Adult precision medicine: learning from the past to enhance the future

[1]  M. Gilbert,et al.  The effect of an adenosine A2A agonist on intra-tumoral concentrations of temozolomide in patients with recurrent glioblastoma , 2018, Fluids and Barriers of the CNS.

[2]  D. Schadendorf,et al.  Improved survival with ipilimumab in patients with metastatic melanoma. , 2010, The New England journal of medicine.

[3]  Rebecca A Betensky,et al.  Mosaic amplification of multiple receptor tyrosine kinase genes in glioblastoma. , 2011, Cancer cell.

[4]  Fuhui Long,et al.  An anatomic transcriptional atlas of human glioblastoma , 2018, Science.

[5]  Sankha S. Basu,et al.  Buparlisib in Patients With Recurrent Glioblastoma Harboring Phosphatidylinositol 3-Kinase Pathway Activation: An Open-Label, Multicenter, Multi-Arm, Phase II Trial. , 2019, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[6]  P. Wen,et al.  Individualized Screening Trial of Innovative Glioblastoma Therapy (INSIGhT): A Bayesian Adaptive Platform Trial to Develop Precision Medicines for Patients With Glioblastoma , 2019, JCO precision oncology.

[7]  D. Schadendorf,et al.  Encorafenib plus binimetinib versus vemurafenib or encorafenib in patients with BRAF-mutant melanoma (COLUMBUS): a multicentre, open-label, randomised phase 3 trial. , 2018, The Lancet. Oncology.

[8]  Jacob K. Asiedu,et al.  The Drug Repurposing Hub: a next-generation drug library and information resource , 2017, Nature Medicine.

[9]  K. Camphausen,et al.  A Phase 2 Study of Concurrent Radiation Therapy, Temozolomide, and the Histone Deacetylase Inhibitor Valproic Acid for Patients With Glioblastoma. , 2015, International journal of radiation oncology, biology, physics.

[10]  I. Laczmanska,et al.  Personalized medicine in oncology. New perspectives in management of gliomas , 2018, Contemporary oncology.

[11]  S. Piantadosi,et al.  Placebo-controlled trial of safety and efficacy of intraoperative controlled delivery by biodegradable polymers of chemotherapy for recurrent gliomas , 1995, The Lancet.

[12]  N. Sanai,et al.  Determination of total and unbound ribociclib in human plasma and brain tumor tissues using liquid chromatography coupled with tandem mass spectrometry , 2019, Journal of pharmaceutical and biomedical analysis.

[13]  M. Prados,et al.  A randomized, double-blind, placebo-controlled, phase 2 study of RMP-7 in combination with carboplatin administered intravenously for the treatment of recurrent malignant glioma. , 2003, Neuro-oncology.

[14]  M. Gilbert,et al.  Does Valproic Acid or Levetiracetam Improve Survival in Glioblastoma? A Pooled Analysis of Prospective Clinical Trials in Newly Diagnosed Glioblastoma. , 2016, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[15]  Mariella G. Filbin,et al.  An Integrative Model of Cellular States, Plasticity, and Genetics for Glioblastoma , 2019, Cell.

[16]  Jae-Geun Yoon,et al.  High-Throughput Chemical Screens Identify Disulfiram as an Inhibitor of Human Glioblastoma Stem Cells , 2012, Oncotarget.

[17]  A. Hauschild,et al.  Improved survival with vemurafenib in melanoma with BRAF V600E mutation. , 2011, The New England journal of medicine.

[18]  L. Cardone,et al.  Drug repurposing for the treatment of glioblastoma multiforme , 2017, Journal of experimental & clinical cancer research : CR.

[19]  E. Leuthardt,et al.  Hyperthermic Laser Ablation of Recurrent Glioblastoma Leads to Temporary Disruption of the Peritumoral Blood Brain Barrier , 2016, PloS one.

[20]  Edward S. Kim,et al.  Predictive biomarkers for response to EGFR-directed monoclonal antibodies for advanced squamous cell lung cancer , 2018, Annals of oncology : official journal of the European Society for Medical Oncology.

[21]  R. Verhaak,et al.  Molecular Evolution of IDH Wild-Type Glioblastomas Treated With Standard of Care Affects Survival and Design of Precision Medicine Trials: A Report From the EORTC 1542 Study. , 2019, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[22]  M. Bynoe,et al.  A2A Adenosine Receptor Regulates the Human Blood-Brain Barrier Permeability , 2014, Molecular Neurobiology.

[23]  K. Srivenugopal,et al.  Disulfiram is a direct and potent inhibitor of human O6-methylguanine-DNA methyltransferase (MGMT) in brain tumor cells and mouse brain and markedly increases the alkylating DNA damage. , 2014, Carcinogenesis.

[24]  Sean P. Palecek,et al.  Hypoxia-enhanced Blood-Brain Barrier Chip recapitulates human barrier function and shuttling of drugs and antibodies , 2019, Nature Communications.

[25]  C. Borlongan,et al.  Facilitation of drug entry into the CNS via transient permeation of blood brain barrier: laboratory and preliminary clinical evidence from bradykinin receptor agonist, Cereport , 2003, Brain Research Bulletin.

[26]  Mitchel S Berger,et al.  Intracerebral infusion of an EGFR-targeted toxin in recurrent malignant brain tumors. , 2008, Neuro-oncology.

[27]  Liang Cheng,et al.  Molecular testing for BRAF mutations to inform melanoma treatment decisions: a move toward precision medicine , 2017, Modern Pathology.

[28]  Susan M. Chang,et al.  Response Assessment in Neuro-Oncology Clinical Trials. , 2017, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[29]  M. Pangalos,et al.  Pioneering government-sponsored drug repositioning collaborations: progress and learning , 2015, Nature Reviews Drug Discovery.

[30]  T. Mikkelsen,et al.  Talampanel with standard radiation and temozolomide in patients with newly diagnosed glioblastoma: a multicenter phase II trial. , 2009, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[31]  Tânia Cova,et al.  Computational modeling in glioblastoma: from the prediction of blood-brain barrier permeability to the simulation of tumor behavior. , 2018, Future medicinal chemistry.

[32]  N. Iqbal,et al.  Imatinib: A Breakthrough of Targeted Therapy in Cancer , 2014, Chemotherapy research and practice.

[33]  M. Plattén EGFRvIII vaccine in glioblastoma-InACT-IVe or not ReACTive enough? , 2017, Neuro-oncology.

[34]  Eric P. Winer,et al.  Breast Cancer Treatment: A Review , 2019, JAMA.

[35]  H. Fine,et al.  Modeling Patient-Derived Glioblastoma with Cerebral Organoids. , 2019, Cell reports.

[36]  C. Viret,et al.  Adenosine receptor signaling: a key to opening the blood–brain door , 2015, Fluids and Barriers of the CNS.

[37]  Ying Cheng,et al.  Overall Survival with Osimertinib in Untreated, EGFR-Mutated Advanced NSCLC. , 2019, The New England journal of medicine.

[38]  D. Garbossa,et al.  NTRK Fusions in Central Nervous System Tumors: A Rare, but Worthy Target , 2020, International journal of molecular sciences.

[39]  Madeline A. Lancaster,et al.  Cerebral organoids model human brain development and microcephaly , 2013, Nature.

[40]  Raymond Y Huang,et al.  ACTR-46. AG-120, A FIRST-IN-class MUTANT IDH1 INHIBITOR IN PATIENTS WITH RECURRENT OR PROGRESSIVE IDH1 MUTANT GLIOMA: UPDATED RESULTS FROM THE PHASE 1 NON-ENHANCING GLIOMA POPULATION , 2017 .

[41]  D. Kraemer,et al.  Cognitive outcomes and long-term follow-up results after enhanced chemotherapy delivery for primary central nervous system lymphoma. , 2000, Neurosurgery.

[42]  B. Oh,et al.  Protein carbon-13 spin systems by a single two-dimensional nuclear magnetic resonance experiment. , 1988, Science.

[43]  Alireza Khoshnevisan,et al.  A conceptually new treatment approach for relapsed glioblastoma: Coordinated undermining of survival paths with nine repurposed drugs (CUSP9) by the International Initiative for Accelerated Improvement of Glioblastoma Care , 2013, Oncotarget.

[44]  K. Kerr,et al.  Precision medicine in NSCLC and pathology: how does ALK fit in the pathway? , 2016, Annals of oncology : official journal of the European Society for Medical Oncology.

[45]  Steven J. M. Jones,et al.  Clonal expansion and epigenetic reprogramming following deletion or amplification of mutant IDH1 , 2017, Proceedings of the National Academy of Sciences.

[46]  Guido Reifenberger,et al.  Molecular Diagnostics of Gliomas Using Next Generation Sequencing of a Glioma‐Tailored Gene Panel , 2017, Brain pathology.

[47]  Colin Watts,et al.  LONGITUDINAL MOLECULAR TRAJECTORIES OF DIFFUSE GLIOMA IN ADULTS , 2019, Nature.

[48]  L. Liau,et al.  Cancer-associated IDH1 mutations produce 2-hydroxyglutarate , 2009, Nature.

[49]  G. Reifenberger,et al.  The 2016 World Health Organization Classification of Tumors of the Central Nervous System: a summary , 2016, Acta Neuropathologica.

[50]  G. Reifenberger,et al.  IDH1(R132H) mutation increases murine haematopoietic progenitors and alters epigenetics , 2012, Nature.

[51]  S. Konduri,et al.  Levetiracetam enhances p53-mediated MGMT inhibition and sensitizes glioblastoma cells to temozolomide. , 2010, Neuro-oncology.

[52]  Víctor M. Pérez-García,et al.  Glioblastoma on a microfluidic chip: Generating pseudopalisades and enhancing aggressiveness through blood vessel obstruction events , 2017, Neuro-oncology.

[53]  R. Kast,et al.  CUSP9* treatment protocol for recurrent glioblastoma: aprepitant, artesunate, auranofin, captopril, celecoxib, disulfiram, itraconazole, ritonavir, sertraline augmenting continuous low dose temozolomide , 2014, Oncotarget.

[54]  R. Bartus,et al.  Cereport (RMP-7) increases carboplatin levels in brain tumors after pretreatment with dexamethasone. , 1999, Neuro-oncology.

[55]  J. Blay,et al.  ACTR-30. UPDATED EFFICACY AND SAFETY OF DABRAFENIB PLUS TRAMETINIB IN PATIENTS WITH RECURRENT/REFRACTORY BRAF V600E–MUTATED HIGH-GRADE GLIOMA (HGG) AND LOW-GRADE GLIOMA (LGG) , 2019, Neuro-Oncology.

[56]  Mark S. Cohen,et al.  The discovery of vemurafenib for the treatment of BRAF-mutated metastatic melanoma , 2016, Expert opinion on drug discovery.

[57]  M. Ahn,et al.  A phase II, multicenter, two cohort study of 160 mg osimertinib in EGFR T790M-positive non-small cell lung cancer patients with brain metastases or leptomeningeal disease who progressed on prior EGFR TKI therapy. , 2020, Annals of oncology : official journal of the European Society for Medical Oncology.

[58]  O. Garaschuk,et al.  Brain tumour cells interconnect to a functional and resistant network , 2015, Nature.

[59]  D. Busam,et al.  An Integrated Genomic Analysis of Human Glioblastoma Multiforme , 2008, Science.

[60]  M. Bynoe,et al.  Adenosine Receptor Signaling Modulates Permeability of the Blood–Brain Barrier , 2011, The Journal of Neuroscience.

[61]  D. Fortin,et al.  Recent Advances in Blood–Brain Barrier Disruption as a CNS Delivery Strategy , 2008, The AAPS Journal.

[62]  Jie Wu,et al.  The emerging treatment landscape of targeted therapy in non-small-cell lung cancer. , 2019, Signal transduction and targeted therapy.

[63]  Remigijus Didziapetris,et al.  Improving the prediction of drug disposition in the brain , 2013, Expert opinion on drug metabolism & toxicology.

[64]  N. Galldiks,et al.  Current status of PET imaging in neuro-oncology , 2019, Neuro-oncology advances.

[65]  A. Bode,et al.  Precision medicine: the foundation of future cancer therapeutics , 2017, npj Precision Oncology.

[66]  E. Cho,et al.  CNS Response to Osimertinib Versus Standard Epidermal Growth Factor Receptor Tyrosine Kinase Inhibitors in Patients With Untreated EGFR-Mutated Advanced Non-Small-Cell Lung Cancer. , 2018, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[67]  G. Ha,et al.  Microtubule-targeting agents can sensitize cancer cells to ionizing radiation by an interphase-based mechanism , 2017, OncoTargets and therapy.

[68]  Raymond Y Huang,et al.  Ivosidenib in Isocitrate Dehydrogenase 1–Mutated Advanced Glioma , 2020, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[69]  Nir Lipsman,et al.  Blood-Brain Barrier Opening in Primary Brain Tumors with Non-invasive MR-Guided Focused Ultrasound: A Clinical Safety and Feasibility Study , 2019, Scientific Reports.

[70]  William A Banks,et al.  Characteristics of compounds that cross the blood-brain barrier , 2009, BMC neurology.

[71]  Pieter Wesseling,et al.  cIMPACT‐NOW update 6: new entity and diagnostic principle recommendations of the cIMPACT‐Utrecht meeting on future CNS tumor classification and grading , 2020, Brain pathology.

[72]  J. Ahn,et al.  Osimertinib improves overall survival in EGFR-mutated non-small cell lung cancer patients with leptomeningeal metastases regardless of T790M mutational status. , 2020, Journal of thoracic oncology : official publication of the International Association for the Study of Lung Cancer.

[73]  R. Wahl,et al.  The effect of regadenoson on the integrity of the human blood–brain barrier, a pilot study , 2017, Journal of Neuro-Oncology.

[74]  S. Choe,et al.  Ivosidenib (AG-120) Induced Durable Remissions and Transfusion Independence in Patients with IDH1-Mutant Relapsed or Refractory Myelodysplastic Syndrome: Results from a Phase 1 Dose Escalation and Expansion Study , 2018, Blood.

[75]  H. Döhner,et al.  Ivosidenib or Enasidenib Combined with Induction and Consolidation Chemotherapy in Patients with Newly Diagnosed AML with an IDH1 or IDH2 Mutation Is Safe, Effective, and Leads to MRD-Negative Complete Remissions , 2018, Blood.

[76]  M. Zanetti A second chance for telomerase reverse transcriptase in anticancer immunotherapy , 2017, Nature Reviews Clinical Oncology.

[77]  S. Yip,et al.  Disulfiram, a drug widely used to control alcoholism, suppresses self-renewal of glioblastoma and overrides resistance to temozolomide , 2012, Oncotarget.

[78]  W. Mason,et al.  EORTC 1709/CCTG CE.8: A phase III trial of marizomib in combination with standard temozolomide-based radiochemotherapy versus standard temozolomide-based radiochemotherapy alone in patients with newly diagnosed glioblastoma. , 2019, Journal of Clinical Oncology.

[79]  Mariella G. Filbin,et al.  Single-cell RNA-seq supports a developmental hierarchy in human oligodendroglioma , 2016, Nature.

[80]  David T. W. Jones,et al.  N2M2 (NOA-20) phase I/II trial of molecularly matched targeted therapies plus radiotherapy in patients with newly diagnosed non-MGMT hypermethylated glioblastoma. , 2018, Neuro-oncology.

[81]  E. Frenkel,et al.  Osmotic blood-brain barrier modification: clinical documentation by enhanced CT scanning and/or radionuclide brain scanning. , 1983, AJR. American journal of roentgenology.

[82]  A. Kim,et al.  Developments in Blood-Brain Barrier Penetrance and Drug Repurposing for Improved Treatment of Glioblastoma , 2018, Front. Oncol..

[83]  Shawn M. Gillespie,et al.  Single-cell RNA-seq highlights intratumoral heterogeneity in primary glioblastoma , 2014, Science.

[84]  R. McLendon,et al.  IDH1 and IDH2 mutations in gliomas. , 2009, The New England journal of medicine.

[85]  E. Maher,et al.  ACTR-31. PHASE 1 STUDY OF AG-881, AN INHIBITOR OF MUTANT IDH1 AND IDH2: RESULTS FROM THE RECURRENT/PROGRESSIVE GLIOMA POPULATION , 2018, Neuro-Oncology.

[86]  M. Rudek,et al.  The effect of regadenoson-induced transient disruption of the blood–brain barrier on temozolomide delivery to normal rat brain , 2016, Journal of Neuro-Oncology.

[87]  Mariella G. Filbin,et al.  Decoupling genetics, lineages, and microenvironment in IDH-mutant gliomas by single-cell RNA-seq , 2017, Science.

[88]  A. Till,et al.  Longitudinal heterogeneity in glioblastoma: moving targets in recurrent versus primary tumors , 2019, Journal of Translational Medicine.

[89]  D. Hanahan,et al.  Roadmap for the Emerging Field of Cancer Neuroscience , 2020, Cell.

[90]  C. Wirtz,et al.  Current state and future perspective of drug repurposing in malignant glioma. , 2019, Seminars in cancer biology.

[91]  D. Haussler,et al.  The Somatic Genomic Landscape of Glioblastoma , 2013, Cell.

[92]  Timothy C Ryken,et al.  Toward precision medicine in glioblastoma: the promise and the challenges. , 2015, Neuro-oncology.

[93]  R. Reddel,et al.  Alternative lengthening of telomeres: models, mechanisms and implications , 2010, Nature Reviews Genetics.

[94]  Volker Hovestadt,et al.  Resolving medulloblastoma cellular architecture by single-cell genomics , 2019, Nature.

[95]  Henry Brem,et al.  Recent Advances in Brain Tumor Therapy: Local Intracerebral Drug Delivery by Polymers , 2004, Investigational New Drugs.

[96]  Pieter Wesseling,et al.  Glioma: experimental models and reality , 2017, Acta Neuropathologica.

[97]  Jennie W. Taylor,et al.  Prospective Feasibility Trial for Genomics-Informed Treatment in Recurrent and Progressive Glioblastoma , 2017, Clinical Cancer Research.

[98]  T. Kuner,et al.  Glutamatergic synaptic input to glioma cells drives brain tumour progression , 2019, Nature.

[99]  Joshua M. Korn,et al.  Comprehensive genomic characterization defines human glioblastoma genes and core pathways , 2008, Nature.

[100]  Gary L. Gallia,et al.  TERT promoter mutations occur frequently in gliomas and a subset of tumors derived from cells with low rates of self-renewal , 2013, Proceedings of the National Academy of Sciences.

[101]  H. Monyer,et al.  Emerging intersections between neuroscience and glioma biology , 2019, Nature Neuroscience.

[102]  S. Piantadosi,et al.  Placebo-controlled trial of safety and efficacy of intraoperative controlled delivery by biodegradable polymers of chemotherapy for recurrent gliomas. The Polymer-brain Tumor Treatment Group. , 1995, Lancet.

[103]  Malorye Allison,et al.  NCATS launches drug repurposing program , 2012, Nature Biotechnology.

[104]  A. Shaw,et al.  Targeting ALK: Precision Medicine Takes on Drug Resistance. , 2017, Cancer discovery.

[105]  P. Vigneri,et al.  Imatinib mesylate in chronic myeloid leukemia: frontline treatment and long-term outcomes , 2016, Expert review of anticancer therapy.

[106]  U. Brown-Glaberman,et al.  HER2-targeted therapy for early-stage breast cancer: a comprehensive review. , 2014, Oncology.

[107]  P. Ascierto,et al.  Combined vemurafenib and cobimetinib in BRAF-mutated melanoma. , 2014, The New England journal of medicine.

[108]  F. Hirsch,et al.  EGFR-directed monoclonal antibodies in combination with chemotherapy for treatment of non-small-cell lung cancer: an updated review of clinical trials and new perspectives in biomarkers analysis. , 2019, Cancer treatment reviews.

[109]  Susan Chang,et al.  Phase III randomized trial of CED of IL13-PE38QQR vs Gliadel wafers for recurrent glioblastoma. , 2010, Neuro-oncology.

[110]  Leland S. Hu,et al.  Is the blood–brain barrier really disrupted in all glioblastomas? A critical assessment of existing clinical data , 2018, Neuro-oncology.

[111]  N. McGranahan,et al.  Biological and therapeutic impact of intratumor heterogeneity in cancer evolution. , 2015, Cancer cell.

[112]  P. Wen,et al.  Use of the Response Assessment in Neuro-Oncology (RANO) criteria in clinical trials and clinical practice , 2019, CNS oncology.

[113]  K. Srivenugopal,et al.  Beyond Brooding on Oncometabolic Havoc in IDH-Mutant Gliomas and AML: Current and Future Therapeutic Strategies , 2018, Cancers.

[114]  R. Stupp,et al.  Use of metformin and outcome of patients with newly diagnosed glioblastoma: Pooled analysis , 2020, International journal of cancer.

[115]  Tracy T Batchelor,et al.  Developmental and oncogenic programs in H3K27M gliomas dissected by single-cell RNA-seq , 2018, Science.

[116]  M. Aghi,et al.  Convection-enhanced delivery in glioblastoma: a review of preclinical and clinical studies. , 2017, Journal of neurosurgery.

[117]  David M. Hyman,et al.  BRAF Inhibition in BRAFV600-Mutant Gliomas: Results From the VE-BASKET Study , 2018, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[118]  R. Bartus,et al.  Cereport™ (RMP-7) Increases the Permeability of Human Brain Microvascular Endothelial Cell Monolayers1 , 1999, Pharmaceutical Research.

[119]  J. Sarkaria,et al.  Baseline Requirements for Novel Agents Being Considered for Phase II/III Brain Cancer Efficacy Trials:Conclusions from the Adult Brain Tumor Consortium's First Workshop on CNS Drug Delivery. , 2020, Neuro-oncology.

[120]  Brian M Alexander,et al.  Adaptive Global Innovative Learning Environment for Glioblastoma: GBM AGILE , 2017, Clinical Cancer Research.

[121]  Philipp M. Altrock,et al.  The mathematics of cancer: integrating quantitative models , 2015, Nature Reviews Cancer.

[122]  Weiguang Wang,et al.  How could a drug used to treat alcoholism also be effective against glioblastoma? , 2013, Expert review of anticancer therapy.

[123]  Arthur André,et al.  Safety and Feasibility of Repeated and Transient Blood–Brain Barrier Disruption by Pulsed Ultrasound in Patients with Recurrent Glioblastoma , 2019, Clinical Cancer Research.

[124]  G. Lenz,et al.  Vinblastine and antihelmintic mebendazole potentiate temozolomide in resistant gliomas , 2018, Investigational New Drugs.

[125]  M. Symons,et al.  Repurposing Mebendazole as a Replacement for Vincristine for the Treatment of Brain Tumors , 2017, Molecular medicine.