advanced or metastatic chordomas, medical treatment is frequently discussed. While chemotherapy is ineffective, it would appear that some molecular targeted therapies, in particular imatinib, could slow down the tumor growth in case-reports, retrospective series, and phase I or II trials. Nineteen publications, between January 1990 and September 2014, have been found describing the activity of these targeted therapies. A systematic analysis of these publications shows that the best objective response with targeted therapies was stabilization in 52 to 69% of chordomas. Given the indolent course of advanced chordoma and because of the absence of randomized trial, the level of evidence to treat chordomas with molecular therapy is low (level III), whatever the drug. Furthermore, we could not draw firm conclusion on the activity of imatinib. Other putative targets have also been described. Therefore, further clinical trials are expected, especially with these targets. Nevertheless, it seems essential, in those future studies, to consider the naturally slow course of the disease.
Insights
Molecular targeted therapies may stabilize advanced chordomas, a rare bone cancer. However, evidence is low, and imatinib
Area of Science:
- Oncology
- Orthopedic Oncology
- Medical Oncology
Background:
- Chordomas are rare primary bone tumors originating from notochordal remnants.
- Despite surgery and radiotherapy, chordomas frequently relapse locally or metastatically.
- Advanced or metastatic chordomas warrant medical treatment discussions.
Purpose of the Study:
- To systematically analyze the efficacy of molecular targeted therapies for advanced or metastatic chordomas.
- To evaluate the best objective response rates and limitations of current molecular therapies.
Main Methods:
- Systematic review of 19 publications from January 1990 to September 2014.
- Analysis focused on case reports, retrospective series, and phase I/II trials of targeted therapies.
- Assessment of objective response, particularly tumor growth stabilization.
Main Results:
- Targeted therapies achieved tumor stabilization in 52% to 69% of chordoma cases.
- Chemotherapy demonstrated ineffectiveness against chordomas.
- The level of evidence for molecular therapy in chordoma is low (Level III) due to the absence of randomized trials.
Conclusions:
- Molecular targeted therapies show potential for stabilizing advanced chordomas, but evidence is limited.
- Firm conclusions on imatinib's activity could not be drawn.
- Further clinical trials are needed, considering the slow progression of chordoma.
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