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Targeting DNA Damage Repair Pathways Beyond PARP Inhibition
1Cancer Research UK Scotland Centre, Institute of Genetics and Cancer, University of Edinburgh, Crewe Road South, Edinburgh, EH4 2XU, UK.
Abstract:
Exploiting DNA damage repair (DDR) vulnerabilities has become a major focus in cancer drug development following the clinical success of poly (ADP-ribose) polymerase (PARP) inhibitors. Beyond PARP, inhibitors of multiple other DDR proteins have progressed from preclinical development to early-phase clinical trials. DNA damage repair inhibitors have shown promise both as a selective therapeutic strategy in genomically selected cancers harbouring specific genetic vulnerabilities, and as a potential treatment approach to overcome innate and acquired PARP inhibitor resistance. This review summarises the most recent DDR inhibitor clinical evidence, focusing on DDR signalling targets; ATR (ataxia telangiectasia and Rad3-related protein serine/threonine kinase), ATM (ataxia telangiectasia mutated kinase), DNA-PK (DNA-dependent protein kinase), CHK1 (checkpoint kinase 1), WEE1 and recently emerging DNA repair targets; RAD51, PolƟ (DNA polymerase theta), WRN (Werner syndrome helicase), USP1 (ubiquitin specific peptidase 1) and PARG (poly(ADP-ribose) glycohydrolase). We highlight both clinical successes and failures of DDR inhibitors as monotherapy or in combination with chemotherapy, PARP and other DDR inhibitors. The challenges that must be addressed to see the true potential of these agents are discussed. Finally, we consider future directions and the necessity for integration of biomarkers and genomic profiling in DDR inhibitor clinical trials to optimally identify patients with tumours genetically vulnerable, and so crucially, take advantage of the selective therapeutic opportunity provided by DDR inhibition.
Insights
DNA damage repair (DDR) inhibitors show promise in cancer therapy, targeting specific vulnerabilities. Further research and biomarker integration are crucial for optimizing their clinical use and overcoming resistance.
Area of Science:
- Oncology
- Molecular Biology
- Genetics
Background:
- The clinical success of poly (ADP-ribose) polymerase (PARP) inhibitors has spurred interest in targeting DNA damage repair (DDR) pathways for cancer treatment.
- Inhibitors of various DDR proteins are advancing into clinical trials, offering potential for targeted therapy in genomically defined cancers and overcoming PARP inhibitor resistance.
Purpose of the Study:
- To review recent clinical evidence of DNA damage repair inhibitors.
- To focus on specific DDR signaling and repair targets including ATR, ATM, DNA-PK, CHK1, WEE1, RAD51, PolƟ, WRN, USP1, and PARG.
- To discuss clinical successes, failures, challenges, and future directions for DDR inhibitors.
Main Methods:
- Literature review of clinical trial data for DDR inhibitors.
- Analysis of monotherapy and combination treatments with chemotherapy, PARP inhibitors, and other DDR inhibitors.
- Discussion of challenges and future research directions, including biomarker integration and genomic profiling.
Main Results:
- DDR inhibitors have demonstrated both successes and failures in clinical trials, as monotherapy or in combination regimens.
- The review highlights the diverse range of DDR targets being investigated, from signaling kinases to DNA repair enzymes.
- Challenges remain in fully realizing the therapeutic potential of these agents.
Conclusions:
- DNA damage repair inhibitors represent a promising therapeutic strategy in oncology, particularly when tailored to specific genetic vulnerabilities.
- Overcoming challenges through biomarker integration and genomic profiling is essential for optimizing patient selection and treatment outcomes.
- Continued research into novel DDR targets and combination strategies is warranted to fully exploit the potential of DDR inhibition in cancer therapy.
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