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Molecular-Targeted Therapy for Tumor-Agnostic Mutations in Acute Myeloid Leukemia
Hironori Arai1,2, Yosuke Minami2, SungGi Chi2
1Department of Hematology and Oncology, Japanese Red Cross Narita Hospital, Iidacho 286-0041, Japan.
Abstract:
Comprehensive genomic profiling examinations (CGPs) have recently been developed, and a variety of tumor-agnostic mutations have been detected, leading to the development of new molecular-targetable therapies across solid tumors. In addition, the elucidation of hereditary tumors, such as breast and ovarian cancer, has pioneered a new age marked by the development of new treatments and lifetime management strategies required for patients with potential or presented hereditary cancers. In acute myeloid leukemia (AML), however, few tumor-agnostic or hereditary mutations have been the focus of investigation, with associated molecular-targeted therapies remaining poorly developed. We focused on representative tumor-agnostic mutations such as the TP53, KIT, KRAS, BRCA1, ATM, JAK2, NTRK3, FGFR3 and EGFR genes, referring to a CGP study conducted in Japan, and we considered the possibility of developing molecular-targeted therapies for AML with tumor-agnostic mutations. We summarized the frequency, the prognosis, the structure and the function of these mutations as well as the current treatment strategies in solid tumors, revealed the genetical relationships between solid tumors and AML and developed tumor-agnostic molecular-targeted therapies and lifetime management strategies in AML.
Insights
This study explores tumor-agnostic mutations in acute myeloid leukemia (AML), identifying potential molecular-targeted therapies. It aims to develop new treatment and lifetime management strategies for AML patients.
Area of Science:
- Genomics
- Oncology
- Molecular Biology
Background:
- Comprehensive genomic profiling (CGP) has advanced solid tumor treatment with tumor-agnostic therapies.
- Hereditary cancer research has led to novel treatments and management strategies.
- Acute myeloid leukemia (AML) lacks similar tumor-agnostic and hereditary mutation-focused therapies.
Purpose of the Study:
- To investigate the potential of developing molecular-targeted therapies for AML using tumor-agnostic mutations.
- To explore genetic links between solid tumors and AML for therapeutic insights.
- To propose lifetime management strategies for AML patients based on these mutations.
Main Methods:
- Analysis of a CGP study from Japan focusing on common tumor-agnostic genes (TP53, KIT, KRAS, BRCA1, ATM, JAK2, NTRK3, FGFR3, EGFR).
- Review of mutation frequency, prognosis, structure, and function.
- Examination of current solid tumor treatment strategies.
Main Results:
- Identified key tumor-agnostic mutations relevant to AML.
- Revealed genetic relationships between solid tumors and AML.
- Established a foundation for developing novel molecular-targeted therapies and management plans for AML.
Conclusions:
- Tumor-agnostic mutations present a promising avenue for AML treatment development.
- Translating findings from solid tumors can inform AML therapeutic strategies.
- The study paves the way for personalized medicine and improved patient outcomes in AML.
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