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.[1][2]
Recent studies have laid the foundation for inferring gene expression from cfDNA (and ctDNA), with EPIC-seq emerging as a notable advancement.[3] This method has substantially raised the bar for the noninvasive inference of expression levels of individual genes, thereby augmenting the assay's applicability in disease characterization, histological classification, and monitoring treatment efficacy.[3][4][5]
ctDNA originates directly from the tumor or from circulating tumor cells (CTCs),[6] which describes viable, intact tumor cells that shed from primary tumors and enter the bloodstream or lymphatic system. The precise mechanism of ctDNA release is unclear. The biological processes postulated to be involved in ctDNA release include apoptosis and necrosis from dying cells, or active release from viable tumor cells.[7][8][9][10][11] Studies in both human (healthy and cancer patients)[12] and xenografted mice[13] show that the size of fragmented cfDNA is predominantly 166bp long, which corresponds to the length of DNA wrapped around a nucleosome plus a linker. Fragmentation of this length might be indicative of apoptotic DNA fragmentation, suggesting that apoptosis may be the primary method of ctDNA release. The fragmentation of cfDNA is altered in the plasma of cancer patients.[14][15]
In healthy tissue, infiltrating phagocytes are responsible for clearance of apoptotic or necrotic cellular debris, which includes cfDNA.[16] ctDNA in healthy patients is only present at low levels but higher levels of ctDNA in cancer patients can be detected with increasing tumor sizes.[17] This possibly occurs due to inefficient immune cell infiltration to tumor sites, which reduces effective clearance of ctDNA from the bloodstream.[16] Comparison of mutations in ctDNA and DNA extracted from primary tumors of the same patients revealed the presence of identical cancer-relevant genetic changes.[18][19] This led to the possibility of using ctDNA for earlier cancer detection and treatment follow up.[20]
^Akca H, Demiray A, Yaren A, Bir F, Koseler A, Iwakawa R, Bagci G, Yokota J (March 2013). "Utility of serum DNA and pyrosequencing for the detection of EGFR mutations in non-small cell lung cancer". Cancer Genetics. 206 (3): 73–80. doi:10.1016/j.cancergen.2013.01.005. PMID23491080.
^Schwarzenbach H, Hoon DS, Pantel K (June 2011). "Cell-free nucleic acids as biomarkers in cancer patients". Nature Reviews. Cancer. 11 (6): 426–37. doi:10.1038/nrc3066. PMID21562580. S2CID6061607.
^Stroun M, Lyautey J, Lederrey C, Olson-Sand A, Anker P (November 2001). "About the possible origin and mechanism of circulating DNA apoptosis and active DNA release". Clinica Chimica Acta; International Journal of Clinical Chemistry. 313 (1–2): 139–42. doi:10.1016/S0009-8981(01)00665-9. PMID11694251.
^Vasioukhin V, Anker P, Maurice P, Lyautey J, Lederrey C, Stroun M (April 1994). "Point mutations of the N-ras gene in the blood plasma DNA of patients with myelodysplastic syndrome or acute myelogenous leukaemia". British Journal of Haematology. 86 (4): 774–779. doi:10.1111/j.1365-2141.1994.tb04828.x. PMID7918071. S2CID26365875.
^Vasioukhin V, Stroun M, Maurice P, Lyautey J, Lederrey C, Anker P (May 1994). K-ras point mutations in the blood plasma DNA of patients with colorectal tumors in Challenges of Modern Medicine. Vol. 5. pp. 141–150.