Catherine Smith, MD

Assoc. Professor In Residence
Medicine

My laboratory focuses on identification of therapeutic resistance mechanisms and novel treatment strategies for acute myeloid leukemia (AML). We have a particular emphasis on AML associated with mutations in Fms-like Tyrosine Kinase-3 (FLT3). FLT3 is mutated in ~30% of AML, with constitutively activating FLT3 internal tandem duplication (ITD) mutations conferring a poor prognosis. We employ a prototypical “bedside to bench and back” approach to the problem of cancer drug resistance, founded on the belief that the ultimate pathway to improved cancer therapy begins with translational studies that utilize samples from patients who have undergone therapy in real time. This strategy allows us to interrogate how tumors can evolve under the selective pressure of cancer therapy and allows us to devise ways to circumvent these evolutionary adaptations.

Publications: 

A phase 1 study of the irreversible FLT3 inhibitor FF-10101 in relapsed or refractory acute myeloid leukemia.

Blood advances

Levis MJ, Perl AE, Schiller GJ, Fathi AT, Roboz GJ, Wang ES, Altman JK, Rajkhowa T, Ando M, Suzuki T, Subach RA, Maier G, Madden T, Johansen M, Cheung K, Kurman M, Smith CC

LNK/SH2B3 as a novel driver in juvenile myelomonocytic leukemia.

Haematologica

Wintering A, Hecht A, Meyer J, Wong EB, Hübner J, Abelson S, Feldman K, Kennedy VE, Peretz CAC, French DL, Maguire JA, Jobaliya C, Vasquez MR, Desai S, Dulman R, Nemecek E, Haines H, Hammad M, El Haddad A, Kogan SC, Abdullaev Z, Chehab FF, Tasian SK, Smith CC, Loh ML, Stieglitz E

FLT3 targeting in the modern era: from clonal selection to combination therapies.

International journal of hematology

Kennedy VE, Smith CC

Genomic Characterization of Newly Diagnosed Acute Myeloid Leukemia in Patients Age 60 Years and Older; A Report from the Beat AML Master Trial.

Blood

Fieke W Hoff, Ying Huang, Rina Li Welkie, Ronan T. Swords, Elie Traer, Eytan M. Stein, Tara L Lin, Prapti A. Patel, Robert H. Collins, Maria R. Baer, Vu H. Duong, William Blum, Martha Arellano, Wendy Stock, Olatoyosi Odenike, Robert L. Redner, Tibor J. Kovacsovics, Michael W. Deininger, Joshua F. Zeidner, Rebecca Olin, Catherine C. Smith, James M. Foran, Gary J. Schiller, Emily K Curran, Kristin L Koenig, Nyla A. Heerema, Timothy Chen, Molly Martycz, Mona Stefanos, Sonja Gullen Marcus, Leonard Rosenberg, Brian J. Druker, Ross L Levine, Amy Burd, Ashley Owen Yocum, Uma M. Borate, Alice Mims, John C. Byrd, Yazan F. Madanat

Influence of Pre-Treatment Features and Therapy Choice By Physicians on Overall Survival in Older Adults with Acute Myeloid Leukemia: A Report from the Beat AML Master Trial.

Blood

Uma M. Borate, Ying Huang, Rina Li Welkie, Ronan T. Swords, Elie Traer, Eytan M. Stein, Tara L Lin, Yazan F. Madanat, Prapti A. Patel, Robert H. Collins, Maria R. Baer, Vu H. Duong, William Blum, Martha Arellano, Wendy Stock, Olatoyosi Odenike, Robert L. Redner, Tibor J. Kovacsovics, Michael W. Deininger, Joshua F. Zeidner, Rebecca Olin, Catherine C. Smith, James M. Foran, Gary J. Schiller, Emily K Curran, Kristin L Koenig, Nyla A. Heerema, Timothy Chen, Molly Martycz, Mona Stefanos, Sonja Gullen Marcus, Leonard Rosenberg, Brian J. Druker, Ross L Levine, Amy Burd, Ashley Owen Yocum, Alice Mims, John C. Byrd

Mediator Kinase/CDK8 Inhibition As a Strategy to Improve FLT3 Inhibitor Activity in Acute Myeloid Leukemia.

Blood

Timothy T. Ferng, Samantha M. Pintar, Vanessa E. Kennedy, Theodore C. Tarver, Shaheen Kabir, Veronica Steri, Juan Antonio Camara Serrano, Fernando Salangsang, Paul Phojanakong, Juwita Hübner, Carolina E. Morales, Jose M. Rivera, Aaron C. Logan, Benjamin Braun, Elliot Stieglitz, Luke A. Gilbert, Catherine C. Smith

Multiomic Single-Cell Sequencing May Distinguish BCR-ABL-Mutated Acute Myeloid Leukemia (AML) from Blast Crisis Chronic Myelogenous Leukemia (BC-CML).

Blood

Vanessa E. Kennedy, Cheryl A C Peretz, Andrew Koh, Elaine Tran, Dalia Dhingra, Saurabh Gulati, Adam Sciambi, Olga Sala-Torra, Lan Beppu, Aaron C. Logan, Alexander Perl, Wendy Stock, Geoffrey L Uy, Ann-Kathrin Eisfeld, Jerald P. Radich, Catherine C. Smith

Threshold Attenuation of Perk Signaling Is Sufficient to Overcome RAS-Mediated FLT3 Inhibitor Resistance.

Blood

Cheryl A C Peretz, Elaine Tran, Bogdan Popescu, Vanessa E. Kennedy, Tanya Kumar, Lisa McGary, Natalie Tantisira, Eric Wong, Jose M. Rivera, Simayijiang Xirenayi, Elliot Stieglitz, Catherine C. Smith, Juwita Hübner

Allosteric SHP2 inhibition increases apoptotic dependency on BCL2 and synergizes with venetoclax in FLT3- and KIT-mutant AML.

Cell reports. Medicine

Popescu B, Stahlhut C, Tarver TC, Wishner S, Lee BJ, Peretz CAC, Luck C, Phojanakong P, Camara Serrano JA, Hongo H, Rivera JM, Xirenayi S, Chukinas JA, Steri V, Tasian SK, Stieglitz E, Smith CC

Structural surfaceomics reveals an AML-specific conformation of integrin ß2 as a CAR T cellular therapy target.

Nature cancer

Mandal K, Wicaksono G, Yu C, Adams JJ, Hoopmann MR, Temple WC, Izgutdina A, Escobar BP, Gorelik M, Ihling CH, Nix MA, Naik A, Xie WH, Hübner J, Rollins LA, Reid SM, Ramos E, Kasap C, Steri V, Serrano JAC, Salangsang F, Phojanakong P, McMillan M, Gavallos V, Leavitt AD, Logan AC, Rooney CM, Eyquem J, Sinz A, Huang BJ, Stieglitz E, Smith CC, Moritz RL, Sidhu SS, Huang L, Wiita AP

The FLT3N701K mutation causes clinical AML resistance to gilteritinib and triggers TKI sensitivity switch to quizartinib.

American journal of hematology

Joshi SK, Pittsenbarger J, Kennedy VE, Peretz CAC, Perl AE, Smith CC, Tyner JW, Druker BJ, Traer E

Association of geriatric measures and global frailty with cognitive decline after allogeneic hematopoietic cell transplantation in older adults.

Journal of geriatric oncology

Huang LW, Shi Y, Andreadis C, Logan AC, Mannis GN, Smith CC, Gaensler KML, Martin TG, Damon LE, Boscardin WJ, Steinman MA, Olin RL

Standardising acute myeloid leukaemia classification systems: a perspective from a panel of international experts.

The Lancet. Haematology

Shallis RM, Daver N, Altman JK, Komrokji RS, Pollyea DA, Badar T, Bewersdorf JP, Bhatt VR, de Botton S, de la Fuente Burguera A, Carraway HE, Desai P, Dillon R, Duployez N, El Chaer F, Fathi AT, Freeman SD, Gojo I, Grunwald MR, Jonas BA, Konopleva M, Lin TL, Mannis GN, Mascarenhas J, Michaelis LC, Mims AS, Montesinos P, Pozdnyakova O, Pratz KW, Schuh AC, Sekeres MA, Smith CC, Stahl M, Subklewe M, Uy GL, Voso MT, Walter RB, Wang ES, Zeidner JF, Žucenka A, Zeidan AM

A phase 1/2 dose escalation study of the BCL-2 inhibitor ZN-d5 and the WEE1 inhibitor azenosertib (ZN-c3) in patients (Pts) with acute myeloid leukemia (AML).

Journal of Clinical Oncology

Catherine Choy Smith, Pankit Vachhani, Guillermo Garcia-Manero, Nicole Renee Grieselhuber, Guru Subramanian Guru Murthy, Astha Bhatia, Jatinder Arora, Hooman Izadi, Anthony Fiorino, Raajit Rampal

Multiomic Single Cell Sequencing Identifies Stemlike Nature of Mixed Phenotype Acute Leukemia and Provides Novel Risk Stratification.

bioRxiv : the preprint server for biology

Peretz CAC, Kennedy VE, Walia A, Delley CL, Koh A, Tran E, Clark IC, Hayford CE, D'Amato C, Xue Y, Fontanez KM, Roy R, Logan AC, Perl AE, Abate A, Olshen A, Smith CC

Microfluidics-free single-cell genomics with templated emulsification.

Nature biotechnology

Clark IC, Fontanez KM, Meltzer RH, Xue Y, Hayford C, May-Zhang A, D'Amato C, Osman A, Zhang JQ, Hettige P, Ishibashi JSA, Delley CL, Weisgerber DW, Replogle JM, Jost M, Phong KT, Kennedy VE, Peretz CAC, Kim EA, Song S, Karlon W, Weissman JS, Smith CC, Gartner ZJ, Abate AR

304 HLA-DPB1 Mismatch Permissiveness and Activating KIR Haplotypes Have Different Impact on Allogeneic Hematopoietic Cell Transplant Survival Outcomes in Acute Leukemias and Myelodysplastic Syndrome.

Transplantation and cellular therapy

Jerry Lee, Raja Rajalingam, Denice Kong, Weiyun Ai, Charalambos B. Andreadis, Lloyd E. Damon, Neil Dunavin, Karin M. Gaensler, Lawrence D. Kaplan, Thomas G. Martin, Rebecca L. Olin, Peter H. Sayre, Catherine C. Smith, Jeffrey L. Wolf, Aaron C. Logan

Venetoclax Plus Gilteritinib for FLT3-Mutated Relapsed/Refractory Acute Myeloid Leukemia.

Journal of clinical oncology : official journal of the American Society of Clinical Oncology

Daver N, Perl AE, Maly J, Levis M, Ritchie E, Litzow M, McCloskey J, Smith CC, Schiller G, Bradley T, Tiu RV, Naqvi K, Dail M, Brackman D, Siddani S, Wang J, Chyla B, Lee P, Altman JK

Abstract LB516A: The irreversible FLT3 inhibitor FF-10101 is active against a diversity of FLT3 inhibitor resistance mechanisms.

Cancer research

Timothy T. Ferng, Daisuke Terada, Makoto Ando, Theodore C. Tarver, Fihr Chaudhary, Kimberly C. Lin, Aaron C. Logan, Catherine C. Smith

Clinical outcomes in patients with relapsed/refractory FLT3-mutated acute myeloid leukemia treated with gilteritinib who received prior midostaurin or sorafenib.

Blood cancer journal

Perl AE, Hosono N, Montesinos P, Podoltsev N, Martinelli G, Panoskaltsis N, Recher C, Smith CC, Levis MJ, Strickland S, Röllig C, Groß-Langenhoff M, Chou WC, Lee JH, Yokoyama H, Hasabou N, Lu Q, Tiu RV, Altman JK

The Irreversible FLT3 Inhibitor FF-10101 Is Active Against a Diversity of FLT3 Inhibitor Resistance Mechanisms.

Molecular cancer therapeutics

Ferng TT, Terada D, Ando M, Tarver TC, Chaudhary F, Lin KC, Logan AC, Smith CC

Molecular profile of FLT3-mutated relapsed/refractory AML patients in the phase 3 ADMIRAL study of gilteritinib.

Blood advances

Smith CC, Levis MJ, Perl AE, Hill JE, Rosales M, Bahceci E

Patterns and predictors of functional decline after alloHCT in older adults.

Transplantation and cellular therapy

Huang LW, Sheng Y, Andreadis C, Logan AC, Mannis GN, Smith CC, Gaensler KML, Martin TG, Damon LE, Huang CY, Olin RL

Allosteric SHP2 Inhibitor RMC4550 Synergizes with Venetoclax in FLT3 and KIT Mutant Acute Myeloid Leukemia.

Blood

Bogdan Popescu, Carlos Stahlhut, Theodore C. Tarver, Timothy T. Ferng, Cheryl Peretz, Sydney Wishner, Paul Phojanakong, Veronica Steri, Eric B. Wong, Jose M. Rivera, Elliot Stieglitz, Catherine C. Smith

Being Present-CML: A Distress Reduction Intervention for Patients with Chronic Myeloid Leukemia Taking Tyrosine Kinase Inhibitors.

Blood

Kelly L. Schoenbeck, Anand Dhruva, Albert Lee, Neha G. Goyal, Adrienne K. Yang, Sa Ra Park, Catherine C. Smith, Neil P Shah, Chloe E. Atreya

RNA Polymerase II Is a Therapeutic Target to Overcome FLT3 Inhibitor Resistance Mediated By the Bone Marrow Microenvironment.

Blood

Timothy T. Ferng, Theodore C. Tarver, Shaheen Kabir, Benjamin Braun, Aaron C. Logan, Luke A. Gilbert, Catherine C. Smith

Single Cell Sequencing of Pediatric Acute Myeloid Leukemia Reveals Clonal Evolution to Relapse on Combination Chemotherapy with Sorafenib.

Blood

Cheryl A C Peretz, Vanessa E Kennedy, Rhonda E. Ries, Soheil Meshinchi, Catherine C. Smith

Venetoclax in Combination with Gilteritinib Demonstrates Molecular Clearance of FLT3 mutation in Relapsed/Refractory FLT3 -Mutated Acute Myeloid Leukemia.

Blood

Naval Daver, Alexander E. Perl, Joseph Maly, Mark Levis, Ellen Ritchie, Mark R. Litzow, James McCloskey, Catherine C. Smith, Gary J. Schiller, Terrence Bradley, Ramon V. Tiu, Kiran Naqvi, Monique Dail, Satya Siddani, Jing Wang, Brenda Chyla, Paul Lee, Jessica K. Altman

AML-091: Clinical Outcomes in Patients with Relapsed/Refractory Acute Myeloid Leukemia Treated with Gilteritinib Who Received Prior Midostaurin or Sorafenib.

Clinical Lymphoma Myeloma & Leukemia

Alexander E Perl, Jessica K Altman, Naoko Hosono, Pau Montesinos, Nikolai Podoltsev, Giovanni Martinelli, Catherine C Smith, Mark J Levis, Christoph Röllig, Marco Groß-Langenhoff, Nahla Hasabou, Qiaoyang Lu, Ramon V Tiu

AML-162: Efficacy and Safety of Venetoclax in Combination with Gilteritinib for Relapsed/Refractory FLT3-Mutated Acute Myeloid Leukemia: Updated Analyses of a Phase 1b Study.

Clinical Lymphoma Myeloma & Leukemia

Jessica K. Altman, Naval Daver, Joseph Maly, Mark Levis, Ellen Ritchie, Mark Litzow, James McCloskey, Catherine C. Smith, Gary Schiller, Terrence Bradley, Ramon V. Tiu, Kiran Naqvi, Satya Siddani, Jing Wang, Paul Lee, Alexander E. Perl

Correction to: Pharmacokinetic Profile of Gilteritinib: A Novel FLT-3 Tyrosine Kinase Inhibitor.

Clinical pharmacokinetics

James AJ, Smith CC, Litzow M, Perl AE, Altman JK, Shepard D, Kadokura T, Souda K, Patton M, Lu Z, Liu C, Moy S, Levis MJ, Bahceci E

Poster: AML-091: Clinical Outcomes in Patients with Relapsed/Refractory Acute Myeloid Leukemia Treated with Gilteritinib Who Received Prior Midostaurin or Sorafenib.

Clinical Lymphoma Myeloma & Leukemia

Alexander E Perl, Jessica K Altman, Naoko Hosono, Pau Montesinos, Nikolai Podoltsev, Giovanni Martinelli, Catherine C Smith, Mark J Levis, Christoph Röllig, Marco Groß-Langenhoff, Nahla Hasabou, Qiaoyang Lu, Ramon V Tiu

Poster: AML-162: Efficacy and Safety of Venetoclax in Combination with Gilteritinib for Relapsed/Refractory FLT3-Mutated Acute Myeloid Leukemia: Updated Analyses of a Phase 1b Study.

Clinical Lymphoma Myeloma & Leukemia

Jessica K. Altman, Naval Daver, Joseph Maly, Mark Levis, Ellen Ritchie, Mark Litzow, James McCloskey, Catherine C. Smith, Gary Schiller, Terrence Bradley, Ramon V. Tiu, Kiran Naqvi, Satya Siddani, Jing Wang, Paul Lee, Alexander E. Perl

The EBF1-PDGFRB T681I mutation is highly resistant to imatinib and dasatinib in vitro and detectable in clinical samples prior to treatment.

Haematologica

Tran TH, Nguyen JV, Stecula A, Akutagawa J, Moorman AV, Braun BS, Sali A, Mullighan CG, Shah NP, Dai Y, Devidas M, Roberts KG, Smith CC, Loh ML

Recurrent Mutations in Cyclin D3 Confer Clinical Resistance to FLT3 Inhibitors in Acute Myeloid Leukemia.

Clinical cancer research : an official journal of the American Association for Cancer Research

Smith CC, Viny AD, Massi E, Kandoth C, Socci ND, Rapaport F, Najm M, Medina-Martinez JS, Papaemmanuil E, Tarver TC, Hsu HH, Le MH, West B, Bollag G, Taylor BS, Levine RL, Shah NP

Phase 1 first-in-human study of irreversible FLT3 inhibitor FF-10101-01 in relapsed or refractory acute myeloid leukemia.

Journal of Clinical Oncology

Mark J. Levis, Catherine Choy Smith, Alexander E. Perl, Gary J. Schiller, Amir Tahmasb Fathi, Gail J. Roboz, Eunice S. Wang, Jessica K. Altman, Makoto Ando, Takeaki Suzuki, Ruth Ann Subach, Gary Maier, Timothy Madden, Mary Johansen, Kin Cheung, Michael R. Kurman

Joint profiling of DNA and proteins in single cells to dissect genotype-phenotype associations in leukemia.

Nature communications

Demaree B, Delley CL, Vasudevan HN, Peretz CAC, Ruff D, Smith CC, Abate AR

Single-cell DNA sequencing reveals complex mechanisms of resistance to quizartinib.

Blood advances

Peretz CAC, McGary LHF, Kumar T, Jackson H, Jacob J, Durruthy-Durruthy R, Levis MJ, Perl A, Huang BJ, Smith CC

Safety and immunogenicity of ChAdOx1 nCoV-19 vaccine administered in a prime-boost regimen in young and old adults (COV002): a single-blind, randomised, controlled, phase 2/3 trial.

Lancet (London, England)

Ramasamy MN, Minassian AM, Ewer KJ, Flaxman AL, Folegatti PM, Owens DR, Voysey M, Aley PK, Angus B, Babbage G, Belij-Rammerstorfer S, Berry L, Bibi S, Bittaye M, Cathie K, Chappell H, Charlton S, Cicconi P, Clutterbuck EA, Colin-Jones R, Dold C, Emary KRW, Fedosyuk S, Fuskova M, Gbesemete D, Green C, Hallis B, Hou MM, Jenkin D, Joe CCD, Kelly EJ, Kerridge S, Lawrie AM, Lelliott A, Lwin MN, Makinson R, Marchevsky NG, Mujadidi Y, Munro APS, Pacurar M, Plested E, Rand J, Rawlinson T, Rhead S, Robinson H, Ritchie AJ, Ross-Russell AL, Saich S, Singh N, Smith CC, Snape MD, Song R, Tarrant R, Themistocleous Y, Thomas KM, Villafana TL, Warren SC, Watson MEE, Douglas AD, Hill AVS, Lambe T, Gilbert SC, Faust SN, Pollard AJ, Oxford COVID Vaccine Trial Group

Clinical Outcomes in Patients with Relapsed/Refractory Acute Myeloid Leukemia Treated with Gilteritinib Who Received Prior Midostaurin or Sorafenib.

Blood

Alexander E. Perl, Jessica K. Altman, Naoko Hosono, Pau Montesinos, Nikolai A. Podoltsev, Giovanni Martinelli, Catherine C. Smith, Mark Levis, Christoph Röllig, Marco Groß-Langenhoff, Nahla Hasabou, Qiaoyang Lu, Ramon V. Tiu

Cognitive Dysfunction Associated with Tyrosine Kinase Inhibitors in Patients with CML in Chronic Phase.

Blood

Kelly L. Schoenbeck, Sirisha Tummala, Neha G. Goyal, Catherine C. Smith, Neil P. Shah

Concurrent FLT3 Inhibitor and IDH Inhibitor Therapy in Patients with Acute Myeloid Leukemia (AML).

Blood

Amir T. Fathi, Alexander E. Perl, Mark Levis, Catherine C. Smith, Andrew M. Brunner, Koichi Takahashi, Philip C. Amrein, Hanno R. Hock, Gabriela S. Hobbs, Rupa Narayan, Jenna A. Moran, Meghan Bergeron, Julia E. Foster, Naval Daver, Courtney D. DiNardo

Efficacy and Safety of Venetoclax in Combination with Gilteritinib for Relapsed/Refractory FLT3-Mutated Acute Myeloid Leukemia in the Expansion Cohort of a Phase 1b Study.

Blood

Naval Daver, Jessica K. Altman, Joseph Maly, Mark Levis, Ellen Ritchie, Mark Litzow, James K. McCloskey, Catherine C. Smith, Gary J. Schiller, Terrence Bradley, Ramon V. Tiu, Wan-Jen Hong, Bo Tong, Qin Qin, Kimberley Dilley, Alexander E. Perl

Excellent Outcomes Achieved with Medical Management of Chronic Phase CML Patients with Non-Hematologic TKI Intolerance.

Blood

Kelly L. Schoenbeck, Sirisha Tummala, Rebecca L. Olin, Neha G. Goyal, Anand Dhruva, Lloyd E. Damon, Aaron C. Logan, Catherine C. Smith, Neil P. Shah

Pharmacokinetic Profile of Gilteritinib: A Novel FLT-3 Tyrosine Kinase Inhibitor.

Clinical pharmacokinetics

James AJ, Smith CC, Litzow M, Perl AE, Altman JK, Shepard D, Kadokura T, Souda K, Patton M, Lu Z, Liu C, Moy S, Levis MJ, Bahceci E

FLT3 Inhibition in Acute Myeloid Leukemia.

Clinical lymphoma, myeloma & leukemia

Smith CC

A phase 1/2 study of the oral FLT3 inhibitor pexidartinib in relapsed/refractory FLT3-ITD-mutant acute myeloid leukemia.

Blood advances

Smith CC, Levis MJ, Frankfurt O, Pagel JM, Roboz GJ, Stone RM, Wang ES, Severson PL, West BL, Le MH, Kayser S, Lam B, Hsu HH, Zhang C, Bollag G, Perl AE

Gilteritinib is a clinically active FLT3 inhibitor with broad activity against FLT3 kinase domain mutations.

Blood advances

Tarver TC, Hill JE, Rahmat L, Perl AE, Bahceci E, Mori K, Smith CC

The growing landscape of FLT3 inhibition in AML.

Hematology. American Society of Hematology. Education Program

Smith CC

Combined Single-Cell DNA Genotyping and Protein Quantification (DAb-seq) in Acute Myeloid Leukemias Reveals Distinct Immunophenotypic Subsets Among Pathogenic Clones.

Blood

Benjamin Demaree, Cyrille Delley, Cheryl Peretz, Harish Vasudevan, David Ruff, Aik Ooi, Catherine C. Smith, Adam Abate

Direct Oral Anticoagulant Use and Outcomes in Patients with High and Intermediate Risk BCR-ABL-Negative Myeloproliferative Neoplasms.

Blood

Kelly L. Schoenbeck, Giselle Salmasi, Miguel Carlos Cerejo, Patricia A. Cornett, Lloyd E. Damon, Aaron C. Logan, Rebecca L. Olin, Andrew D. Leavitt, Catherine C. Smith

Efficacy of Venetoclax Combination Therapy with Hypomethylating Agents in Patients with Relapsed Acute Myeloid Leukemia after Allogeneic Hematopoietic Cell Transplantation.

Blood

Varun Mittal, Mimi Lo, Lloyd E. Damon, Karin L. Gaensler, Thomas G. Martin, Rebecca L. Olin, Catherine C. Smith, Aaron C. Logan

Emerging Mutations at Relapse in Patients with FLT3-Mutated Relapsed/Refractory Acute Myeloid Leukemia Who Received Gilteritinib Therapy in the Phase 3 Admiral Trial.

Blood

Catherine C. Smith, Mark J. Levis, Alexander E. Perl, Giovanni Martinelli, Andreas Neubauer, Ellin Berman, Pau Montesinos, Maria R. Baer, Richard A. Larson, Wen-Chien Chou, Hisayuki Yokoyama, Christian Recher, Sung-Soo Yoon, Jason E. Hill, Matt Rosales, Erkut Bahceci

Single Cell Sequencing Reveals Evolution of Tumor Heterogeneity of Acute Myeloid Leukemia on Quizartinib.

Blood

Cheryl A Cohler Peretz, Lisa H F McGary, Tanya F Kumar, J Hunter Jackson, Jose Jacob, Robert Durruthy-Durruthy, Chunxiao Zhang, Mark J. Levis, Alexander E. Perl, Anskar Yu Hung Leung, Catherine C. Smith

Symptom Assessment in Patients with BCR-ABL-Negative Myeloproliferative Neoplasms at an Academic Medical Institution.

Blood

Kelly L. Schoenbeck, Miguel Carlos Cerejo, Patricia A. Cornett, Lloyd E. Damon, Karin L. Gaensler, Andrew D. Leavitt, Aaron C. Logan, Gabriel N. Mannis, Rebecca L. Olin, Giselle Salmasi, Peter H. Sayre, Neil P. Shah, Anand Dhruva, Chloe E. Atreya, Catherine C. Smith

Fusion driven JMML: a novel CCDC88C-FLT3 fusion responsive to sorafenib identified by RNA sequencing.

Leukemia

Chao AK, Meyer JA, Lee AG, Hecht A, Tarver T, Van Ziffle J, Koegel AK, Golden C, Braun BS, Sweet-Cordero EA, Smith CC, Dvorak CC, Loh ML, Stieglitz E

Functional Status as Measured by Geriatric Assessment Predicts Inferior Survival in Older Allogeneic Hematopoietic Cell Transplantation Recipients.

Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation

Huang LW, Sheng Y, Andreadis C, Logan AC, Mannis GN, Smith CC, Gaensler KML, Martin TG, Damon LE, Steinman MA, Huang CY, Olin RL

Use of FLT3 Inhibitors in AML.

Clinical Lymphoma Myeloma & Leukemia

Catherine C. Smith

Clonal Selection with RAS Pathway Activation Mediates Secondary Clinical Resistance to Selective FLT3 Inhibition in Acute Myeloid Leukemia.

Cancer discovery

McMahon CM, Ferng T, Canaani J, Wang ES, Morrissette JJD, Eastburn DJ, Pellegrino M, Durruthy-Durruthy R, Watt CD, Asthana S, Lasater EA, DeFilippis R, Peretz CAC, McGary LHF, Deihimi S, Logan AC, Luger SM, Shah NP, Carroll M, Smith CC, Perl AE

Functional Status Is Associated with Outcomes after Allogeneic Stem Cell Transplantation for Older Patients with Hematologic Malignancies.

Blood

Li-Wen Huang, Chiung-Yu Huang, Charalambos Andreadis, Aaron C. Logan, Gabriel N. Mannis, Catherine C. Smith, Karin M. Gaensler, Thomas G. Martin, Lloyd E. Damon, Michael A. Steinman, Rebecca L. Olin

Impact of Minimal Residual Disease and Achievement of Complete Remission/Complete Remission with Partial Hematologic Recovery (CR/CRh) on Overall Survival Following Treatment with Gilteritinib in Patients with Relapsed/Refractory (R/R) Acute Myeloid Leuke

Blood

Mark J. Levis, Alexander E. Perl, Jessica K. Altman, Jorge E. Cortes, Catherine C. Smith, Maria R. Baer, David F. Claxton, Joseph G. Jurcic, Ellen K. Ritchie, Stephen A. Strickland, Raoul Tibes, Jason E. Hill, Matt Rosales, Erkut Bahceci

Longitudinal Monitoring of AML Tumors with High-Throughput Single-Cell DNA Sequencing Reveals Rare Clones Prognostic for Disease Progression and Therapy Response.

Blood

Dennis J. Eastburn, Christine M. McMahon, Robert Durruthy-Durruthy, Martin Carroll, Catherine C. Smith, Alexander E. Perl

Quizartinib, an FLT3 inhibitor, as monotherapy in patients with relapsed or refractory acute myeloid leukaemia: an open-label, multicentre, single-arm, phase 2 trial.

The Lancet. Oncology

Cortes J, Perl AE, Döhner H, Kantarjian H, Martinelli G, Kovacsovics T, Rousselot P, Steffen B, Dombret H, Estey E, Strickland S, Altman JK, Baldus CD, Burnett A, Krämer A, Russell N, Shah NP, Smith CC, Wang ES, Ifrah N, Gammon G, Trone D, Lazzaretto D, Levis M

Post hoc exploratory analysis of two phase 2 trials of quizartinib monotherapy in patients (pts) with FLT3 -ITD–mutated (mu) relapsed/refractory (R/R) AML with or without prior 1st-generation FLT3 tyrosine kinase inhibitors (TKI) treatment.

Journal of Clinical Oncology

Mark J. Levis, Catherine Choy Smith, Koji Ishizuka, Ken Kobayashi, Meena Arunachalam, Yibin Wang, Deborah Lazzaretto, Jorge E. Cortes

Selective inhibition of FLT3 by gilteritinib in relapsed or refractory acute myeloid leukaemia: a multicentre, first-in-human, open-label, phase 1-2 study.

The Lancet. Oncology

Perl AE, Altman JK, Cortes J, Smith C, Litzow M, Baer MR, Claxton D, Erba HP, Gill S, Goldberg S, Jurcic JG, Larson RA, Liu C, Ritchie E, Schiller G, Spira AI, Strickland SA, Tibes R, Ustun C, Wang ES, Stuart R, Röllig C, Neubauer A, Martinelli G, Bahceci E, Levis M

Deep molecular response to gilteritinib to improve survival in FLT3 mutation-positive relapsed/refractory acute myeloid leukemia.

Journal of Clinical Oncology

Jessica K. Altman, Alexander E. Perl, Jorge E. Cortes, Catherine Choy Smith, Mark Robert Litzow, Jason E Hill, Richard A. Larson, Charles Liu, Ellen K. Ritchie, Stephen Anthony Strickland, Eunice S. Wang, Andreas Neubauer, Giovanni Martinelli, Erkut Bahceci, Mark J. Levis

Heterogeneous resistance to quizartinib in acute myeloid leukemia revealed by single-cell analysis.

Blood

Smith CC, Paguirigan A, Jeschke GR, Lin KC, Massi E, Tarver T, Chin CS, Asthana S, Olshen A, Travers KJ, Wang S, Levis MJ, Perl AE, Radich JP, Shah NP

Pharmacokinetics and pharmacodynamics of gilteritinib in patients with relapsed or refractory acute myeloid leukemia.

Journal of Clinical Oncology

Catherine Choy Smith, Mark J. Levis, Mark Robert Litzow, Alexander E. Perl, Jessica K. Altman, Stan Gill, Geoffrey Yuen, Peter Bonate, Takeshi Kadokura, Angela Joubert James, Charles Liu, Itsuro Nagase, Ogert Fisniku, Erkut Bahceci

The MERTK/FLT3 inhibitor MRX-2843 overcomes resistance-conferring FLT3 mutations in acute myeloid leukemia.

JCI insight

Minson KA, Smith CC, DeRyckere D, Libbrecht C, Lee-Sherick AB, Huey MG, Lasater EA, Kirkpatrick GD, Stashko MA, Zhang W, Jordan CT, Kireev D, Wang X, Frye SV, Earp HS, Shah NP, Graham DK

Long-term outcomes of patients with intermediate-risk acute myeloid leukemia treated with autologous hematopoietic cell transplant in first complete remission.

Leukemia & lymphoma

Mannis GN, Martin TG, Damon LE, Logan AC, Olin RL, Flanders MD, Ai WZ, Gaensler KM, Kaplan LD, Sayre PH, Smith CC, Wolf JL, Andreadis C

Resistance to FLT3 Inhibitors.

Resistance to Tyrosine Kinase Inhibitors

Timothy Ferng, Catherine Choy Smith

Identifying Drug-Resistant Mutations in Ebf1-Pdgfrb Ph-like Acute Lymphoblastic Leukemia.

Blood

Thai Hoa Tran, Jonathan Van Nguyen, Catherine C. Smith, Kathryn G. Roberts, Charles G. Mullighan, Neil P. Shah, Mignon L. Loh

Pharmacokinetic Profile and Pharmacodynamic Effects of ASP2215, a Selective, Potent Inhibitor of FLT3/AXL, in Patients with Relapsed or Refractory Acute Myeloid Leukemia: Results from a First-in-Human Phase 1/2 Study.

Blood

Catherine C. Smith, Mark J. Levis, Mark R Litzow, Alexander E. Perl, Jessica K. Altman, Stanley C. Gill, Takeshi Kadokura, Geoffrey J. Yuen, Ogert Fisniku, Charles Liu, Itsuro Nagase, Briana Sargent, Noreen Welter, Erkut Bahceci

Recurrent Mutations in CCND3 Confer Clinical Resistance to FLT3 Inhibitors.

Blood

Catherine C. Smith, Aaron D. Viny, Evan S. Massi, Cyriac Kandoth, Nicholas D. Socci, Henry Hsu, Brian West, Gideon Bollag, Barry S. Taylor, Ross L. Levine, Neil P. Shah

Results of a first-in-human, phase I/II trial of ASP2215, a selective, potent inhibitor of FLT3/Axl in patients with relapsed or refractory (R/R) acute myeloid leukemia (AML).

Journal of Clinical Oncology

Mark J. Levis, Alexander E. Perl, Jessica K. Altman, Jorge E. Cortes, Ellen K. Ritchie, Richard A. Larson, Catherine Choy Smith, Eunice S. Wang, Stephen Anthony Strickland, Maria R. Baer, Mark Robert Litzow, David Claxton, Gary J. Schiller, Celalettin Ustun, Charles Liu, Stan Gill, Briana Sargent, Erkut Bahceci

Characterizing and Overriding the Structural Mechanism of the Quizartinib-Resistant FLT3 "Gatekeeper" F691L Mutation with PLX3397.

Cancer discovery

Smith CC, Zhang C, Lin KC, Lasater EA, Zhang Y, Massi E, Damon LE, Pendleton M, Bashir A, Sebra R, Perl A, Kasarskis A, Shellooe R, Tsang G, Carias H, Powell B, Burton EA, Matusow B, Zhang J, Spevak W, Ibrahim PN, Le MH, Hsu HH, Habets G, West BL, Bollag G, Shah NP

Computer aided drug discovery of highly ligand efficient, low molecular weight imidazopyridine analogs as FLT3 inhibitors.

European journal of medicinal chemistry

Frett B, McConnell N, Smith CC, Wang Y, Shah NP, Li HY

A phase I study of targeted, dose-escalated intravenous busulfan in combination with etoposide as myeloablative therapy for autologous stem cell transplantation in acute myeloid leukemia.

Clinical lymphoma, myeloma & leukemia

Mannis GN, Andreadis C, Logan AC, Damon LE, Benet LZ, Ai WZ, Gaensler KM, Kaplan LD, Koplowicz YB, Linker CA, Olin RL, Sayre PH, Smith CC, Sudhindra A, Venstrom JM, Wolf JL, Martin TG

Long-Term Outcomes of 133 Patients with Intermediate-Risk Acute Myeloid Leukemia Treated with High-Dose Chemotherapy and Autologous Stem Cell Rescue in First Complete Remission.

Biology of Blood and Marrow Transplantation

Gabriel N. Mannis, Thomas G. Martin, Lloyd E. Damon, Aaron C. Logan, Weiyun Ai, Karin M.L. Gaensler, Lawrence D. Kaplan, Anuj Mahindra, Rebecca L. Olin, Peter Sayre, Catherine C. Smith, Jeffrey L. Wolf, Jimmy Hwang, Charalambos Andreadis

Overcoming myelosuppression due to synthetic lethal toxicity for FLT3-targeted acute myeloid leukemia therapy.

eLife

Warkentin AA, Lopez MS, Lasater EA, Lin K, He BL, Leung AY, Smith CC, Shah NP, Shokat KM

MRX2843, a Novel Dual MerTK-FLT3 Inhibitor with Activity Against Resistance-Conferring FLT3 Mutations in Acute Myeloid Leukemia.

Blood

Katherine A Minson, Catherine C Smith, Alisa B. Lee-Sherick, Deborah DeRyckere, Elisabeth Lasater, Amanda A. Hill, Xiaodong Wang, Stephen V. Frye, H. Shelton Earp, Neil P. Shah, Douglas K. Graham

Regulation of Stat5 by FAK and PAK1 in Oncogenic FLT3- and KIT-Driven Leukemogenesis.

Cell reports

Chatterjee A, Ghosh J, Ramdas B, Mali RS, Martin H, Kobayashi M, Vemula S, Canela VH, Waskow ER, Visconte V, Tiu RV, Smith CC, Shah N, Bunting KD, Boswell HS, Liu Y, Chan RJ, Kapur R

Hematopoietic cell transplantation comorbidity index (HCT-CI) is predictive of adverse events and overall survival in older allogeneic transplant recipients.

Journal of geriatric oncology

Keller JW, Andreadis C, Damon LE, Kaplan LD, Martin TG, Wolf JL, Ai WZ, Venstrom JM, Smith CC, Gaensler KM, Hwang J, Olin RL

FLT3 inhibitors in AML: are we there yet?

Current hematologic malignancy reports

Sudhindra A, Smith CC

Crenolanib is a selective type I pan-FLT3 inhibitor.

Proceedings of the National Academy of Sciences of the United States of America

Smith CC, Lasater EA, Lin KC, Wang Q, McCreery MQ, Stewart WK, Damon LE, Perl AE, Jeschke GR, Sugita M, Carroll M, Kogan SC, Kuriyan J, Shah NP

A Phase 1 Study of Targeted, Dose-Escalated Intravenous Busulfan in Combination with Etoposide As Preparative Therapy for Autologous Stem Cell Transplant in Acute Myeloid Leukemia.

Biology of Blood and Marrow Transplantation

Gabriel N. Mannis, Lloyd E. Damon, Weiyun Ai, Charalambos Andreadis, Karin ML. Gaensler, Lawrence D. Kaplan, Aaron C. Logan, Anuj Mahindra, Rebecca L. Olin, Peter Sayre, Catherine C. Smith, Jeffrey M. Venstrom, Jeffrey L. Wolf, Thomas G. Martin

Comprehensive Geriatric Assessment Identifies Significant Functional Impairments in Older Hematopoietic Cell Transplant Recipients.

Biology of Blood and Marrow Transplantation

Rebecca L. Olin, Charalambos Andreadis, Thomas G. Martin, Jeffrey L. Wolf, Lawrence D. Kaplan, Weiyun Ai, Jeffrey M. Venstrom, Aaron C. Logan, Catherine C. Smith, Karin ML. Gaensler, Jimmy Hwang, Lloyd E. Damon

Phase I evaluation of XL019, an oral, potent, and selective JAK2 inhibitor.

Leukemia research

Verstovsek S, Tam CS, Wadleigh M, Sokol L, Smith CC, Bui LA, Song C, Clary DO, Olszynski P, Cortes J, Kantarjian H, Shah NP

Disease diversity and FLT3 mutations.

Proceedings of the National Academy of Sciences of the United States of America

Smith CC

Delayed Hematopoietic Recovery After Autologous Stem Cell Transplantation In Patients Receiving Arsenic Trioxide-Based Therapy For Acute Promyelocytic Leukemia.

Blood

Gabriel N. Mannis, Lloyd E Damon, Weiyun Z. Ai, Charalambos Andreadis, Karin ML Gaensler, Neel K. Gupta, Lawrence D. Kaplan, Andrew D. Leavitt, Aaron C. Logan, Rebecca L. Olin, Peter H. Sayre, Catherine C Smith, Jeffrey M Venstrom, Thomas G Martin

Preclinical and Clinical Resistance Mechanisms To The Investigational Selective FLT3 Inhibitor PLX3397 In FLT3-ITD+ Acute Myeloid Leukemia (AML).

Blood

Catherine C Smith, Kimberly Lin, Elisabeth Lasater, Whitney Stewart, Lauren E Damon, Andrew Kasarskis, Ali Bashir, Matthew Pendleton, Robert Sebra, Alexander E Perl, Mai H Le, Chao Zhang, Brian West, Neil P. Shah

Association of age-dependent liver injury and fibrosis with immune cell populations.

Liver international : official journal of the International Association for the Study of the Liver

Collins BH, Holzknecht ZE, Lynn KA, Sempowski GD, Smith CC, Liu S, Parker W, Rockey DC

The Role of Kinase Inhibitors in the Treatment of Patients with Acute Myeloid Leukemia.

American Society of Clinical Oncology Educational Book

Catherine C. Smith, Neil P. Shah

Activity of ponatinib against clinically-relevant AC220-resistant kinase domain mutants of FLT3-ITD.

Blood

Smith CC, Lasater EA, Zhu X, Lin KC, Stewart WK, Damon LE, Salerno S, Shah NP

Hematopoietic Cell Transplantation Comorbidity Index (HCT-CI) Is Predictive of Serious Adverse Events and Overall Survival in Older Allogeneic Transplant Recipients.

Biology of Blood and Marrow Transplantation

Jesse W. Keller, Lloyd E. Damon, Lawrence D. Kaplan, Thomas G. Martin, Jeffrey L. Wolf, Weiyun Z. Ai, Charalambos Andreadis, Jeffrey M. Venstrom, Catherine C. Smith, Karin M.L. Gaensler, Jimmy Hwang, Rebecca L. Olin

The role of kinase inhibitors in the treatment of patients with acute myeloid leukemia.

American Society of Clinical Oncology educational book. American Society of Clinical Oncology. Annual Meeting

Smith CC, Shah NP

Constitutively Activating Mutations At the FLT3 Activation Loop Residue D835 Are Associated with Clinical Resistance to AC220.

Blood

Catherine C Smith, Jason Chin, Elisabeth Lasater, Amy L. Paguirigan, Kimberly Lin, Whitney Stewart, Sara Salerno, Lauren E Damon, Mark J. Levis, Alexander E Perl, Kevin Travers, Andrew Kasarskis, Jerald P. Radich, Neil Shah

Crenolanib (CP-868,596) Is a Potent and Selective Type I FLT3 Inhibitor That Retains Activity Against AC220 Resistance-Causing FLT3 Kinase Domain Mutants.

Blood

Catherine C Smith, Elisabeth Lasater, Melissa Mccreery, Kimberly Lin, Whitney Stewart, Lauren E Damon, Alexander E Perl, Grace R Jeschke, Mayumi Sugita, Martin Carroll, Scott C. Kogan, Neil Shah

Single Molecule Real Time (SMRT™) Sequencing Sensitively Detects the Evolution of Polyclonal and Compound BCR-ABL Mutations in Patients Who Relapse On Kinase Inhibitor Therapy.

Blood

Catherine C Smith, Michael Brown, Wendy T Parker, Kimberly Lin, Kevin Travers, Susana Wang, Susan Branford, Neil Shah

Mechanisms of Resistance to Targeted Therapies in Acute Myeloid Leukemia and Chronic Myeloid Leukemia.

American Society of Clinical Oncology Educational Book

Catherine C. Smith, Neil P. Shah

Abstract 1893: Activating NRAS mutations are associated with FLT3-independent resistance to the clinically active FLT3 inhibitor AC220 in vitro.

Experimental and Molecular Therapeutics

Elisabeth A. Lasater, Catherine C. Smith, Neil P. Shah

Validation of ITD mutations in FLT3 as a therapeutic target in human acute myeloid leukaemia.

Nature

Smith CC, Wang Q, Chin CS, Salerno S, Damon LE, Levis MJ, Perl AE, Travers KJ, Wang S, Hunt JP, Zarrinkar PP, Schadt EE, Kasarskis A, Kuriyan J, Shah NP

Mechanisms of resistance to targeted therapies in acute myeloid leukemia and chronic myeloid leukemia.

American Society of Clinical Oncology educational book. American Society of Clinical Oncology. Annual Meeting

Smith CC, Shah NP

A Phase II Open-Label, Ac220 Monotherapy Efficacy Study In Patients with Refractory/Relapsed Flt3-Itd Positive Acute Myeloid Leukemia: Updated Interim Results.

Blood

Jorge E. Cortes, Alexander E Perl, Catherine C. Smith, Tibor Kovacsovics, Herve Dombret, Hartmut Dohner, Bjorn Steffen, Arnaud Pigneux, Philippe Rousselot, Juergen Krauter, Giovanni Martinelli, Elihu H. Estey, Alan K Burnett, Anthony D. Ho, Norbert Ifrah, Theo de Witt, Robert Corringham, Joyce James, David Lilienfeld, Eugen Leo, Guy Gammon, Mark J. Levis

Activity of Allosteric, Switch-Pocket, ABL/FLT3 Kinase Inhibitor DCC2036 Against Cultured and Primary AML Progenitors with FLT-ITD or FLT3 Kinase Domain Mutations.

Blood

Warren Fiskus, Catherine C. Smith, Jacqueline Smith, Scott C. Wise, Elisabeth Lasater, Lauren E. Damon, Sara Salerno, Allan Fleming, Ruben Reyes, Siddhartha Ganguly, Mark S. Berger, Tom J. Rutkoski, Joseph McGuirk, Neil Shah, Kapil N. Bhalla

PLX3397 Is An Investigational Selective FLT3 Inhibitor That Retains Activity Against the Clinically-Relevant FLT3-ITD/F691L “Gatekeeper” Mutation in Vitro.

Blood

Catherine C. Smith, Alexander E Perl, Elisabeth Lasater, Chao Zhang, Grace R Jeschke, Lauren E. Damon, Martin Carroll, Neil P. Shah

Single Molecule Real Time (SMRT™) Sequencing Sensitively Detects Polyclonal and Compound BCR-ABL in Patients Who Relapse on Kinase Inhibitor Therapy,.

Blood

Catherine C. Smith, Michael Brown, Jason Chin, Corynn Kasap, Sara Salerno, Lauren E. Damon, Kevin Travers, Susana Wang, Andrew Kasarskis, Eric Schadt, Neil Shah

Validation of FLT3-ITD As a Therapeutic Target in Human Acute Myeloid Leukemia.

Blood

Catherine C. Smith, Jason Chin, Qi Wang, Sara Salerno, Lauren E. Damon, Jeremy P. Hunt, Mark J. Levis, Alexander E Perl, Kevin Travers, Susana Wang, Andrew Kasarskis, Eric Schadt, John Kuriyan, Neil Shah

Is it downhill from here? Eliminating leukemic stem cells and curing chronic myeloid leukemia.

Clinical cancer research : an official journal of the American Association for Cancer Research

Smith CC, Shah NP

Cost-effectiveness of private umbilical cord blood banking.

Obstetrics and gynecology

Kaimal AJ, Smith CC, Laros RK, Caughey AB, Cheng YW

Fluorometric assay for N-acetylprocainamide.

Clinical chemistry

Matusik E, Gibson TP

Metal substitutions incarbonic anhydrase: a halide ion probe study.

Biochemical and biophysical research communications

Smith RJ, Bryant RG