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Molecular Tumor Board for Unicorns: Outcomes for rare and ultra-rare cancers using an N-of-One personalized treatment
Bryan H Louie1, Shumei Kato1, Jordan S Lim1
1Center for Personalized Cancer Therapy and Division of Hematology and Oncology, Department of Medicine, UC San Diego Moores Cancer Center, La Jolla, CA, USA.
Abstract:
Treatment of rare/ultra-rare tumors is an unmet need due to a lack of standardized therapies and clinical trials. We developed the Molecular Tumor Board (MTB), a multidisciplinary team that integrates molecular profiling to generate personalized, N-of-One treatments for advanced cancers. This study evaluates 112 patients with rare/ultra-rare tumors who presented to the MTB and were evaluable for clinical therapeutic outcome. Overall, 46/112 patients (41%) received a treatment regimen with a high degree of matching between tumor molecular alterations and drugs given (reflected by a high Matching Score (≥50%)). Patients with a high versus low Matching Score experienced significantly longer progression-free survival (p = 0.005) and overall survival (p = 0.047), and higher rates of clinical benefit (stable disease ≥6 months, partial response, or complete response) (54% vs. 32% p = 0.027). The MTB facilitated personalized N-of-One matching of drugs to tumor molecular alterations, which was associated with improved clinical outcomes in patients with rare/ultra-rare cancers.
Insights
Personalized N-of-One treatments guided by molecular profiling improve outcomes for rare and ultra-rare cancers. A Molecular Tumor Board approach demonstrated longer survival and clinical benefit in patients with advanced disease.
Area of Science:
- Oncology
- Genomics
- Precision Medicine
Background:
- Rare and ultra-rare tumors present significant treatment challenges due to limited standardized therapies and clinical trials.
- Personalized medicine approaches are crucial for addressing the unmet needs in treating these complex cancers.
Purpose of the Study:
- To evaluate the effectiveness of a Molecular Tumor Board (MTB) in generating personalized, N-of-One treatment strategies for patients with rare/ultra-rare advanced cancers.
- To assess the association between molecularly matched therapies and clinical outcomes in this patient population.
Main Methods:
- A multidisciplinary Molecular Tumor Board (MTB) integrated molecular profiling data to guide treatment decisions.
- 112 patients with rare/ultra-rare tumors were evaluated for clinical therapeutic outcomes based on MTB recommendations.
- A high Matching Score (≥50%) indicated a strong correlation between tumor molecular alterations and prescribed drugs.
Main Results:
- 41% of patients received a treatment regimen with a high Matching Score.
- Patients with a high Matching Score demonstrated significantly longer progression-free survival (p=0.005) and overall survival (p=0.047).
- Higher rates of clinical benefit (stable disease ≥6 months, partial response, or complete response) were observed in the high Matching Score group (54% vs. 32%, p=0.027).
Conclusions:
- The Molecular Tumor Board effectively facilitated personalized N-of-One drug matching based on tumor molecular alterations.
- This precision oncology approach is associated with improved clinical outcomes, including survival and response rates, in patients with rare/ultra-rare cancers.
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