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Circulating tumor DNA

Circulating tumor DNA (ctDNA) is tumor-derived fragmented DNA in the bloodstream that is not associated with cells. ctDNA should not be confused with cell-free DNA (cfDNA), a broader term which describes DNA that is freely circulating in the bloodstream, but is not necessarily of tumor origin. Because ctDNA may reflect the entire tumor genome, it has gained traction for its potential clinical utility; "liquid biopsies" in the form of blood draws may be taken at various time points to monitor tumor progression throughout the treatment regimen.

Methods
Pre-analytical considerations When blood is collected in EDTA tubes and stored, the white blood cells begin to lyse and release genomic wild type DNA in to the sample in quantities typically many fold higher than the ctDNA is present in. This makes detection of mutations or other ctDNA biomarkers more difficult. The use of commercially available cell stabilisation tubes can prevent or delay the lysis of white cells thereby reducing the dilution effect of the ctDNA. Sherwood et al. demonstrated superior detection of KRAS mutations in matched samples collected in both EDTA K3 and Streck BCT tubes. Extraction of ctDNA The main appeal of ctDNA analysis is that it is extracted in a non-invasive manner through blood collection. Acquisition of cfDNA or ctDNA typically requires collection of approximately 3mL of blood into EDTA-coated tubes. The use of EDTA is important to reduce coagulation of blood. The plasma fractions of blood can be separated through a centrifugation step. ctDNA or cfDNA can be subsequently extracted from these fractions. Although serum tends to have greater levels of cfDNA, this is primarily attributed to DNA from lymphocytes. High levels of contaminating cfDNA is sub-optimal because this can decrease the sensitivity of ctDNA detection. Therefore, the majority of studies use plasma for ctDNA isolation. Plasma is then processed again by centrifugation to remove residual intact blood cells. The supernatant is used for DNA extraction, which can be performed using commercially available kits. Analysis of ctDNA The analysis of ctDNA after extraction requires the use of various amplification and sequencing methods. These methods can be separated into two main groups based on whether the goal is to interrogate all genes in an untargeted approach, or if the goal is to monitor specific genes and mutations in a targeted approach. Untargeted approaches A whole genome or whole exome sequencing approaches may be necessary to discover new mutations in tumor DNA while monitoring disease burden or tracking drug resistance. Untargeted approaches are also useful in research to observe tumor heterogeneity or to discover new drug targets. However, while untargeted methods may be necessary in certain applications, it is more expensive and has lower resolution. This makes it difficult to detect rare mutations, or in situations where low ctDNA levels are present (such as minimal residual disease). Furthermore, there can be problems distinguishing between DNA from tumor cells and DNA from normal cells using a whole genome approach. Whole genome or exome sequencing typically use high throughput DNA sequencing technologies. Limiting the sequencing to only the whole exome instead can decrease expense and increase speed, but at the cost of losing information about mutations in the non-coding regulatory regions of DNA. Size-selection of short fragments (4 to 4.3 in 105. Publicly available cancer databases were used to construct a library of probes against recurrent mutations in cancer by calculating their recurrence index. The protocol was optimized for the low DNA levels observed in ctDNA collection. Then the isolated DNA undergoes deep sequencing for increased sensitivity. This technique allows for the interrogation of hundreds of DNA regions. The ctDNA detection sensitivity of CAPP-Seq is reported to be 2.5 molecules in 1,000,000. Tagged amplicon deep sequencing Tagged amplicon deep sequencing (TAM-Seq) allows targeted sequencing of entire genes to detect mutations in ctDNA. First a general amplification step is performed using primers that span the entire gene of interest in 150-200bp sections. Then, a microfluidics system is used to attached adaptors with a unique identifier to each amplicon to further amplify the DNA in parallel singleplex reactions. This technique was shown to successfully identify mutations scattered in the TP53 tumor suppressor gene in advanced ovarian cancer patients. The sensitivity of this technique is 1 in 50. Safe-sequencing Safe-sequencing (Safe-Seq) was originally described by Bert Vogelstein and his group at Johns Hopkins University. Safe-Seq decreases the error rate of massively parallel sequencing in order to increase the sensitivity to rare mutants. It achieves this by addition of a unique identifier (UID) sequence to each DNA template. The DNA is then amplified using the added UIDs and sequenced. All DNA molecules with the same UID (a UID family) should have the same reported DNA sequence since they were amplified from one molecule. However, mutations can be introduced through amplification, or incorrect base assignments may be called in the sequencing and analysis steps. The presence of the UID allows these methodology errors to be separated from true mutations of the ctDNA. A mutation is considered a 'supermutant' if 95% of the sequenced reads are in agreement. The sensitivity of this approach is 9 in 1 million. In duplex sequencing, randomized double-stranded DNA act as unique tags and are attached to an invariant spacer. Tags are attached to both ends of a DNA fragment (α and β tags), which results in two unique templates for PCR - one strand with an α tag on the 5' end and a β tag on the 3' end and the other strand with a β tag on the 5' end and an α tag on the 3' end. These DNA fragments are then amplified with primers against the invariant sequences of the tags. The amplified DNA is sequenced and analyzed. DNA with the duplex adaptors are compared and mutations are only accepted if there is a consensus between both strands. This method takes into account both errors from sequencing and errors from early stage PCR amplification. The sensitivity of the approach to discovering mutants is 1 in 10^7. Integrated digital error suppression-enhanced CAPP-Seq Integrated digital error suppression (iDES) improves CAPP-Seq analysis of ctDNA in order to decrease error and therefore increase sensitivity of detection. == Considerations ==
Considerations
"Normal" vs tumor DNA detection One of the challenges in using ctDNA as a cancer biomarker is whether ctDNA can be distinguished with cfDNA from normal cells. cfDNA is released by non-malignant cells during normal cellular turnover, but also during procedures such as surgery, radiotherapy, or chemotherapy. It is thought that leukocytes are the primary contributors to cfDNA in serum. ==Research==
Research
ctDNA in cancer screening The clinical utility of ctDNA for the detection of primary disease is in part limited by the sensitivity of current technology to detect small tumors with low levels of ctDNA present and a priori unknown somatic mutations. ctDNA in cancer monitoring Evidence of disease by traditional imaging methods, such as CT, PET or MRI may be absent after tumor resection. Therefore, ctDNA analysis poses a potential avenue to detect minimal residual disease (MRD), and thus the possibility of tumor recurrence, in cases where bulk tumors are absent by conventional imaging methods. However, the authors acknowledge that ctDNA analysis is not without limitations; plasma samples collected post-operatively were only able to predict recurrence at 36 months in 48% of cases., colorectal cancer and melanoma. These approaches are now used in the detection of minimal residual disease using both tumor-informed and tumor-agnostic approaches. ctDNA as a prognostic biomarker The question of whether measurement of the amount or qualities of ctDNA could be used to determine outcomes in people with cancer has been a subject of study. As of 2015 this was very uncertain. Although some studies have shown a trend of higher ctDNA levels in people with high stage metastatic cancer, ctDNA burden does not always correlate with traditional cancer staging. However, some recent studies in 2025 have proven that ctDNA monitorization in pancreatic cancer can be used as an independent predictor of overall survival (OS) when evaluated together with clinical risk factors, showing a higher accuracy than conventionally used techniques (CT scan or CA19.9 monitorization in serum). Cancer research The emergence of drug-resistant tumors due to intra- and inter-tumoral heterogeneity an issue in treatment efficacy. A minor genetic clone within the tumor can expand after treatment if it carries a drug-resistant mutation. Initial biopsies can miss these clones due to low frequency or spatial separation of cells within the tumor. For example, since a biopsy only samples a small part of the tumor, clones that resides in a different location may go unnoticed. This can mislead research that focuses on studying the role of tumor heterogeneity in cancer progression and relapse. The use of ctDNA in research can alleviate these concerns because it could provide a more representative 'screenshot' of the genetic diversity of cancer at both primary and metastatic sites. For example, ctDNA has been shown to be useful in studying the clonal evolution of a patient's cancer before and after treatment regimens. Early detection of cancer is still challenging but recent progress in the analysis of the epigenetic features of cfDNA, or the fragmentation pattern unlock improve the sensitivity of liquid biopsy. Furthermore, ctDNA analysis is an emerging tool for understanding the clonal composition of metastatic tumors, detecting different mutations on a genomic scale, studying subclonal diversity that affects the prognosis of the disease as different resistant phenotypes can be found and the appearance of new mechanisms of genomic and transcriptomic resistance to treatment. Challenges for implementation Implementation of ctDNA in clinical practice is largely hindered by the lack of standardized methods for ctDNA processing and analysis. Standardization of methods for sample collection (including time of collection), downstream processing (DNA extraction and amplification), quantification and validation must be established before ctDNA analysis can become a routine clinical assay. Furthermore, creation of a panel of 'standard' tumor-associated biomarkers may be necessary given the resolution of current ctDNA sequencing and detection methods. Sequencing tumor-specific aberrations from plasma samples may also help exclude contaminating cfDNA from analysis; elevated levels of cfDNA from normal cells may be attributed to non-cancer related causes. These sequencing techniques can also determine the clonal evolution of cancer, tumor heterogeneity and drug resistance mechanisms involved in cancer. ==See also==
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