• Genomic Classification and Clinical Outcome in Rhabdomyosarcoma: A Report From an International Consortium. 

  Shern, JF; Selfe, J; Izquierdo, E; Patidar, R; Chou, H-C; Song, YK; Yohe, ME; Sindiri, S; Wei, J; Wen, X; Rudzinski, ER; Barkauskas, DA; Lo, T; Hall, D; Linardic, CM; Hughes, D; Jamal, S; Jenney, M; Chisholm, J; Brown, R; Jones, K; Hicks, B; Angelini, P; George, S; Chesler, L; Hubank, M; Kelsey, A; Gatz, SA; Skapek, SX; Hawkins, DS; Shipley, JM; Khan, J

  <h4>Purpose</h4>Rhabdomyosarcoma is the most common soft tissue sarcoma of childhood. Despite aggressive therapy, the 5-year survival rate for patients with metastatic or recurrent disease remains poor, and beyond ...
• Elucidating Prostate Cancer Behaviour During Treatment via Low-pass Whole-genome Sequencing of Circulating Tumour DNA. 

  Sumanasuriya, S; Seed, G; Parr, H; Christova, R; Pope, L; Bertan, C; Bianchini, D; Rescigno, P; Figueiredo, I; Goodall, J; Fowler, G; Flohr, P; Mehra, N; Neeb, A; Rekowski, J; Eisenberger, M; Sartor, O; Oudard, S; Geffriaud-Ricouard, C; Ozatilgan, A; Chadjaa, M; Macé, S; Lord, C; Baxter, J; Pettitt, S; Lambros, M; Sharp, A; Mateo, J; Carreira, S; Yuan, W; de Bono, JS

  <h4>Background</h4>Better blood tests to elucidate the behaviour of metastatic castration-resistant prostate cancer (mCRPC) are urgently needed to drive therapeutic decisions. Plasma cell-free DNA (cfDNA) comprises normal ...
• Frequency and Prognostic Impact of <i>ALK</i> Amplifications and Mutations in the European Neuroblastoma Study Group (SIOPEN) High-Risk Neuroblastoma Trial (HR-NBL1). 

  Bellini, A; Pötschger, U; Bernard, V; Lapouble, E; Baulande, S; Ambros, PF; Auger, N; Beiske, K; Bernkopf, M; Betts, DR; Bhalshankar, J; Bown, N; de Preter, K; Clément, N; Combaret, V; Font de Mora, J; George, SL; Jiménez, I; Jeison, M; Marques, B; Martinsson, T; Mazzocco, K; Morini, M; Mühlethaler-Mottet, A; Noguera, R; Pierron, G; Rossing, M; Taschner-Mandl, S; Van Roy, N; Vicha, A; Chesler, L; Balwierz, W; Castel, V; Elliott, M; Kogner, P; Laureys, G; Luksch, R; Malis, J; Popovic-Beck, M; Ash, S; Delattre, O; Valteau-Couanet, D; Tweddle, DA; Ladenstein, R; Schleiermacher, G (2021-06-11)

  <h4>Purpose</h4>In neuroblastoma (NB), the ALK receptor tyrosine kinase can be constitutively activated through activating point mutations or genomic amplification. We studied <i>ALK</i> genetic alterations in high-risk ...
• Investigating the phosphinic acid tripeptide mimetic DG013A as a tool compound inhibitor of the M1-aminopeptidase ERAP1. 

  Wilding, B; Pasqua, AE; E A Chessum, N; Pierrat, OA; Hahner, T; Tomlin, K; Shehu, E; Burke, R; Richards, GM; Whitton, B; Arwert, EN; Thapaliya, A; Salimraj, R; van Montfort, R; Skawinska, A; Hayes, A; Raynaud, F; Chopra, R; Jones, K; Newton, G; Cheeseman, MD

  ERAP1 is a zinc-dependent M1-aminopeptidase that trims lipophilic amino acids from the N-terminus of peptides. Owing to its importance in the processing of antigens and regulation of the adaptive immune response, dysregulation ...
• Precision genomics for prostate cancer patient stratification 

  Seed, G (2020-10-31)

  Introduction Several unmet needs currently persist in advanced prostate cancers. Despite recent discoveries, such as identification of deficient DNA repair pathways as a key driver of prostate tumour development, relatively ...
• Biological Role of MYCN in Medulloblastoma: Novel Therapeutic Opportunities and Challenges Ahead. 

  Shrestha, S; Morcavallo, A; Gorrini, C; Chesler, L

  The constitutive and dysregulated expression of the transcription factor MYCN has a central role in the pathogenesis of the paediatric brain tumour medulloblastoma, with an increased expression of this oncogene correlating ...
• Histamine signaling and metabolism identify potential biomarkers and therapies for lymphangioleiomyomatosis. 

  Herranz, C; Mateo, F; Baiges, A; Ruiz de Garibay, G; Junza, A; Johnson, SR; Miller, S; García, N; Capellades, J; Gómez, A; Vidal, A; Palomero, L; Espín, R; Extremera, AI; Blommaert, E; Revilla-López, E; Saez, B; Gómez-Ollés, S; Ancochea, J; Valenzuela, C; Alonso, T; Ussetti, P; Laporta, R; Xaubet, A; Rodríguez-Portal, JA; Montes-Worboys, A; Machahua, C; Bordas, J; Menendez, JA; Cruzado, JM; Guiteras, R; Bontoux, C; La Motta, C; Noguera-Castells, A; Mancino, M; Lastra, E; Rigo-Bonnin, R; Perales, JC; Viñals, F; Lahiguera, A; Zhang, X; Cuadras, D; van Moorsel, CHM; van der Vis, JJ; Quanjel, MJR; Filippakis, H; Hakem, R; Gorrini, C; Ferrer, M; Ugun-Klusek, A; Billett, E; Radzikowska, E; Casanova, Á; Molina-Molina, M; Roman, A; Yanes, O; Pujana, MA (2021-08-11)

  Inhibition of mTOR is the standard of care for lymphangioleiomyomatosis (LAM). However, this therapy has variable tolerability and some patients show progressive decline of lung function despite treatment. LAM diagnosis ...
• Fragment-based discovery of HSP70 inhibitors 

  O'Connor, S (2021-01-31)

  Heat Shock Protein 70s (HSP70s) are key molecular chaperones that are overexpressed in many cancers and are often associated with metastasis and poor prognosis. Dual silencing of two isoforms, HSP72 and HSC70, using siRNA ...
• Characterizing the metabolic fate of (2S, 4R)-4-[18F]fluoroglutamine and its applications in cancer imaging and treatment response monitoring 

  Nguyen, N (2021-01-31)

  Glutaminolysis is a crucial pathway to provide the nitrogen source in addition to the carbon source from the glycolysis pathway for proliferating cells. (2S, 4R)-4-[18F]fluoroglutamine (4-[18F]fluoroglutamine) is potentially ...
• Targeting therapeutic vulnerabilities associated with EWS fusion proteins in Ewing sarcoma 

  Romo Morales, A (2021-01-31)

  Ewing sarcoma (ES) is a are and aggressive bone tumour affecting children and young adults and that requires better therapeutic options to improve patient outcomes. ES is characterised by chromosomal rearrangements producing ...
• The PTEN and ATM axis controls the G1/S cell cycle checkpoint and tumorigenesis in HER2-positive breast cancer. 

  Bassi, C; Fortin, J; Snow, BE; Wakeham, A; Ho, J; Haight, J; You-Ten, A; Cianci, E; Buckler, L; Gorrini, C; Stambolic, V; Mak, TW (2021-05-31)

  The tumor suppressor PTEN is disrupted in a large proportion of cancers, including in HER2-positive breast cancer, where its loss is associated with resistance to therapy. Upon genotoxic stress, ataxia telangiectasia mutated ...
• Evolution of kinase polypharmacology across HSP90 drug discovery. 

  Antolin, AA; Clarke, PA; Collins, I; Workman, P; Al-Lazikani, B (2021-05-27)

  Most small molecules interact with several target proteins but this polypharmacology is seldom comprehensively investigated or explicitly exploited during drug discovery. Here, we use computational and experimental methods ...
• Impairment of a distinct cancer-associated fibroblast population limits tumour growth and metastasis. 

  Jungwirth, U; van Weverwijk, A; Evans, RJ; Jenkins, L; Vicente, D; Alexander, J; Gao, Q; Haider, S; Iravani, M; Isacke, CM (2021-06-10)

  Profiling studies have revealed considerable phenotypic heterogeneity in cancer-associated fibroblasts (CAFs) present within the tumour microenvironment, however, functional characterisation of different CAF subsets is ...
• PET imaging to monitor NET-1 and GD2 expression in neuroblastoma 

  Turnock, S (2021-01-31)

  Within neuroblastoma (NB), approximately 50% of patients are considered high-risk, presenting widespread distant metastasis and, frequently, MYCN amplification. NB is further characterised by the selective presence of the ...
• Droplet digital PCR-based detection of circulating tumor DNA from pediatric high grade and diffuse midline glioma patients. 

  Izquierdo, E; Proszek, P; Pericoli, G; Temelso, S; Clarke, M; Carvalho, DM; Mackay, A; Marshall, LV; Carceller, F; Hargrave, D; Lannering, B; Pavelka, Z; Bailey, S; Entz-Werle, N; Grill, J; Vassal, G; Rodriguez, D; Morgan, PS; Jaspan, T; Mastronuzzi, A; Vinci, M; Hubank, M; Jones, C (2021-01-27)

  <h4>Background</h4>The use of liquid biopsy is of potential high importance for children with high grade (HGG) and diffuse midline gliomas (DMG), particularly where surgical procedures are limited, and invasive biopsy ...
• Pathogenic ACVR1^R206H activation by Activin A-induced receptor clustering and autophosphorylation. 

  Ramachandran, A; Mehić, M; Wasim, L; Malinova, D; Gori, I; Blaszczyk, BK; Carvalho, DM; Shore, EM; Jones, C; Hyvönen, M; Tolar, P; Hill, CS

  Fibrodysplasia ossificans progressiva (FOP) and diffuse intrinsic pontine glioma (DIPG) are debilitating diseases that share causal mutations in ACVR1, a TGF-β family type I receptor. ACVR1^R206H is a frequent ...
• An antibody-drug conjugate with intracellular drug release properties showing specific cytotoxicity against CD7-positive cells. 

  Zhang, J; Jain, A; Milhas, S; Williamson, DJ; Mysliwy, J; Lodge, A; Thirlway, J; Al Nakeeb, M; Miller, A; Rabbitts, TH (2021-05-18)

  Refractory T cell acute leukaemias that no longer respond to treatment would benefit from new modalities that target T cell-specific surface proteins. T cell associated surface proteins (the surfaceome) offer possible ...
• Biomarkers Associating with PARP Inhibitor Benefit in Prostate Cancer in the TOPARP-B Trial. 

  Carreira, S; Porta, N; Arce-Gallego, S; Seed, G; Llop-Guevara, A; Bianchini, D; Rescigno, P; Paschalis, A; Bertan, C; Baker, C; Goodall, J; Miranda, S; Riisnaes, R; Figueiredo, I; Ferreira, A; Pereira, R; Crespo, M; Gurel, B; Nava Rodrigues, D; Pettitt, SJ; Yuan, W; Serra, V; Rekowski, J; Lord, CJ; Hall, E; Mateo, J; de Bono, JS (2021-05-27)

  PARP inhibitors are approved for treating advanced prostate cancers (APCs) with various defective DNA repair genes; however, further studies to clinically qualify predictive biomarkers are warranted. Herein we analyzed ...
• Near-InfraRed PhotoImmunoTherapy (NIR-PIT) for the local control of solid cancers: Challenges and potentials for human applications. 

  Paraboschi, I; Turnock, S; Kramer-Marek, G; Musleh, L; Barisa, M; Anderson, J; Giuliani, S

  Near-InfraRed PhotoImmunoTherapy (NIR-PIT) is a novel cancer-targeted treatment effected by a chemical conjugation between a photosensitiser (e.g. the NIR phthalocyanine dye IRDye700DX) and a cancer-targeting moiety (e.g. ...
• Armored CAR T-Cells: The Next Chapter in T-Cell Cancer Immunotherapy. 

  Hawkins, ER; D'Souza, RR; Klampatsa, A

  Chimeric antigen receptor (CAR) T-cell therapy engineers T-cells to express a synthetic receptor which redirects effector function to the tumor, to improve efficacy and reduce toxicities associated with conventional ...

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